Company Presentations

View the 2020 Slate of Presenting Companies!

This meeting features presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

Collectively, the 2020 slate of 120 presenting companies are working on a vast number of unique products, tools and services. Don’t miss your chance to meet with senior executives from the following companies.

All company presentations will be pre-recorded and available on-demand for attendees to watch at their convenience.

2020 Company Presenters

AGTC
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Healios_85
Homology



Precision-Biosciences_90
ReNeuron_80
Sangamo_80SCM-Lifescience
StemBioSys
ViaCyte

ACCELERATED BIOSCIENCES
Yuta Lee, CEO
Carlsbad, CA
(Private)
Accelerated Biosciences is a regenerative medicine company. We are sourcing the highest quality immune-privileged human Trophoblast Stem Cells (hTSCs) with no socio-ethical controversy. We are focused on supplying well-characterized cell types from its primary hTSC source. hTS cells are pluripotent, immune-privileged, ethically derived, virus and pathogen-free, easy to manufacture, genetically stable, and GTP compliant. We have optimized expansion to 75+ population doublings with a 22 hour doubling time. We have a robust and unencumbered IP portfolio of 28 issued patents worldwide. We are available for partnering and licensing opportunities.
www.acceleratedbio.com

ADAPTIMMUNE
Adrian Rawcliffe, CEO
Philadelphia, PA
(NASDAQ: ADAP)
Adaptimmune, a leader in T-cell therapy, has clinical trials ongoing for three wholly owned SPEAR T-cells (ADP-A2M4, ADP-A2M4CD8, and ADP-A2AFP) in multiple solid tumor indications. The Company’s unique SPEAR (Specific Peptide Enhanced Affinity Receptor) T‑cell platform enables the engineering of T-cells to target and destroy cancer, including solid tumors. We have generated a strong pipeline of affinity-enhanced T-cell therapies, with multiple INDs open. We use these therapies to harness the body’s own immune system to find and destroy diseased cells. Our SPEAR T-cell therapies offer promise to patients that often have no other options. We are working hard to make that promise a reality. Adaptimmune partners with industry leaders in all areas of it’s business.
www.adaptimmune.com

ADVERUM BIOTECHNOLOGIES
Laurent Fischer, M.D., CEO
Redwood City, CA
(NASDAQ: ADVM)
Adverum is a clinical-stage gene therapy company targeting unmet medical need in ocular and rare diseases. We develop gene therapy product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Our core capabilities include clinical development, novel vector discovery, and in-house manufacturing expertise, specifically in scalable process development, assay development, and current Good Manufacturing Practices (cGMP) quality control. Our team has extensive clinical development experience and is laser-focused on advancing novel gene therapies for patients.
www.adverum.com

AEGLE THERAPEUTICS
Shelley Hartman, CEO
Miami, FL
(Private)
Aegle Therapeutics is developing a first-in-class therapy using extracellular vesicles, including exosomes, secreted by allogeneic bone marrow derived mesenchymal stem cells to treat severe dermatological conditions including dystrophic epidermolysis bullosa, a rare pediatric blistering disease.
www.aegletherapeutics.com

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AGTC
Sue Washer, President and CEO
Alachua, FL and Cambridge, MA
(NASDAQ: AGTC)
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic, and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. Initially focusing on ophthalmology, our goal is to preserve or hopefully be able to improve vision in some cases. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Our preclinical programs build on our industry-leading AAV manufacturing technology and expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology, and CNS disorders.
www.agtc.com

AIVITA BIOMEDICAL
Hans Keirstead, Ph.D., CEO
Irvine, CA
(Private)
AIVITA Biomedical is a personalized vaccine company designing immuno-oncology therapies that target the seed of all cancers: tumor-initiating cells. We take a unique pan-antigenic approach — targeting all neoantigens, rather than a select few — allowing our treatments to overcome cancer’s tendency to mutate over time. Our patient-specific treatments have shown tremendous promise in eradicating tumors without harmful side effects in our melanoma and glioblastoma clinical studies. We have also leveraged our approach to create a proprietary skincare line, which dedicates 100% of net proceeds towards advancing revolutionary treatments for patients with cancer, and continue to explore additional applications in human health.
www.aivitabiomedical.com

AKRON BIOTECH
David Smith, President and Chief Commercial Officer
Boca Raton, FL
(Private)
Akron Biotech is an innovative biotechnology company with a strategic focus on supplying GMP-qualified raw materials and services to the regenerative medicine industry. Akron manufactures a range of products for cell therapy discovery, development, and commercialization. These products include growth factors, sera and purified proteins, custom-made cell culture media, tailored scaffolds with biomaterials, and proprietary cryopreservation formulations. In addition, our services and capabilities such as raw materials qualification, logistics and packaging optimization, bioassay design, validation and regulatory services are one of the many opportunities for Akron to provide our customers unique knowledge and expertise. As an ISO-compliant company, Akron supports clients with rigorous documentation and quality standards to fullfil their regulatory demands. Our unique capabilities allow us to seamlessly transition from R&D to preclinical and clinical development with minimal change control and thus drive the emerging regenerative medicine sector to unmet clinical needs through affordable and seamless manufacturing options.
www.akronbiotech.com

ALLOVIR
Ann Leen, Ph.D., Co-Founder and Chief Scientific Officer
Cambridge, MA and Houston, TX
(NASDAQ: ALVR)
AlloVir is a leading late clinical-stage cell therapy company developing highly innovative allogeneic T cell therapies to treat and prevent devastating viral diseases. Our innovative and proprietary virus-specific T cell, or VST, therapy platform allows us to generate off-the-shelf VSTs designed to restore immunity in patients with T cell deficiencies who are at risk from the devastating consequences of viral diseases. There is an urgent medical need for therapies to treat a large number of patients suffering from viral diseases who currently have limited or no treatment options. To date, we have generated five innovative, allogeneic, off-the-shelf VST therapy candidates targeting 12 different devastating viruses, the most advanced of which has successfully completed a proof-of-concept trial across five viruses and is entering initial pivotal trials. www.allovir.com

AMERICAN GENE TECHNOLOGIES
Jeff Galvin, CEO
Rockville, MD
(Private)
American Gene Technologies (AGT) is a clinical-stage gene and cell therapy company with a gene-delivery platform for the rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. It expects to take its patented lead candidate for an HIV cure into the clinic in 2020. AGT has received eight patents for its immuno-oncology approach to stimulate gamma-delta (γδ) T-cells to destroy a variety of solid tumors. It has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. This treatment was granted an FDA Orphan Drug Designation and it is expected to reach the clinic in 2022.
www.americangene.com

AMICUS THERAPEUTICS
Bradley Campbell, President and Chief Operating Officer
Cranbury, NJ
(NASDAQ: FOLD)
Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel high quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. The Company’s portfolio of product opportunities is led by a novel medicine for Fabry disease, Galafold®, that has achieved widespread global approval, a differentiated biologic, AT-GAA, for Pompe disease in the clinic, and an industry-leading rare disease gene therapy portfolio.
www.amicusrx.com

ANEMOCYTE
Stefano Baila, Ph.D, Director, Operations and Business Development
Gerenzano, Italy
(Private)
Anemocyte is a biotech manufacturing organization active in the field of cell and gene therapies (CGTs) that addresses the sector’s needs by offering one stop shop solutions and fostering exciting innovations.
www.anemocyte.com

APOSCIENCE
Hendrik Jan Leonard Ankersmit, M.D., CEO
Vienna, Austria
(Private)
Aposcience is an Austrian biopharmaceutical company headquartered in Vienna. The company has developed a proprietary, innovative therapeutic derived from white blood cells. APOSEC is the secretome of stressed leukocytes and is manufactured under GMP conditions. Batch-to-batch comparability, stability, and viral clearance were proven to regulatory authority. In December 2019 the randomized, double-blinded, multi-centre clinical Phase II trial was approved national authorities; the indication for this trial is diabetic foot ulcer.
www.aposcience.com

ARTIVA BIOTHERAPEUTICS
Tom Farrell, President and CEO
San Diego, CA
(Private)
Artiva Biotherapeutics is a private biotech company advancing a pipeline of off-the-shelf, allogeneic NK cell therapies, including CAR-NK cell therapies, for the treatment of hematologic cancers or solid tumors. Artiva was founded on the understanding that NK cells hold enormous therapeutic potential as cancer therapies, and the dedication and expertise to overcome technological barriers in the scaling and manufacturing of these cells. At Artiva, our mission is to deliver highly effective cell therapies that are also safe and immediately available and accessible to any cancer patient who stands to benefit. Artiva’s platform delivers scalability, quality, and cryopreservation to support a pipeline of safe, effective, versatile, and accessible product candidates.
www.artivabio.com

ARUVANT
Will Chou, M.D., CEO
New York, NY
(Private)
Aruvant Sciences is a private clinical-stage gene therapy company focused on developing and commercializing transformative therapies for patients with severe hematological conditions. The company’s near-term focus is on sickle cell disease, with a subsequent expansion into β-thalassemia. Aruvant’s lead candidate, ARU-1801, consists of autologous cells that are genetically modified with a lentiviral vector that encodes a novel, highly potent, anti-sickling γ-globin. ARU-1801 was designed to address the limitations of current curative treatment options, such as low donor availability and the need for more toxic, intensive chemotherapy conditioning regimens for stem cell transplants.
www.aruvant.com

ASKBIO
Sheila Mikhail, CEO
Research Triangle Park, NC
(Private)
Founded in 2001 by gene therapy pioneers, Asklepios BioPharmaceutical is a fully-integrated, privately-held gene therapy company with global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, UK. We are dedicated to develop life saving medicines that cure genetic disease. Our clinical-stage programs include Pompe disease and congestive heart failure, as well as out-licensed clinical indications for hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne muscular dystrophy (Bamboo Therapeutics acquired by Pfizer). Our AAV gene therapy platform includes Pro10™, an industry-leading, proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. An early innovator in the space, AskBio holds more than 500 patents in areas such as AAV production, and chimeric and self-complementary capsids.
www.askbio.com

