Company Presentations

Company Presenters Announced!

The Cell & Gene Meeting on the Mesa includes a pipeline loaded with partnering opportunities. Collectively, the slate of 80 presenting companies are working on a number of unique products with an extensive range of possible therapeutic applications. Don’t miss your chance to meet with senior executives from the companies below.

2019 Company Presenters

Adaptimmune_95AGTC
BlueRock_90
DiscGenics_100
Fibrocell_90
Healios_85
Homology

MaxCyte

Precision-Biosciences_90
ReNeuron_80
Sangamo_80SCM-Lifescience
Sentien_65
StemBioSys
Terumo-BCT
ViaCyte

4D MOLECULAR THERAPEUTICS
David Kirn, M.D., Co-Founder and CEO
Oct. 3 | 12:00pm | Cognate Bioservices Ballroom
Emeryville, CA
(Private)
4DMT is a gene therapy company with a transformative discovery platform – Therapeutic Vector Evolution – that empowers the company to invent optimized and proprietary AAV vectors, each specifically tailored for the treatment of a rare disease with a high unmet medical need. www.4dmoleculartherapeutics.com

ABEONA THERAPEUTICS
João Siffert, M.D., CEO, Head of Research and Development, and Chief Medical Officer
Oct. 3 | 2:30pm | Cognate Bioservices Ballroom
New York, NY
(NASDAQ: ABEO)
Abeona Therapeutics is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The company’s multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. www.abeonatherapeutics.com

Adaptimmune_165x65

ADAPTIMMUNE
Kimberly Freeman, VP, Commercialization Planning
Oct. 3 | 2:15pm | BlueRock Therapeutics Ballroom
Oxfordshire, UK
(NASDAQ: ADAP)
Adaptimmune, a leader in T-cell therapy, has clinical trials ongoing for three wholly owned SPEAR T-cells (ADP-A2M4 (MAGE-A4), ADP-A2M10 (MAGE-A10), and ADP-A2AFP (AFP)) 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. The foundation of our TCR therapies is the work we did with NY-ESO SPEAR T-cells, which we transitioned to GSK in 2018, showing responses in two solid tumors and treating more than 90 patients in six different indications. www.adaptimmune.com

AGTC_100x65

AGTC
Sue Washer, President and CEO
Oct. 2 | 5:15pm | Cognate Bioservices Ballroom
Alachua, FL
(NASDAQ: AGTC)
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP), achromatopsia (ACHM CNGB3 and ACHM CNGA3), and X-linked retinoschisis (XLRS). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), other ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture, and physical delivery of gene therapy products. www.agtc.com

AIVITA BIOMEDICAL
Hans Keirstead, Ph.D., Chairman and CEO
Oct. 2 | 11:00am | Cognate Bioservices Ballroom
Irvine, CA
(Private)
Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to develop cutting edge therapies and commercial products that improve quality of life. AIVITA has developed a next-generation, personalized cancer immunotherapy that primes the patient’s immune system to target tumor cells, including the tumor-initiating cells which drive the growth and spread of the disease. Successive improvements in manufacturing at AIVITA have made this personalized treatment reliable, quick and economical to produce. The treatment is the subject of a Phase Ib trial for melanoma, two Phase II clinical trials for ovarian cancer and glioblastoma, and a commercialization application under review for melanoma. All sales of AIVITA’s proprietary rejuvenating skincare line, ROOT OF SKIN™, support these trials. www.aivitabiomedical.com

AMERICAN GENE TECHNOLOGIES
David Pauza, Ph.D., Chief Scientific Officer
Oct. 4 | 10:45am | Cognate Bioservices Ballroom
Rockville, MD
(Private)
America Gene Technologies (AGT) is a gene and cell therapy company with a proprietary gene-delivery platform to rapidly develop cures to infectious diseases, cancers, and monogenic disorders. AGT’s mission is to transform lives through genetic medicines that rid the body of disease. Its lead candidate for an HIV cure is expected to enter the clinic in 2019. In parallel, AGT has a patented immuno-oncology approach to stimulate the body’s immune system to destroy cancer. AGT has also developed a synthetic gene to cure Phenylketonuria (PKU). This treatment received an Orphan Drug Designation from the U.S. Food and Drug Administration and is expected to reach the clinic in 2020. www.americangene.com

AMICUS THERAPEUTICS
Bradley Campbell, President and COO
Oct. 3 | 2:00pm | BlueRock Therapeutics Ballroom
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. www.amicusrx.com

ARUVANT SCIENCES
Paul Oppold, CEO
Oct. 3 | 2:15pm | Cognate Bioservices Ballroom
Cincinatti, OH
(Private)
Aruvant Sciences is a clinical-stage biotech company focused on developing innovative gene therapies for patients suffering with hemoglobinopathies. The lead candidate in Aruvant’s pipeline, ARU-1801, is an investigational gene therapy for sickle cell disease and beta thalassemia. ARU-1801 utilizes proprietary technology intended to increase functioning red blood cells by inserting a modified fetal hemoglobin gene into autologous stem cells through a lentiviral vector. Furthermore, the Reduced Intensity Conditioning (RIC) regimen of ARU-1801 has been designed to improve patient experience and access. ARU-1801 is currently being investigated in a Phase I/II clinical study. Two patients have been dosed in the study so far, both of whom have shown promising results. Aruvant is led by a team with a successful track record of identifying innovative therapies and rapidly executing on clinical development plan to deliver value to patients. www.aruvant.com