ASPECT BIOSYSTEMS
Tamer Mohamed, CEO
Vancouver, Canada
(Private)
Aspect Biosystems is a privately-held biotechnology platform company pioneering the microfluidic 3D bioprinting of human tissues. The Company’s proprietary technology has the potential to shape every aspect of human health by enabling the creation of human tissues for medical research, therapeutic discovery, and regenerative medicine. Aspect is focused on partnering with academic institutions and biopharma companies to facilitate high-value discovery and development. The Company is also advancing internal tissue therapeutic programs for regenerative medicine, with an initial focus on metabolic diseases, and musculoskeletal injuries and disorders.
www.aspectbiosystems.com

ASPEN NEUROSCIENCE
Howard Federoff, M.D., Ph.D., CEO
San Diego, CA
(Private)
Aspen Neuroscience is a development-stage, private biotechnology company that uses innovative genomic approaches combined with stem cell biology to deliver patient-specific, restorative cell therapies that modify the course of Parkinson disease. Aspen’s therapies are based upon the scientific work of world-renowned stem cell scientist, Dr. Jeanne Loring, who has developed a novel method for autologous neuron replacement.
www.aspenneuroscience.com

ATARA BIOTHERAPEUTICS
Pascal Touchon, President and CEO
San Francisco, CA
(NASDAQ: ATRA)
Atara is a pioneer in T-cell immunotherapy leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with severe diseases including solid tumors, hematologic cancers, and autoimmune disease. With our lead program in Phase III, Atara is the most advanced allogeneic T-cell immunotherapy company, intending to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. Our platform leverages the unique biology of EBV T-cells and has the capability to treat a wide range of EBV-associated diseases, or other severe diseases through incorporation of engineered CARs (chimeric antigen receptors) or TCRs (T-cell receptors). Atara is applying this one platform to create a robust pipeline including: tab-cel® (tabelecleucel) in Phase III development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease (EBV+ PTLD); ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation CAR-T immunotherapies for both solid tumors and hematologic malignancies.
www.atarabio.com

AUTOLUS
Christian Itin, Ph.D., Chairman and CEO
London, UK
(NASDAQ: AUTL)
Autolus is a biopharmaceutical company founded on advanced cell programming technology pioneered by Dr. Martin Pule and was spun-out from University College London in 2014. Since its inception, the company has undergone rapid growth, systematically adding the capabilities needed to manufacture and develop its programmed T-cell product candidates. Autolus is committed to bringing life-changing treatments to cancer patients by reprogramming their own T-cells to combat the treatment of haematological malignancies and solid tumours. Autolus believe its programmed T-cell therapies have the potential to offer cancer patients substantial benefits over the existing standard of care, including the potential for cure in some patients.
www.autolus.com

AVECTAS
Michael Maguire, Ph.D., CEO
Kildare, Ireland
(Private)
Avectas is a cell engineering technology business focused on improving the cost, manufacturing, and patient outcomes for the next generation of cellular therapies. Avectas is developing a unique cell engineering platform, SOLUPORE™, to enable the ex vivo manufacture of gene modified cell therapy products. SOLUPORE™ is a patented, non-viral, cell engineering technology that permeabilizes the target cell membrane and allows efficient transfer of cargo into cells whilst retaining very high levels of cell viability and functionality.
www.avectas.com

AVERY THERAPEUTICS
Jordan Lancaster, Ph.D., CEO
Tucson, AZ
(Private)
Avery Therapeutics is a preclinical-stage company developing advanced therapies for patients suffering from cardiovascular diseases. Avery’s lead candidate is an allogeneic tissue engineered cardiac graft, MyCardia™ in development for treatment of chronic heart failure. Using Avery’s proprietary manufacturing process, MyCardia can be manufactured at scale, cryopreserved, and shipped ready to use. Avery is leveraging its proprietary tissue platform to pursue other cardiovascular indications.
www.averythera.com

AVEXIS, A NOVARTIS COMPANY
Leah Bloom, SVP, External Innovation and Strategic Alliances
Bannockburn, IL
(NASDAQ: NVS)
AveXis, a Novartis company, is the world’s leading gene therapy company, redefining the possibilities for patients and families affected by life-threatening genetic diseases through our innovative gene therapy platform. Founded in 2013, the goal of AveXis’ cutting-edge science is to address the underlying, genetic root cause of diseases. AveXis pioneered foundational research, establishing AAV9 as an ideal vector for gene transfer in diseases affecting the central nervous system, laying the groundwork to build a best-in-class, transformational gene therapy pipeline. AveXis received its first U.S. Food and Drug Administration approval in May 2019 for the treatment of spinal muscular atrophy (SMA). AveXis is also pursuing gene therapies for Rett syndrome (AVXS-201), a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene (AVXS-301) and Friedreich’s ataxia (AVXS-401).
www.avexis.com

AVROBIO
Geoff MacKay, President and CEO
Cambridge, MA
(NASDAQ: AVRO)
Avrobio’s mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle, and bone. Our clinical-stage programs include Fabry disease, Gaucher disease, and cystinosis, and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato™ gene therapy platform, our foundation designed to scale gene therapy worldwide.
www.avrobio.com

AXOVANT GENE THERAPIES
Pavan Cheruvu, M.D., CEO
New York, NY
(NASDAQ: AXGT)
Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinson’s disease. Axovant is focused on accelerating product candidates into and through clinical trials with a team of experts in gene therapy development and through external partnerships with leading gene therapy organizations.
www.axovant.com

BELLICUM
Rick Fair, CEO
Houston, TX
(NASDAQ: BLCM)
Bellicum is a clinical-stage biopharmaceutical company striving to deliver cures through controllable cell therapies. The company’s next-generation product candidates are differentiated by powerful cell signaling technologies designed to produce more effective CAR-T and CAR-NK cell therapies. Bellicum currently has three product candidates: BPX-601, a GoCAR-T® targeting PSCA for the treatment of pancreatic cancer; BPX-603, a GoCAR-T targeting tumors expressing HER2; and a GoCAR-NK program targeting BCMA for the treatment of multiple myeloma.
www.bellicum.com

BIOCENTRIQ
Haro Hartounian, Ph.D., SVP and General Manager
Newark, NJ
(Private)
BioCentriq is the Cell and Gene Therapy Development and Manufacturing Center at the New Jersey Innovation Institute in Newark, NJ. It is a state-of-the-art process development and cGMP manufacturing center equipped with two GMP suites for cell and gene therapy clinical production and a cell and gene therapy process development pilot plant. BioCentriq is available for pre-competitive group sponsored projects and fee-for-service proprietary projects. We enable rapid growth of new scale-up and manufacturing technologies, customized as needed for each client. We also partner with those who want to develop and test new manufacturing technologies. Our clients come to us for fast and cost-effective development services that are secure, confidential, and flexible. We do not retain any intellectual property; all IP belongs solely to the client.
www.biocentriq.com

BIOGEN
Al Sandrock, M.D., Ph.D., EVP, Research & Development
Junghae Suh, Ph.D., VP, Gene Therapy Accelerator Unit
Cambridge, MA
(NASDAQ: BIIB)
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuro-immunology, Alzheimer’s disease and dementia, neuromuscular disorders, movement disorders, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.
www.biogen.com

BONE THERAPEUTICS
Miguel Forte, M.D., Ph.D., CEO
Gosselies, Belgium
(NASDAQ: BOTHE)
Bone Therapeutics is a listed, clinical-stage, cell therapy company committed to rebuilding lives of patients suffering from debilitating bone and joint conditions. Leveraging its extensive expertise in mesenchymal stromal cells, bone physiology, and patented manufacturing technology, Bone Therapeutics is developing a commercially ready, allogeneic and off-the-shelf bone cell therapy platform, ALLOB, and a protein solution for osteoarthritis pain, JTA-004, which are designed to offer patients and physicians best-in-class treatment options. Currently in Phase II/III clinical development, Bone Therapeutics’ advanced product portfolio targets large orthopaedic conditions with high unmet needs, such as knee osteoarthritis, unhealed fractures, and spinal fusion.
www.bonetherapeutics.com

BRAINSTORM CELL THERAPEUTICS
Stacy Lindborg, Ph.D., EVP and Head of Global Clinical Research
New York, NY
(NASDAQ: BCLI)
Brainstorm Cell Therapeutics is developing novel adult stem cell therapies for debilitating neurodegenerative disorders such as Amytrophic Lateral Scelorosis (ALS, also known as Lou Gherig Disease) Progressive MS, and other neurodegenerative diseases.
www.brainstorm-cell.com

 

CABALETTA BIO
Steven Nichtberger, M.D., President, CEO and Co-Founder
Philadelphia, PA
(NASDAQ: CABA)
Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T-cell therapies for patients with B-cell-mediated autoimmune diseases. The CABA (Cabaletta Approach to selective B-cell Ablation) platform, in combination with Cabaletta’s proprietary technology, utilizes CAAR (Chimeric AutoAntibody Receptor) T-cells that are designed to selectively bind and eliminate B-cells that express disease-causing autoantibodies while sparing normal B-cells. CAAR T-cells were designed based on chimeric antigen receptor, or CAR, T-cell technology that is clinically validated and commercially approved to treat B-cell cancers. Our lead product candidate, DSG3-CAART, is entering clinical development as a potential treatment for patients with mucosal pemphigus vulgaris, a prototypical B-cell-mediated autoimmune disease. Our lead preclinical product candidate, MuSK-CAART, has entered IND-enabling studies, and is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. Several undisclosed programs are also in development.
www.cabalettabio.com

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CALADRIUS BIOSCIENCES
Douglas Losordo, M.D., Chief Medical Officer, EVP, Global Head of Research and Development
Basking Ridge, NJ
(NASDAQ: CLBS)
Caladrius Biosciences is a clinical-stage biopharmaceutical company dedicated to the development of cellular therapies designed to reverse, not manage, disease. We are developing a first-in-class cell therapy product that is based on the notion that our body contains finely tuned mechanisms for self-repair. Our technology leverages and enables these mechanisms in the form of specific cells, using formulations and modes of delivery unique to each medical indication.
www.caladrius.com

CAPRICOR THERAPEUTICS
Linda Marban, Ph.D., CEO
Los Angeles, CA
(NASDAQ: CAPR)
Capricor Therapeutics is a cutting-edge biotech company focused on the discovery and development of novel therapies for the treatment and prevention of diseases. Capricor’s lead product candidate, allogeneic Cardiosphere-Derived Cells (CDCs), known as CAP-1002, is in late-stage clinical development as a treatment for Duchenne muscular dystrophy. In addition, the Company is conducting research and development on its exosomes platform technology for a variety of inflammatory and cardiac indications. In response to the fight against a global pandemic, our team is applying its cell and exosome technologies against COVID-19, including the development of a potential vaccine.
www.capricor.com

CARIBOU BIOSCIENCES
Rachel Haurwitz, Ph.D., President & CEO
Berkeley, CA
(Private)
Caribou is a pioneer in the revolutionary field of CRISPR genome editing. Caribou’s proprietary technology puts it at the forefront of the development of allogeneic cellular therapies. Caribou is developing an internal, wholly-owned pipeline of off-the-shelf CAR-T cell therapies and gene-edited natural killer (NK) cell therapies. The company’s focus is centered on oncology and the pipeline includes programs for both liquid and solid tumor targets. Caribou’s next-gen CRISPR gene editing technology drives higher specificity editing than first-generation CRISPR-Cas9. The company deploys gene editing to carry out multiple edits to boost T-cell persistence and to prevent GvHD. CB-010, the company’s lead program, is an allogeneic anti-CD19 CAR-T therapy where PD-1 has been removed from the CAR-T genome. The durability of anti-tumor activity achieved with CB-010 in vivo significantly surpasses the durability of CAR-Ts where PD-1 remains on.
www.cariboubio.com

CARISMA THERAPEUTICS
Steven Kelly, President and CEO
Philadelphia, PA
(Private)
Carisma Therapeutics is a biopharmaceutical company developing a differentiated and proprietary cell therapy platform focused on engineered macrophages, cells that play a crucial role in both the innate and adaptive immune response. The first applications of the platform, developed in collaboration with the University of Pennsylvania, are autologous chimeric antigen receptor (CAR)-macrophages for the treatment of solid tumors.
www.carismatx.com


CARMA CELL THERAPIES (MAXCYTE)
Shruti Abbato, EVP, Business Development
Gaithersburg, MD
(LON: MXCT)
CARMA Cell Therapies is a wholly-owned subsidiary of MaxCyte and is focused on the advancement of novel mRNA-based cell therapies for cancer and other diseases with serious unmet needs. MaxCyte developed CARMA, a novel and proprietary platform for the development of non-viral, human messenger RNA (mRNA)-based, chimeric antigen receptor (CAR) or T-cell receptor (TCR) redirected immune cell therapies. CARMA utilizes MaxCyte’s Flow Electroporation® technology for highly efficient, non-viral, delivery of one or more mRNA into unmanipulated peripheral blood mononuclear cells (PBMCs) or other immune cells, resulting in a unique class of cell therapies with the potential for improved safety and efficacy over conventional cell therapy approaches. CARMA also offers the potential for a significantly streamlined, scalable, and cost-effective GMP manufacturing process without the complexity of virus-based products. MaxCyte expects CARMA Cell Therapies to be self-funded by end of 2020.
www.maxcyte.com/carma-cell-therapies

CASTLE CREEK BIOSCIENCES
John Maslowski, CEO
Exton, PA
(Private)
Castle Creek Biosciences is a privately-held company that develops and commercializes gene therapies for patients with rare and serious genetic diseases. The company’s lead gene therapy candidate, FCX-007, is being evaluated for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe and debilitating form of epidermolysis bullosa (EB). The company is also advancing clinical research evaluating a diacerein topical ointment, CCP-020, for the treatment of epidermolysis bullosa simplex (EBS) and other forms of EB. In addition, Castle Creek Biosciences is developing FCX-013, a gene therapy for the treatment of moderate to severe localized scleroderma. Castle Creek Biosciences is a portfolio company of Paragon Biosciences.
www.castlecreekbio.com

CELLECT BIOTECHNOLOGY
Amos Ofer, Ph.D., Chief Operating Officer
Saba, Israel
(NASDAQ:APOP)
Cellect Biotechnology was founded in 2011 after 10 years of academic research and is developing a first-in-class and potentially transformative cell selection technology platform, the ApoGraft. The technology is based on a revolutionary approach of exposure of stem cell-containing cell populations to an apoptosis-inducing environment, causing selective cell death in specific populations of differentiated cells, while not harming and even expanding stem cells. The technology, protected by nine patent families, has broad potential application, enabling the stem cell based industry to produce safe, robust, and affordable stem cells for any usage and indication. ApoGraft’s first application in clinical development is the prevention of GvHD in bone marrow transplantation recipients. Early Phase I/II study results show good engraftment, perfect safety score, and no ApoGraft-related toxicity. Future potential applications include curing of autoimmune diseases and inducing immune tolerance in solid organ transplantation. Cellect’s pipeline also includes purification and expansion of mesenchymal stem cells and CAR-T production.
www.cellect.co

CELLINO
Nabiha Saklayen, Ph.D., CEO and Co-Founder
Cambridge, MA
(Private)
Cellino is the first cell and tissue foundry to offer single cell precision for regenerative medicine. Cellino’s platform technology combines laser optics, machine learning, and stem cell biology to automate the iPSC reprogramming and differentiation processes, resulting in highly-functional iPSC-derived cells and tissues.
www.cellinobio.com

CELLS FOR CELLS
Roberto Bobadilla Ph.D., CEO
Las Condes, Chile
(Private)
Cells for Cells is a Chilean biotechnological company dedicated to the research, development and commercialization of innovative cellular therapies, complying with high standards of scientific, technological and international quality, through manufacturing processes certified under ISO 9001: 2015. Each therapy is produced in our labs with GMP standards, being the first biotech company, with such high-quality standards at Latin American level. Our therapies are applied by duly certified specialists.
www.c4c.cl

CENTURY THERAPEUTICS
Gregory Russotti, Ph.D., Chief Technology Officer
Philadelphia, PA
(Private)
Century Therapeutics is harnessing the power of stem cells to develop curative cell therapy products for cancer that overcome the limitations of first generation cell therapies. Our genetically engineered, universal iPSC-derived immune effector cell products (NK and T-cells) are designed to specifically target hematologic and solid tumor cancers. Our commitment to developing off-the-shelf cell therapies will expand patient access and provide an unparalleled opportunity to advance the course of cancer care. Century was launched in 2019 by founding investor Versant Ventures in partnership with Fujifilm and Leaps by Bayer.
www.centurytx.com

CELYAD
Filippo Petti, CEO
Mont-Saint-Guibert, Belgium
(NASDAQ: CYAD)
Celyad is a clinical-stage biopharmaceutical company focused on the development of CAR-T cell based therapies. The company’s lead clinical program is focused on the treatment of relapsed/refractory acute myeloid leukemia (r/r AML) and is currently evaluating the first-in-class autologous (personalized) NKG2D-based CAR-T therapy, CYAD-01, and next-generation candidate, CYAD-02, in multiple Phase I clinical trials. Celyad is also developing a non-gene edited, allogeneic (donor derived) NKG2D-based CAR-T therapy referred to as CYAD-101, which is currently in Phase I development for the treatment of patients with metastatic colorectal cancer (mCRC). The company is also advancing next generation, allogeneic CAR-T candidates using its short hairpin RNA (shRNA) technology platform, led by CYAD-211, a BCMA targeting CAR-T therapy for the treatment of multiple myeloma (MM).
www.celyad.com

CEVEC
Nicole Faust, Ph.D., CEO
Cologne, Germany
(Private)
CEVEC Pharmaceuticals is a leading provider of high performance cell technology for the manufacturing of advanced biotherapeutics from R&D to manufacturing scale. The company’s portfolio comprises platform technologies for gene therapy viral vectors, viral vaccines, Exosomes and complex recombinant proteins. ELEVECTA® is specifically designed for AAV manufacturing, while CAP® addresses Adenoviral vectors, Lentiviral vectors, Oncolytic viruses, and Exosomes.
www.cevec.com

 

CORNING
Keith Olson, Ph.D, VP, CLS Strategy and Technology
New York, NY
(NASDAQ: GLW)
Corning is one of the world’s leading innovators in materials science, with a 169 year track record of life-changing inventions. Corning applies its unparalleled expertise in glass science, ceramic science, and optical physics along with its deep manufacturing and engineering capabilities to develop category-defining products that transform industries and enhance people’s lives. Today, we are a leader in laboratory tools and products, cell culture solutions, bioprocess vessels, and specialty surfaces. We are also helping to provide more reliable access to medicine with our revolutionary glass packaging for drug storage and delivery.
www.corning.com

 

COVANCE
Steve Anderson, Ph.D., Chief Scientific Officer
Burlington, NC
(NASDAQ: CVD)
Covance is a business segment of LabCorp, a leading global life sciences company, which provides contract research services to the drug, medical device and diagnostics, crop protection, and chemical industries. Covance and LabCorp are at the forefront of the development cell and gene therapies based on its well-established, unique expertise and coordinated capabilities in scientific, regulatory and commercial areas across preclinical, clinical and post-approval phases. COVANCE is a registered trademark and the marketing name for Covance Inc. and its subsidiaries around the world.
www.covance.com

 

CRYOPORT
Mark Sawicki, Ph.D., Chief Commercial Officer
Brentwood, TN
(NASDAQ: CYRX)
Cryoport is the life sciences industry’s most trusted global provider of temperature-controlled logistics solutions for temperature-sensitive life sciences commodities, serving the biopharmaceutical market with leading edge logistics solutions for biologic materials, such as regenerative medicine, including immunotherapies, stem cells, and CAR-T cells. Cryoport also provides a range of bioservices to the life sciences industry, including biostorage for biological specimens, materials and samples. Cryoport’s solutions are used by points-of-care, CRO’s, central laboratories, pharmaceutical companies, manufacturers, university researchers et al; as well as the reproductive medicine market, primarily in IVF and surrogacy; and the animal health market, primarily in the areas of vaccines and reproduction. Cryoport’s proprietary Cryoport Express® Shippers, Cryoportal® Logistics Management Platform, leading-edge Smartpak II® Condition Monitoring System and geo-sensing technology, paired with unparalleled cold chain logistics expertise and 24/7 client support, make Cryoport the end-to-end cold chain logistics partner that the industry trusts.
www.cryoport.com

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CYNATA THERAPEUTICS
Ross Macdonald, Ph.D., CEO
Melbourne, Australia
(ASX: CYP)
Cynata Therapeutics Limited is an Australian clinical-stage stem cell and regenerative medicine company developing a therapeutic mesenchymal stem cell (MSC) platform technology, Cymerus™, originating from the University of Wisconsin-Madison, a world leader in stem cell research. The proprietary Cymerus technology addresses a critical shortcoming in existing methods of production of multi-donor derived MSCs for therapeutic use, which is the ability to achieve consistent, economic manufacture at commercial scale. Cymerus utilises induced pluripotent stem cells (iPSCs) to produce MSCs through a proprietary process that is independent of donor limitations providing an “off-the-shelf” stem cell platform for therapeutic product manufacture. A successful world-first clinical trial in GvHD has been completed, meeting all safety and efficacy endpoints. The Company expects to commence three Phase II trials during 2020, in GvHD, osteoarthritis, and COVID-19. Cynata has a strategic partnership with Fujifilm, which is one of the Company’s larger shareholders.
www.cynata.com

CYTIVA
Catarina Flyborg, General Manager, Cell Therapy
Marlborough, MA
(NYSE: DHR)
Cytiva is a global provider of technologies and services that help advance and accelerate the development and manufacture of therapeutics. Previously GE Healthcare Life Sciences, Cytiva’s diverse portfolio includes well-recognized brands such as ÄKTA, Amersham, Biacore, FlexFactory, HyClone, MabSelect, Whatman and Xcellerex. Cytiva brings speed, efficiency and capacity to research and manufacturing workflows, enabling the delivery of transformative medicines to patients.
www.cytivalifesciences.com

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DISCGENICS
Flagg Flanagan, Chairman and CEO
Salt Lake City, UT
(Private)
DiscGenics is a privately-held, clinical-stage biopharmaceutical company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. As the only company in the world to develop an allogeneic cell therapy derived from intervertebral disc cells to treat diseases of the disc, DiscGenics believes it has a unique opportunity to harness the restorative potential of the human body to heal millions of patients suffering from the debilitating effects of back pain.
www.discgenics.com

EGENESIS
Paul Sekhri, President and CEO
Cambridge, MA
(Private)
eGenesis’ goal is to advance the field of transplantation and make available safe and reliable xeno organs, tissues, and cells to patients in need. eGenesis uses gene editing technology such as CRISPR to directly address the key virology and immunology hurdles that have impeded xenotransplantation to date. eGenesis is advancing an initial product toward the clinic for kidney transplant, with the longer-term potential of addressing a broader organ recipient population and expanding the applicability of xenotransplantation into other areas such as cell therapy.
www.egenesisbio.com

ELEVATEBIO
Mitch Finer, Ph.D., Chief Scientific Officer and President of ElevateBio BaseCamp
Cambridge, MA
(Private)
ElevateBio is a Cambridge-based creator and operator of a portfolio of innovative cell and gene therapy companies. It begins with an environment where scientific inventors can transform their visions for cell and gene therapies into reality for patients with devastating and life-threatening diseases. Working with leading academic researchers, medical centers, and corporate partners, ElevateBio’s team of scientists, drug developers, and company builders are creating a portfolio of therapeutics companies that are changing the face of cell and gene therapy and regenerative medicine. Core to ElevateBio’s vision is BaseCamp, a centralized state-of-the-art innovation and manufacturing center, providing fully integrated capabilities, including basic and transitional research, process development, clinical development, cGMP manufacturing, and regulatory affairs across multiple cell and gene therapy and regenerative medicine technology platforms. ElevateBio portfolio companies, as well as select strategic partners are supported by ElevateBio BaseCamp in the advancement of novel cell and gene therapies.
www.elevate.bio

FLEXION THERAPEUTICS
Adam Muzikant, Ph.D., SVP, Business Development
Burlington, MA
(NASDAQ: FLXN)
Flexion Therapeutics is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis (OA). Our first product, ZILRETTA® (triamcinolone acetonide extended-release injectable suspension), is the first and only FDA-approved extended-release, intra-articular (in the joint) injection for OA knee pain. Our pipeline includes FX201, an intra-articular gene therapy candidate being developed for symptomatic relief and disease modification in patients with knee OA. FX201 is designed to durably stimulate the production of an anti-inflammatory protein, IL-1Ra, whenever inflammation is present in the joint. Enrollment in a Phase I clinical trial with FX201 in patients with knee OA began in Q1 2020. We are also developing FX301, a locally delivered NaV1.7 inhibitor formulated for extended release from a thermosensitive hydrogel, for control of post-surgical pain. FX301 is expected to enter clinical trials in 2021.
www.flexiontherapeutics.com

FREQUENCY THERAPEUTICS
Chris Loose, Ph.D., Co-Founder and Chief Scientific Officer
Woburn, MA
(NASDAQ: FREQ)
Frequency Therapeutics is a leader in the development of medicines designed to activate progenitor cells within the body to treat degenerative diseases. The Company’s progenitor cell activation (PCA) approach stimulates progenitor cells to create functional tissue with the aim of developing disease modifying therapies. Frequency’s lead product candidate, FX-322, is designed to regenerate auditory hair cells to restore hearing function. In a FX-322 Phase I/II study, statistically significant and clinically meaningful improvements in key measures of hearing function in patients with sensorineural hearing loss were observed. Early clinical data for FX-322 so far includes this first ever hearing “signal” from a regenerative therapeutic, a favorable safety profile, and an ability to deliver FX-322 to the target within the ear. FX-322 is being evaluated in an ongoing Phase IIa study. Frequency also is evaluating additional diseases where its PCA approach could create functional tissue, including a discovery program in multiple sclerosis.
www.frequencytx.com

FUJIFILM CELLULAR DYNAMICS
Takeshi Yamamoto, President and CEO
Madison, WI
(Private)
Fujifilm Celluar Dynamics is an iPSC company providing multiple cell types known as iCell products for the drug discovery research market and also cell therapy services. For use in therapeutics we offer a reprogramming technology license and/or iPSC lines manufactured under cGMP grade. The company has several of its own therapeutic pipelines and is able to bring them into the clinical through its state-of-the-art cGMP facility. The company also offers CDMO services for customers.
www.fujifilmcdi.com

GAMIDA CELL
Michele Korfin, Chief Operating and Chief Commercial Officer
Boston, MA and Jerusalem, Israel
(NASDAQ: GMDA)
Gamida Cell is an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases. We harness our cell expansion platform to create therapies with the potential to redefine standards of care in areas of serious medical need.
www.gamida-cell.com

GENERATION BIO
Phillip Samayoa, Ph.D., Strategy and Portfolio Development
Cambridge, MA
(NASDAQ: GBIO)
Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The company’s non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The company’s efficient, scalable manufacturing process supports Generation Bio’s mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.
www.generationbio.com

GENPREX
Michael Redman, Chief Operating Officer and EVP
Austin, TX
(NASDAQ: GNPX)
Genprex is a clinical-stage gene therapy company developing potentially life-changing technologies for patients with cancer and diabetes. The Company’s lead product candidate, OncoprexTM, is being evaluated as a treatment for non-small cell lung cancer (NSCLC). Oncoprex has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells using a novel formulation containing the TUSC2 suppressor gene. This suppressor gene is reduced or absent in over 80% of lung cancers of all histologies. In particular, Oncoprex modulates the immune response against cancer cells and has shown clinical evidence of converting “cold” tumors (resistant to treatment) to “hot” tumors. Cold tumors are responsible for tumor progression in several cancers, especially NSCLC. OncoprexTM received Fast Track designation in combination with osimertinib (Tagrisso®) in 2020. Genprex will also be conducting a Phase I/II clinical trial in combination with Pembroluzimab (Keytruda®).
www.genprex.com

 

GENSIGHT BIOLOGICS
Magali Taiel, M.D., Chief Medical Officer
Paris, France
(NASDAQ: SIGHT)
GenSight Biologics is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, LUMEVOQ® (GS010; lenadogene nolparvovec), is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to the eye by intravitreal injection to offer patients a sustainable functional visual recovery.
www.gensight-biologics.com

GINER LIFE SCIENCES
Linda Tempelman, Ph.D., Chief Scientific Officer
Newton, MA
(Private)
Giner Life Sciences (GLS) is a privately-held company founded to focus on the development and commercialization of oxygenated cell therapies. GLS utilizes electrochemical technology to produce oxygen from body water as a fully implantable engineering solution to alleviate the low oxygen environment of membrane capsules. Membrane (macro)capsules have been demonstrated to protect allogeneic cell therapies from the immune system of the patient. The GLS technology includes: the oxygenator, the oxygen-enabled cell capsule and the control, power and recharging system for a fully implantable system with U.S. and international patents. Oxygenation is essential for therapies where the therapeutic dose requires a high cell density system to minimize implant size. GLS is applying this platform technology to several cell therapies, including the treatment of diabetes. GLS is establishing partnerships with companies with unique cellular offerings (engineered cells and stem cell derived products) who are seeking cell therapy implant systems.
www.ginerlifesciences.com

GLYCOSTEM THERAPEUTICS
Troels Jordansen, CEO
Oss, The Netherlands
(Private)
Glycostem has developed a platform technology for the expansion and differentiation of allogeneic hematopoietic stem and progenitor cells derived from umbilical cord blood. This enables off-the-shelf, safe and low production cost products. Glycostem’s platform technology enables a closed system cell culture process for the expansion of CD34+ hematopoietic stem and progenitor cells, and further differentiation into fully functional immune cells, i.e. Natural Killer (NK) cells. Glycostem has treated 10 elderly and fragile AML patients in a Phase I setting with an excellent safety profile and strong indication of clinical efficacy. Year one survival data showed 80% compared to the general AML population data showing 35%. A GMP license was received for the company’s own manufacturing unit in 2019, and pivotal trials in acute myeloid leukemia (AML) and multiple myeloma (MM) will commence 2H 2020.
www.glycostem.com

GYROSCOPE THERAPEUTICS
Nadia Waheed, M.D., Chief Medical Officer
London, UK
(Private)
Our ultimate goal is to slow, or possibly stop, the progression of dry-AMD. Patients in our Phase I/II clinical trial, known as the FOCUS study, receive a single dose of GT005 through an injection below their retina. Syncona, our lead investor, helped us create the only retinal gene therapy company to combine discovery research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London, UK, with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness.
www.gyroscopetx.com

 

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HEALIOS K.K.
Richard Kincaid, Executive Officer and Chief Finance Officer
Tokyo, Japan
(TSE: 4593)
HEALIOS K.K. is a Japan-based, world leader in regenerative medicine. Healios researches and develops regenerative medicine products in both the somatic stem cell and induced pluripotent stem cell (iPSC) fields. Healios combines expertise and resources in iPS cells, gene-editing, and cell manufacturing technology to bring therapies and cures to patients with intractable diseases. Healios is currently conducting two pivotal clinical studies in Japan, to commercialize a somatic stem cell product to treat ischemic stroke and acute respiratory distress syndrome (ARDS). In addition, Healios has established a next generation immuno-privileged universal donor iPSC platform to advance therapies in several domains including immuno-oncology, ophthalmology, and organ buds.
www.healios.co.jp

HELIXMITH
Sunyoung Kim, CEO
Seoul, Korea and San Diego, CA
(KOSDAQ: 084990.KQ)
Helixmith is an R&D-focused biopharmaceutical company based in Seoul and San Diego, developing new and innovative biopharmaceuticals based upon its proprietary scientific platform technology to address unmet medical needs. Currently, the company is actively focusing on developing the proprietary plasmid DNA-based drug Engensis at various clinical stages in the U.S., Korea, and China for cardiovascular and neurological diseases including, but not limited to, painful DPN, diabetic foot ulcer (DFU), amyotrophic lateral sclerosis (ALS), and Charcot-Marie-Tooth disease (CMT). The company also has an early phase pipeline in AAV-based gene therapy and CAR-T cell therapy.
www.helixmith.com

HOMOLOGY MEDICINES
Arthur Tzianabos, Ph.D., President and CEO
Bedford, MA
(NASDAQ: FIXX)
Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases with significant unmet medical needs by curing the underlying cause of the disease. Homology’s proprietary platform is designed to utilize its human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to precisely and efficiently deliver genetic medicines in vivo either through a gene therapy or nuclease-free gene editing modality across a broad range of genetic disorders. Homology has a management team with a successful track record of discovering, developing, and commercializing therapeutics with a particular focus on rare diseases, and intellectual property covering its suite of 15 AAVHSCs. Homology believes that its compelling preclinical data, scientific expertise, product development strategy, manufacturing capabilities, and intellectual property position it as a leader in the development of genetic medicines.
www.homologymedicines.com

IOVANCE BIOTHERAPEUTICS
Maria Fardis, Ph.D., CEO
San Carlos, CA
(NASDAQ: IOVA)
IOVANCE Biotherapeutics is focused on the development and commercialization of autologous cellular immunotherapies optimizing personalized, tumor-directed Tumor Infiltrating Lymphocytes (TIL). IOVANCE is conducting four Phase II clinical trials to assess the efficacy and safety of autologous Tumor Infiltrating Lymphocytes for treatment of patients with Metastatic Melanoma, Squamous Cell Carcinoma of the Head and Neck, Non-Small Cell Lung Cancer (NSCLC), and Cervical Cancer.
www.iovance.com

I PEACE
Koji Tanabe, Ph.D., CEO
Palo Alto, CA
(Private)
I Peace’s proprietary manufacturing platform enables the fully-automated mass production of discrete iPSCs from multiple donors in a single room. Increasing the available number of clinical-grade iPSC lines allows our customers to take differentiation propensity into account to select the most appropriate iPSC line for their clinical research at significantly reduced cost. Our goal is to create iPSCs for every individual that become his/her stem cell for life. We have built a GMP facility authorized by the PMDA (Japanese FDA equivalent) that is capable of full automation to generate iPSCs on a mass scale to dramatically reduce cost.
www.ipeace.com

KRIYA THERAPEUTICS
Shankar Ramaswamy, M.D., Co-Founder and CEO
Palo Alto, CA
(Private)
Kriya Therapeutics is a next-generation gene therapy company focused on designing and developing transformative gene therapies for highly prevalent diseases. With core operations in the Bay Area, California and Research Triangle Park, North Carolina, Kriya is establishing a platform for the development of gene therapies targeting serious chronic diseases affecting millions of patients. The company’s initial pipeline includes multiple programs for the treatment of metabolic diseases including Type 1 Diabetes, Type 2 Diabetes, and severe obesity.
www.kriyatx.com

KUUR THERAPEUTICS
Kurt Gunter, M.D., Chief Medical Officer
Houston, TX
(Private)
Kuur Therapeutics is committed to transforming the lives of patients with cancer by developing off-the-shelf CAR-engineered NKT cell therapies. NKT cells demonstrate several important biological differences when compared to conventional T-cells, which we believe render them well suited for the development of off-the-shelf CAR products. In addition, we have engineered innovative CAR constructs to further optimize the function, potency, and therapeutic efficacy of CAR-NKT cells. The first CAR-NKT product (KUR-501) is in a Phase I clinical trial in pediatric patients with relapsed/refractory neuroblastoma. Our second CAR-NKT cell candidate (KUR-502) is an allogeneic product targeting CD19+ B-cell malignancies. The FDA has allowed the Phase I study to begin and we are anticipating first patient treatment in 1H 2020. Our third CAR-NKT cell candidate (KUR-503) is another allogeneic product targeting the GPC3 molecule, which is highly expressed in hepatocellular (liver) cancer; we anticipate beginning human studies KUR-503 in 2021.
www.kuurtx.com

LIFESPROUT
Sashank Reddy, M.D., Ph.D., Founder
Baltimore, MD
(Private)
LifeSprout is a privately-held company founded out of Johns Hopkins University that is developing revolutionary products for aesthetic and regenerative medicine. The company’s Regenerative Matrix platform leverages advances in materials science to create products that look and feel like the body’s own tissues. LifeSprout is turning this technology into a suite of products for the millions of patients with tissue losses from aging, cancer, metabolic disease, and orphan indications. The company’s lead product Lumina™ is a breakthrough family of aesthetic fillers enabling immediate, natural restoration with biostimulatory effects. LifeSprout’s next generation cell therapy products use the power of the platform to promote true tissue regeneration.
www.lifesproutbio.com

 

LOCANA
Jim Burns, Ph.D., CEO
San Diego, CA
(Private)
Locana is creating a new class of gene therapies by treating  disease causing mRNA involved in disease. The Locana platform enables a portfolio of gene therapy systems to modify RNA across multiple mechanisms of action with the goal of creating precise and durable genetic medicines for the benefit of patients without DNA modification.
www.locanabio.com

LOGICBIO THERAPEUTICS
Fred Chereau, CEO
Lexington, MA
(NASDAQ: LOGC)
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide™, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.
www.logicbio.com

LUMINARY THERAPEUTICS
Debra Bowes, Chief Business Officer
Minneapolis, MN
(Private)
Luminary Therapeutics is an early-stage cell therapy company focused on autologous non-viral next generation therapies for cancer and autoimmune disorders.
www.luminarytx.com

MAMMOTH BIOSCIENCES
Peter Nell, Ph.D., Chief Business Officer and Head of Therapeutics
San Francisco, CA
(Private)
Mammoth Biosciences is harnessing the diversity of nature to power the next generation of CRISPR products. Through its discovery of novel CRISPR systems, the company is enabling the full potential of its platform to read and write the code of life. With a distinguished team including co-founder and CRISPR-Cas genome editing co-inventor Jennifer Doudna, Mammoth is addressing challenges across healthcare, agriculture, environmental monitoring, biodefense, and more. Mammoth’s efforts are addressed towards CRISPR-based diagnostics applying our proprietary DETECTR(TM) platform, and gene editing approaches towards ex vivo and in vivo therapeutics benefiting from our novel CRISPR systems providing unique properties. We are currently actively exploring partnerships in both areas.
www.mammoth.bio.com

MEDIPOST AMERICA
Antonio Lee, Ph.D., CEO and Global Head, Business Development
Pangyo, South Korea
(KOSDAQ: 078160)
MEDIPOST operates the largest private cord blood bank CELLTREE® in South Korea with over 255,000 units of private cord blood bank under storage. Each year, over 20,000 private cord blood units are collected and stored at CELLTREE®. MEDIPOST’s research and development is focused on novel off-the-shelf, allogeneic cell therapeutics using human Umbilical Cord Blood-derived Mesenchymal Stromal Cells (hUCB-MSCs) with clinical-stage assets in the disease areas of osteoarthritis (OA), broncho-pulmonary dysplasia (BPD) and Alzheimer’s disease (AD). Preclinical-stage pipeline includes alopecia and diabetic nephropathy. MEDIPOST America Inc. is wholly-owned subsidiary of MEDIPOST with commercial and development rights for the Americas and Europe. www.medi-post.com

MINERVA BIOTECHNOLOGIES
Cynthia Bamdad, Ph.D., CEO
Waltham, MA
(Private)
Minerva Biotechnologies discovered that the growth and pluripotency of naïve stem cells is mediated by the same growth factor receptor, MUC1*, and growth factor, NME7AB, that mediate growth and metastatic potential of cancer cells. We elucidated the molecular mechanisms that limit stem cell self-replication but allow cancer cells to self-replicate indefinitely. We learned how to override the pluripotency/differentiation switch to make naïve stem cell proliferate, without spontaneous differentiation, until we trigger the switch. Conversely, we developed therapeutic agents that block MUC1* and others that block NME7AB, which block cancer growth. Minerva makes NME7AB-containing AlphaSTEM naïve stem cell media and a synthetic peptide that flips the switch to initiate differentiation. Minerva holds an FDA-approved IND for 1st-in-human trial for a CAR T that targets MUC1* for the treatment of metastatic breast cancers; trial is open at the Fred Hutchinson (NCT04020575). No therapeutic that targets MUC1* has ever been tested in humans.
www.minervabio.com

MOLMED, NOW PART OF AGC BIOLOGICS
Luca Alberici, Ph.D., Chief Business Officer
Milan, Italy
(MLM.MI)
MolMed is a biotechnology company focused on research, development, production, and clinical validation of gene and cell therapies for the treatment of cancer and rare diseases. Thanks to the experience and know-how acquired in over 20 years of activity, MolMed is a reference player in the cell and gene sector, able to manage all phases of research up to the commercialization of advanced gene and cell therapies. Thanks to its authorized facilities, MolMed offers GMP development and production services for gene and cell therapies, providing resources and experience in carrying out studies from the preclinical phase to marketing.
www.molmed.com

OPSIS THERAPEUTICS
Nick Manusos, President and CEO
Madison, WI
(Private)
Opsis Therapeutics was founded in 2016 as a joint venture with FUJIFILM. We are developing advanced medicines for patients suffering from inherited retinal disease (IRD), inherited macular degenerations (IMD), and age-related macular degeneration (AMD). Our team has developed methods to manufacture authentic, highly pure, highly scalable, fully cryopreservable, and rigorously tested human photoreceptors (and RPE) that offer the potential for dramatic improvement in the care for patients suffering from permanent vision loss.
www.opsistx.com

ORCHARD THERAPEUTICS
Bobby Gaspar, M.D., Ph.D., CEO
London, UK
(NASDAQ: ORTX)
Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSK’s rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families, and caregivers is immense and current treatment options are limited or do not exist.
www.orchard-tx.com

OVIZIO
Emilie Viey, Ph.D., CEO
Brussels, Belgium
(Private)
Ovizio Imaging Systems develops and commercializes a unique technology platform that unifies real-time quantitative imaging with an automation and dye-free approach. We help our customers to monitor their cell cultures in key applications in cell and gene therapies and vaccines. While we support global customers, we have a strong footprint in the U.S. We develop our own instruments and disposables that are manufactured by our European CMOs.
www.ovizio.com

OXFORD BIOMEDICA
Jason Slingsby, Ph.D., Chief Business Officer
Oxford, UK
(LON: OXB.L)
Oxford Biomedica (OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford Biomedica has built a sector leading lentiviral vector delivery platform (LentiVector®), which we leverage to develop in vivo and ex vivo products both in-house and with partners. OXB has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases, and respiratory disease. OXB has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Axovant Gene Therapies, Orchard Therapeutics, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, and Imperial Innovations, through which it has long-term economic interests in other potential gene and cell therapy products. Additionally, the group has signed a Clinical and Commercial Supply Agreement with AstraZeneca for the manufacture of the adenovirus based COVID-19 vaccine candidate, AZD1222.
www.oxb.com

PASSAGE BIO
Jill Quigley, Chief Operations Officer
Philadelphia, PA
(NASDAQ: PASG)
Passage Bio is a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders with limited or no approved treatment options. The company is based in Philadelphia, PA and has a research, collaboration and license agreement with the University of Pennsylvania and its Gene Therapy Program (GTP). The GTP conducts discovery and IND-enabling preclinical work and Passage Bio conducts all clinical development, regulatory strategy, and commercialization activities under the agreement. The company has a development portfolio of six product candidates, with the option to license eleven more, with lead programs in GM1 gangliosidosis, frontotemporal dementia, and Krabbe disease.
www.passagebio.com

PDC*LINE PHARMA
Eric Halioua, President and CEO
Liège, Belgium
(Private)
PDC*line Pharma is a clinical-stage cell therapy company that develops a new class of potent and off-the-shelf therapeutic cancer vaccines based on a proprietary cell line of Plasmacytoid Dendritic Cells (PDC*line). Based on a robust preclinical package and a first-in-human Phase Ib in melanoma, PDC*line Pharma is initiating a clinical development in lung cancer with a new candidate (PDC*lung) and neoantigens (PDC*Neo). PDC*line Pharma comprises a team of 23 persons based in Liège, Belgium and in Grenoble, France. The company has raised nearly €32M. In March 2019, PDC*line Pharma was granted an exclusive license in South Korea and an exclusive option in other Asian countries to LG Chem, for the development and commercialization of PDC*lung cancer vaccine for lung cancer. The total deal value is 108M€ plus significant tiered royalties on net sales in Asia.
www.pdc-line-pharma.com

PLURISTEM THERAPEUTICS
Yaky Yanay, President and CEO
Haifa, Israel
(NASDAQ: PSTI)
Pluristem is a clinical-stage regenerative medicine company developing a platform of novel biological products. The placenta-based cell therapy products are off-the-shelf, requiring no tissue matching prior to administration. Each product releases a distinct combination of therapeutic proteins in response to signals the patient’s body to stimulate the body’s own regenerative mechanisms, and is been evaluated to treat conditions such as inflammation, ischemia, muscle injuries, severe COVID-19 cases complicated by Acute Respiratory Distress Syndrome (ARDS), hematological disorders, or exposure to radiation. The Company’s proprietary three-dimensional expansion technology can be used to grow cells in mass quantities with batch-to-batch consistency at Pluristem’s state-of-the-art manufacturing facility. The facility enables Pluristem to control its supply chain, and the purity and potency of its cell products, all at a significantly lower cost of goods. Pluristem has a strong IP position, Company-owned and operated, GMP-certified manufacturing and research facilities, and strategic relationships with leading international collaborations.
www.pluristem.com

POLYPLUS-TRANSFECTION
Géraline Guerin-Peyrou, Chief Marketing Officer
Illkirch, France
(Private)
Polyplus-transfection applies its 18+ year expertise to the development of novel transfection solutions. Polyplus-transfection is the leading supplier of a key critical component for viral vectors manufacturing for gene and cell therapy. We are proud to provide qualified and GMP-grade transfection reagents suitable from R&D to clinical trial and commercial scale, accompanied by a strong scientific and regulatory support. In addition, we provide a range of effective transfection reagents to deliver most nucleic acids, including DNA and siRNA in vitro and in vivo.
www.polyplus-transfection.com

POSEIDA THERAPEUTICS
Matthew Spear, M.D., Chief Medical Officer
San Diego, CA
(Private)
Poseida Therapeutics is a clinical-stage biopharmaceutical company dedicated to utilizing our proprietary gene engineering platform technologies to create next generation cell and gene therapeutics with the capacity to cure. We have discovered and are developing a broad portfolio of product candidates in a variety of indications based on our core proprietary platforms, including our non-viral piggyBac DNA Modification System, Cas-CLOVER site-specific gene editing system and nanoparticle- and AAV-based gene delivery technologies. Our core platform technologies have utility, either alone or in combination, across many cell and gene therapeutic modalities and enable us to engineer our wholly-owned portfolio of product candidates that are designed to overcome the primary limitations of current generation cell and gene therapeutics.
www.poseida.com

 

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PRECISION BIOSCIENCES
Cindy Atwell, VP, Business Development and Alliance Management
Durham, NC
(NASDAQ: DTIL)
Precision BioSciences is dedicated to improving life through its proprietary genome editing platform, “ARCUS.” Precision leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and create healthy and sustainable food and agriculture solutions. Precision is actively developing product candidates in three innovative areas: allogeneic CAR-T immunotherapy, in vivo gene correction, and food.
www.precisionbiosciences.com

 

RECOMBINETICS
Daniel Carlson, Ph.D., Chief Scientific Officer
Eagan, MN
(Private)
Recombinetics (RCI) provides gene-editing solutions for some of the world’s most intractable challenges, from improving human health to sustainably feeding an ever-growing population. Founded in 2008, RCI is widely recognized as the global thought leader in livestock gene editing and applied technology. RCI has developed proprietary toolkits and know-how for deployment in agriculture, regenerative medicine, and therapeutic development, commercialized through its subsidiaries Acceligen, Regenevida, and Surrogen. Regenevida and Surrogen are wholly-owned subsidiaries of RCI and directly align with the mission of the Alliance for Regenerative Medicine. Regenevida is a therapeutic company that leverages RCI’s swine engineering systems for cultivating human cells, tissues, and organs within living pigs for human therapeutic applications. Surrogen develops custom-tailored pig models that either simulate human disease, or report treatment outcomes. These models enable more effective therapeutic development, including cell and gene therapies, which will accelerate the conversion of discoveries to medical solutions.
www.recombinetics.com

 

REGENERATIVE PATCH TECHNOLOGIES
Jane Lebkowski, Ph.D., President
Menlo Park, CA
(Private)
Regenerative Patch Technologies (RPT) was founded to advance the development of bio-engineered implants for the treatment of vision loss. RPT’s lead product is called CPCB-RPE1 and is a composite implant consisting of human retinal pigmented epithelial (RPE) cells polarized on an ultrathin, biocompatible, parylene membrane. The properties of the parylene membrane are designed to mimic those of the native Bruch’s membrane. This implant serves as a tissue replacement for the diseased RPE cell layer and Bruch’s membrane found in patients with the advanced form of dry age-related macular degeneration. The company has now completed enrollment and one year follow-up subjects in a Phase I/IIa clinical trial and is progressing a Phase IIb clinical trial in patients with geographic atrophy.
www.regenerativepatch.com

REGENXBIO
Curran Simpson, SVP, Product Development and Chief Technology Officer
Rockville, MD
(NASDAQ: RGNX)
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
www.regenxbio.com

ReNeuron_135x65

RENEURON GROUP
Olav Hellebo, CEO
Bridgend, UK
(LON: RENE)
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, ReNeuron has therapeutic candidates in clinical development for motor disability as a result of stroke, for critical limb ischaemia, and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential new nanomedicine targeting cancer and as a potential delivery system for gene therapy treatments.
www.reneuron.com

ROOTPATH
Xi Chen, Ph.D., CEO
Watertown, MA
(Private)
RootPath is a Massachusetts based preclinical-stage biotechnology company pioneering synthetic immunology and its therapeutic applications. With our Synthetic Immunology platform, we are the only group in the world that can ‘Read’, ‘Write’, and ‘Mine’ the immune system fast and inexpensively enough to enable personalized discovery of therapeutic TCRs. Our lead program is a personalized cell therapy for solid tumors. Future opportunities include infectious and autoimmune diseases. RootPath was launched in 2017 by Nest.bio Ventures and has raised $18M in seed and Series A financing.
www.rootpath.com

SANGAMO THERAPEUTICS
Mark McClung, Chief Business Officer and EVP
Brisbane, CA
(NASDAQ: SGMO)
Sangamo Therapeutics is focused on translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing, and gene regulation. www.sangamo.com

SCM LIFESCIENCE
BG Rhee, Ph.D., CEO
Incheon, Korea
(KOSDAQ: 298060)
SCM Lifescience is a clinical-stage biopharmaceutical company specializing in the development of innovative stem cell and immune cell therapeutics. The company is using its proprietary subfractionation culturing method to isolate homogeneous population of clonal mesenchymal stem cells, serving as basis to its stem cell pipelines: SCM-CGH for treatment of chronic graft-versus-host disease, and SCM-AGH for treatment of atopic dermatitis and acute pancreatitis. It has recently expanded into the immuno-oncology sector through its U.S.-based subsidiary, CoImmune, incorporating allogeneic, non-viral use CAR-CIK, and dendritic cell cancer vaccine into its development. With strong capabilities around cell therapy research and development and the US-based cGMP manufacturing facility, SCM Lifescience is focused on accelerating product candidates through clinical trials to provide benefits to patients in high unmet medical need.
www.scmlifescience.com

SENSORION
Nawal Ouzren, CEO
Montpellier, France
(EPA: ALSEN)
Sensorion is a biotech company, founded in 2009 as a spin-out from INSERM, focused as a “pure player” on developing therapies to treat, prevent, and restore in the field of hearing loss. With our primary strength in the inner ear and neurosciences, we combine world-class scientific excellence and top-tier execution capabilities to deliver first-in-class therapeutics. Sensorion has built an international network of in-house experts, partners, and key opinion leaders. Our leadership has exceptional, solid experience in research, development, management, and business development within the biopharmaceutical industry. Together with our partners, we are committed to build and develop an industry leading pipeline of innovative treatments for hearing loss.
www.sensorion-pharma.com

SENTI BIOSCIENCES
Tim Lu, M.D., Ph.D, Co-Founder and CEO
San Francisco, CA
(Private)
Senti Biosciences is a next-generation therapeutics company that is developing gene circuits and programming cells for tremendous therapeutic value. Senti’s mission is to outsmart complex diseases with more intelligent medicines that will transform people’s lives. By programming cells to respond, adapt, and make decisions, Senti is creating smarter therapies with computer-like logic, enhanced functionality, and greater therapeutic control. Senti’s product candidates are designed to address major challenges in cancer treatment by equipping them with gene circuits that allow them to overcome cancer immune evasion, and more precisely target and eliminate cancer cells while sparing healthy tissue. Senti Biosciences is based in South San Francisco and was founded in 2016 by Drs. Tim Lu, Philip Lee, Jim Collins, and Wilson Wong. Senti is proud to count NEA, 8VC, Amgen Ventures, Lux Capital, Menlo Ventures, Pear Ventures, Allen & Company, Nest.Bio, Omega Funds, and LifeForce Capital among its investors.
www.sentibio.com

SENTIEN BIOTECHNOLOGIES
Brian Miller, Co-Founder and CEO
Lexington, MA
(Private)
Sentien Biotechnologies is a privately-held, clinical-stage company developing novel ex-vivo cell therapy applications to treat conditions caused by systemic, immune-mediated inflammation. Sentien’s lead product, SBI-101, integrates allogeneic mesenchymal stromal cells (MSCs) within an extracorporeal, hollow-fiber device. By immobilizing MSCs within a blood-filtration device, SBI-101 allows prolonged exposure of patient blood to the MSCs, resulting in the dynamic delivery of MSC-secreted factors at doses that are unattainable by direct injection. SBI-101 is being evaluated in a Phase Ib/IIa study in subjects with dialysis-requiring acute kidney injury. An interim readout from the study provides evidence of anti-inflammatory and wound healing effects consistent with the SBI-101 therapeutic hypothesis. Building on this therapeutic hypothesis, SBI-101 is being pursued in COVID-19 patients suffering from severe systemic inflammation. Sentien’s technology can be applied to additional systemic inflammatory indications in both acute and chronic diseases, focusing on complex conditions where an advanced therapeutic may offer compelling advantages.
www.sentienbiotech.com

SERNOVA
Philip Toleikis, Ph.D., President and CEO
London, Ontario, Canada
(TSX-V: SVA)
Sernova is developing regenerative medicine therapeutic technologies using the Cell Pouch System, a medical device and immune protected therapeutic cells to improve the treatment and quality of life of people with chronic metabolic diseases such as insulin-dependent diabetes, blood disorders including hemophilia and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. The Cell Pouch along with therapeutic cells has been shown to provide long-term safety and efficacy and has been proven to provide a biologically compatible environment for insulin-producing cells in humans. In early assessments of its ongoing Phase I/II clinical trial, Sernova has shown the presence of blood levels of c-peptide both during glucose tolerance tests as well as under fasting conditions.
www.sernova.com

SKYLAND ANALYTICS
Bob Di Scipio, CEO
Niwot, CO
(Private)
Skyland Analytics streamlines drug product, process, and patient data management by offering cloud-based software solutions that ensure Part 11-compliance, data transparency, and data integrity throughout the product lifecycle and across internal teams and external partners. With deep expertise in manufacturing, IT systems, data analytics, and product quality, we are uniquely positioned to provide innovative technology that accelerates commercialization and meets evolving business and regulatory demands.
www.skylandanalytics.net

STANDARDS COORDINATING BODY FOR REGENERATIVE MEDICINE
Richard McFarland, M.D., Ph.D., President
Gaithersburg, MD
(Private)
The Standards Coordinating Body (SCB) is a non-profit organization with a mission to “coordinate the accelerated advancement and improved awareness of the standards and best practices that address the rapidly evolving needs of the global regenerative medicine advanced therapy community.” SCB, through its FDA-funded work, looks to complement the current processes of standards organizations to ensure that new or revised standards provide the greatest benefits to the regenerative medicine community. SCB engages the broader community in the identification, prioritization, and advancement of potential standards; coordinates and communicates about standards activities across the regenerative medicine community; and educates the community about available standards, the standards development processes, and standards implementation. SCB was co-founded by the Alliance for Regenerative Medicine, and builds upon Section 3036 of the 21st Century Cures Act, which calls for the coordination and prioritization of standards creation to support the development and review of regenerative advanced therapies.
www.standardscoordinatingbody.org

StemBioSys_170x65

STEMBIOSYS
Bob Hutchens, President and CEO
San Antonio, TX
(Private)
StemBioSys is a privately-held biomedical company. Our technologies represent the next evolution in cell research and are branded to the research market under the CELLvo™ label. The centerpiece of these technologies is our broad range of CELLvo™ Matrices. These cell derived microenvironment allow a variety of cells to replicate more rapidly and express markers indicative of potency beyond that seen with traditional tissue culture substrates. The company also has several cell products isolated and expanded on our matrixies. Additionally, earlier this year, StemBioSys acquired CarTox. Together with CarTox, we are developing a novel approach for preclinical cardiac toxicity safety testing. Finally, recently the company and its scientific founder began development of a cell product that uses our proprietary matrix technology to treat Type 1 Diabetes.
www.stembiosys.com

STEMPEUTICS
Pawan Gupta, M.D., Ph.D., SVP, Medical and Regulatory Affairs
Bangalore, India
(Private)
Stempeutics Research Bangalore is a late-stage life science company focused on developing and commercializing novel therapeutics based on adult stem cells. The company’s flagship product is Stempeucel® drug, an on-demand, off-the-shelf product based on pooled, allogeneic Mesenchymal Stromal Cells derived from bone marrow of healthy, adult volunteers. Stempeucel® is being developed for the treatment of Critical Limb Ischemia (CLI), Osteoarthritis (OA), Diabetic Foot Ulcer (DFU) Perianal fistuala due to Crohn’s disease and ARDS due to COVID 19. In 2017, DCGI granted manufacturing and marketing approval for Stempeucel® product for the treatment of CLI due to Buerger’s disease patients.
www.stempeutics.com

SYNTHEGO
Robert Deans, Ph.D., Chief Scientific Officer
Redwood City, CA
(Private)
Synthego is a genome engineering company that enables the acceleration of life science research and development in cell and gene therapy in the ultimate pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s full-stack platform vertically integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance both basic research and therapeutic development programs. The company’s new approach to tackling the complexity of biology introduces engineering rigor to biological research with transformative effectiveness and agility to accommodate the growing technology space. By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge products and services that leverage the power of genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology.
www.synthego.com

TALARIS THERAPEUTICS
Scott Requadt, CEO
Louisville, KY
(Private)
Talaris Therapeutics is a late clinical-stage biotechnology company that is developing transformative cell therapies with the potential to eliminate the burden of chronic immunosuppression for organ transplant recipients and to induce durable remissions in patients with severe auto-immune and immune-mediated disorders. FCR001 is an investigational, allogeneic cell therapy developed to induce or restore patients’ immune tolerance by establishing stable donor chimerism, giving the recipient an immune system that includes both donor and recipient cells. Phase II data have shown that FCR001 can durably free a significant proportion of Living Donor Kidney Transplant (LDKT) recipients from all chronic immunosuppression by about 12 months after their transplant, without rejection of their transplanted organ. Every patient that was able to discontinue immunosuppression as a result of FCR001 has remained off immunosuppression without rejecting their organ, with median follow-up >5 years post-transplant. Talaris recently launched a Phase III trial in LDKT.
www.talaristx.com

TENAYA THERAPEUTICS
Faraz Ali, CEO
San Francisco, CA
(Private)
Tenaya Therapeutics is a biopharmaceutical company with a mission to discover, develop, and deliver potentially curative treatments that target the underlying causes of heart disease. Tenaya is advancing first-in-class product candidates from three separate platforms – cellular regeneration, gene therapy, and precision medicine. The cellular regeneration platform uses AAV vectors to deliver proprietary transcription factors that drive in vivo reprogramming of resident cardiac fibroblasts into cardiomyocytes, with an initial focus on heart failure post-myocardial infarction. The gene therapy platform uses AAV vectors for the targeted delivery and expression of therapeutic payloads to specific cells in the heart, with an initial focus on the treatment of genetically-defined cardiomyopathies. The precision medicine platform uses isogenic iPSC-derived cardiomyocytes as human disease models to identify new heart failure targets and to screen for therapeutic compounds in a modality agnostic manner, with an initial focus on small molecules for the treatment of genetically-defined dilated cardiomyopathies.
www.tenayatherapeutics.com

TERUMO BLOOD AND CELL TECHNOLOGIES
Colleen Riley, SVP, Innovation and Development
Lakewood, CO
(TYO: 45430)
Terumo Blood and Cell Technologies is a medical device company. Our products, software and services enable customers to collect and prepare blood and cells to help treat challenging diseases and conditions. Our associates around the world believe in the potential of blood and cells to do even more for patients than they do today.
www.terumobct.com

THERMO FISHER SCIENTIFIC
Richard Snyder, Ph.D.,
VP, Science and Technology, Pharma Services Group, Viral Vector Services
Waltham, MA
(NASDAQ: TMO)
Several human clinical trials have been carried out using AAV and LV vectors for the treatment of various diseases. Many of these trials have demonstrated safety as well as therapeutic efficacy, and commercialized products have been licensed for sale. Viral gene transfer vector manufacturing for in vivo and ex vivo applications has largely been in support of early phase clinical trials, but as product candidates move to later development stages, demand is rapidly increasing for commercial grade vectors at a variety of scales. Decisions regarding vector design, manufacturing platform, product configuration, process control, and regulatory strategy have an impact on timelines and resources, raw materials sourcing, and analytical testing. To date, viral vector manufacturing and testing has utilized a diverse set of technologies and workflows. Establishing standardized workflows based on robust technologies will facilitate reproducible and streamlined manufacturing of products with consistent CQAs, broader access, and scalability. Developing standardized platforms that support an efficient path to the clinic and onto the marketplace while reducing risk helps to bring these cutting edge cell and gene therapies to patients in need. www.patheon.com

TMUNITY THERAPEUTICS
Usman Azam, M.D., President and CEO
Philadelphia, PA
(Private)
Tmunity Therapeutics is a private clinical stage biotherapeutics company delivering the potential of next-generation T-cell immunotherapies in cancer. Tmunity integrates collaborations with the University of Pennsylvania with groundbreaking scientific, clinical, manufacturing expertise and a demonstrated track record of its founders and management. The company is developing a diversified portfolio of novel treatments that exhibit best-in-class control over T-cell activation and direction in the body. These personalized immunotherapies are advancing rapidly in the clinic. With headquarters in Philadelphia, Tmunity also has a dedicated cGMP manufacturing facility in Norristown, PA. Tmunity has raised 243 million dollars since inception and has 75 employees.
www.tmunity.com

TREEFROG THERAPEUTICS
Kevin Alessandri, Ph.D., CEO and Chief Technology Officer
Bordeaux, France
(Private)
TreeFrog Therapeutics is a start-up company aiming at providing access to cell therapies for millions of patients. TreeFrog Therapeutics has developed C-Stem, a high-throughput cell encapsulation technology allowing the mass-production and differentiation of stem cells in industrial bioreactors. This proprietary technology platform provides an end-to-end and scalable solution that will dramatically improve the quality of therapeutic cells and reduce treatment costs. Since its incorporation in November 2018, TreeFrog Therapeutics has experienced strong acceleration, securing over €13M in 2019. In January 2020, TreeFrog Therapeutics joined the FrenchTech120, an elite program for the fastest growing technology companies in France. The team – currently 26 staff – moved into a brand new production facility of 13,000 sq. ft. in June 2020.
www.treefrog.fr

TRIUMVIRA IMMUNOLOGICS
Paul Lammers, M.D., President and CEO
Austin, TX
(Private)
Triumvira Immunologics is an immunotherapy company with the vision of developing novel T-cell therapies that are safer and more efficacious than current cancer treatments, including chimeric antigen receptor (CAR) and engineered T-cell receptor (TCR) therapies. Our proprietary T-cell Antigen Coupler (TAC) technology recruits the entire natural T-cell receptor and is independent of the major histocompatibility complex (MHC), allowing for the development of better therapies for a broader range of patients with solid or liquid malignancies and also with diseases other than cancer. Our TAC platform offers competitive advantages over CAR-T and TCR therapies, including: co-opting the natural T-cell signaling pathways; design of a safer and more effective T-cell therapy; demonstration of superiority in preclinical models; a strong IP platform including composition of matter applications, and freedom to operate; an expanding pipeline including solid and liquid tumor indications.
www.triumvira.com

UNIQURE
Jonathan Garen, Chief Business Officer
Lexington, MA
(NASDAQ: QURE)
uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases.
www.uniqure.com

VASCUGEN
Carter Cliff, Founder and CEO
Madison, WI
(Private)
Vascugen is an induced pluripotent stem cell (iPSC) therapy company advancing its novel vasculogenic cell technology to develop and manufacture engineered vascular cells. Our vasculogenic cells have the potential to directly address ischemic disease conditions and to serve as a novel platform for delivery of therapeutic payloads in cardiovascular, solid tumor, and other indications. Vascugen’s research and development facility includes an integrated manufacturing facility for early-stage clinical manufacture of our products.
www.vascugen.com

VERIGRAFT
Petter Bjorquist, Ph.D., CEO
Gothenburg, Sweden
(Private)
VERIGRAFT is a Swedish biotechnology company with a unique, breakthrough technology in the field of advanced therapies and regenerative medicine. We make transplantation possible without the severe risks of immunosuppression, and develop advanced therapies and tissue engineered products that will be able to help millions of patients with serious diseases.
www.verigraft.com

VIACYTE
Kevin D’Amour, Ph.D., Chief Scientific Officer and VP, Research
San Diego, CA
(Private)
ViaCyte is a privately-held clinical stage, regenerative medicine company developing novel stem cell-derived cell replacement therapies, with initial focus on developing products with the potential to provide a functional cure for Type 1 Diabetes (T1D). ViaCyte’s three product candidates utilize proprietary stem cell-derived pancreatic progenitor cells, which are implanted under the skin in durable cell delivery devices. The pancreatic cells are expected to mature and secrete insulin and other endocrine hormones in response to blood glucose levels. PEC-Direct is being developed for high-risk T1D patients with hypoglycemia unawareness, severe hypoglycemic episodes, and minimal to no insulin-producing beta cell function; it requires chronic immunosuppression. PEC-Encap expands the indication to all T1D patients, delivering pancreatic progenitor cells in an immunoprotective device. PEC-QT is being developed to expand usage to all T1D and T2D patients who require exogenous insulin by using cells specifically engineered to be immune-evasive to eliminate the need for immunosuppressants.
www.viacyte.com

VIGENE BIOSCIENCES
Jeffrey Hung, Ph.D., Chief Commercial Officer
Rockville, MD
(Private)
Vigene Biosciences is an awarding winning world leader in plasmid and viral vector development and manufacturing. Vigene features integrated plasmid and viral vector production and analytical service offerings from its 71,000 sq. ft. of start-of-the-art facility with 10 GMP cleanroom suites. Vigene’s mission is to make gene therapy affordable. On the basic research side, Vigene is developing, manufacturing, and distributing state-of-the-art AAV, lentivirus, retrovirus, adenovirus, and plasmid based reagents including HHMI/Janelia Research Campus AAV Biosensors. On the cGMP clinical production side, Vigene combines the proven production technologies with rigorous regulatory compliant cGMP production to meet the needs and expectations of clinical and commercial material clients. Vigene offers FDA and EMA compliant cGMP production for AAV, lentivirus, adenovirus, retrovirus, and plasmids to global pharmaceutical and biotech companies, governmental agencies, and non-profit organization.
www.vigenebio.com

VINETI
Amy DuRoss, Co-Founder and CEO
San Francisco, CA
(Private)
Vineti creates the essential software solution to drive and scale personalized therapies, such as cell and gene therapy. Our leading Personalized Therapy Management (PTM) platform delivers simplicity, compliance, and patient safety to medicine’s most complex supply chain, providing the next-generation technology that advanced therapies need. Today’s personalized therapies require new technology strategies. Vineti combines leading software expertise with deep, first-hand biopharma experience to develop a cloud-based “platform of record” that ensures quality, scale, security, efficiency, traceability, and patient safety. The Vineti platform also provides actionable insights to continually optimize the therapy process, accelerating time to revenue and decreasing costs. Vineti partners with a range of small and large pharmaceutical developers, and offers the only independent platform to have been part of successful, expedited cell therapy approvals in the U.S. and EU. Vineti supports hundreds of medical centers worldwide and was named a World Economic Forum Technology Pioneer in 2019.
www.vineti.com

VISICELL MEDICAL
Mya Thu, Co-Founder and CEO
San Diego, CA
(Private)
Visicell, “GPS for cell therapy”, provides non-invasive, imaging-based cell tracking solutions to customers in a multibillion dollar cell therapy industry. Our patent-pending solutions arm users with early and rapid clinical information for timely decision making on treatment strategy by tracking therapeutic cells and their proper tumor and target engagement in the body. We offer products and services via B2B sale, and establish strategic partnership for long term vision. Our mission is to transform how diseases are diagnosed and treated through designing, developing, and commercializing theranostic nanomaterials that provide real-time insight to patients, doctors, and researchers on product performances to guide treatment strategy as well as accelerate development from laboratory to market.
www.visicellmedical.com

VIVEBIOTECH
Natalia Elizalde, Ph.D., Business Development Director
San Sebastian, Spain
(Private)
VIVEbiotech is a GMP CDMO fully focused on the development and GMP manufacture of lentiviral vectors. VIVEbiotech is currently working for customers based in Europe, the U.S., and Asia, manufacturing lentiviral vectors for different applications such as rare diseases and onco-cell therapy (CAR-Ts, TCRs, CAR-NKs, TILs), among others. One of VIVEbiotech´s main features is our specialization in virology. Based on it, the R&D department is focusing its efforts on the development of more cost-effective processes and safer lentiviral vectors (worldwide patented technology called Lentisoma). VIVEbiotech is now immersed in an expansion plan that will soon allow commercial scale manufacturing of lentiviral vectors.
www.vivebiotech.com

WINDMIL THERAPEUTICS
Don Hayden, Chairman and CEO
Philadelphia, PA
(Private)
WindMIL Therapeutics is a clinical-stage company developing a novel class of autologous cell therapies based on marrow-infiltrating lymphocytes (MILs™) for cancer immunotherapy. As the leader in bone marrow-derived T-cell therapies, WindMIL translates novel insights in bone marrow immunology into life-saving cancer immunotherapeutics for patients. The company’s proprietary process to activate, transform, and expand T-cells offers unique immunotherapeutic advantages, including inherent tumor-specificity, high cytotoxic potential, and long persistence.
www.windmiltx.com

 

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