ASTELLAS INSTITUTE FOR REGENERATIVE MEDICINE
Yoshitsugu Shitaka, Ph.D., President
Oct. 4 | 11:00am | Cognate Bioservices Ballroom
Marlborough, MA
(Private)
The Astellas Institute for Regenerative Medicine (AIRM) was established in May 2016 following Astellas’ acquisition of Ocata Therapeutics. Headquartered in Marlborough, MA, and supported by a research team in Tsukuba, Japan, AIRM is a wholly-owned subsidiary of Astellas and serves as the company’s global hub for regenerative medicine and cell therapy research in ophthalmology and other therapeutic areas that have few or no available treatment options. Together with AIRM, Astellas is focused on early discovery of cell therapy products for a variety of diseases through a combination of in-house research and external collaborations, using existing technologies and the creation of proprietary technologies. www.astellas.com

ATARA BIOTHERAPEUTICS
Pascal Touchon, , President and CEO
Oct. 2 | 11:15am | BlueRock Therapeutics Ballroom
San Francisco, CA
(NASDAQ: ATRA)
Atara Biotherapeutics is a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune, and viral diseases. Atara’s technology platform leverages research collaborations with leading academic institutions with the company’s scientific, clinical, regulatory, and manufacturing expertise. Atara’s pipeline includes tab-cel® (tabelecleucel), which is in Phase III development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disorder (EBV+ PTLD) as well as in earlier stage development for other EBV-associated hematologic malignancies and solid tumors, including nasopharyngeal carcinoma (NPC); T-cell immunotherapies targeting EBV antigens believed to be important for the potential treatment of multiple sclerosis; and next-generation chimeric antigen receptor T-cell (CAR-T) immunotherapies. The company was founded in 2012 and is co-located in South San Francisco and Southern California. Our Southern California hub is anchored by the state-of-the-art Atara T-cell Operations and Manufacturing (ATOM) facility in Thousand Oaks, California. www.atarabio.com

ATHERSYS
Gil Van Bokkelen, Ph.D., Chairman and CEO
Oct. 3 | 5:45pm | BlueRock Therapeutics Ballroom
Cleveland, OH
(NASDAQ: ATHX)
Athersys is an international biotechnology company engaged in the development of therapeutic products designed to extend and enhance the quality of human life. The company is developing its MultiStem® cell therapy product, a patented, adult-derived “off-the-shelf” stem cell product, initially for disease indications in the critical care spectrum including for neurological, cardiovascular, and inflammatory and immune disease, and other areas. The company has several ongoing clinical trials evaluating this potential regenerative medicine product. These include an ongoing Phase III pivotal trial for treating ischemic stroke (which has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. Food and Drug Administration, and is being conducted under an SPA), a Phase II program in treating Acute Respirator Distress Syndrome (ARDS), which has also received Fast Track Designation, and multiple other programs. Athersys has forged strategic partnerships and a broad network of collaborations to further advance MultiStem cell therapy toward commercialization. www.athersys.com

AUTOLUS
Chris Vann, Chief Commercial Officer
Oct. 3 | 2:30pm | BlueRock Therapeutics Ballroom
London, UK
(NASDAQ: AUTL)
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 tumors. Autolus believes 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
Oct. 4 | 9:30pm | Cognate Bioservices Ballroom
Kildare, Ireland
(Private)
Avectas is a cell engineering technology business, enabling the manufacture of cell therapies. Avectas’ technology, Solupore, delivers advanced molecules such as mRNA, proteins, and gene editing tools to a range of primary cell types including T-cells for immuno-oncology and gene editing applications. Avectas is initially validating its technology through partnership and licensing agreements with cell therapy companies whilst, in parallel, embarking on an ‘own product’ strategy through strategic placement of the technology in leading immunology academic centres, in-licensing, and acquisition. www.avectas.com

AVROBIO
Geoff MacKay, President and CEO
Oct. 2 | 5:30pm | BlueRock Therapeutics Ballroom
Cambridge, MA
(NASDAQ: AVRO)
AVROBIO is a Phase II clinical-stage gene therapy company. AVROBIO is focused on the development of its gene therapy candidate, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis, and Pompe disease. The company’s plato™ platform includes a proprietary vector system, automated cell manufacturing solution, and refined conditioning regimen with therapeutic drug monitoring. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. www.avrobio.com

AXOVANT GENE THERAPIES
Shankar Ramaswamy, M.D., Chief Business Officer
Oct. 3 | 4:30pm | BlueRock Therapeutics Ballroom
New York, NY
(NASDAQ: AXGT)
Axovant, part of the Roivant family of companies, is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (including 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

BIOLIFE SOLUTIONS
Mike Rice, CEO
Oct. 4 | 9:45am | Cognate Bioservices Ballroom
Bothell, WA
(NASDAQ: BLFS)
BioLife Solutions is a leading supplier of cell and gene therapy biopreservation tools. Our proprietary CryoStor® freeze media and HypoThermosol® shipping and storage media are highly valued in the regenerative medicine, biobanking, and drug discovery markets. These biopreservation media products are serum-free and protein-free, fully defined, and are formulated to reduce preservation-induced cell damage and death. Our recently acquired ThawStar® family of automated cell thawing devices reduce therapeutic and economic risk for cell and gene therapy developers by reducing the potential of administering a non-viable dose. www.biolifesolutions.com

Bluebird-Bio_160x65

BLUEBIRD BIO
Jeffrey Walsh, Chief Strategy Officer
Oct. 2 | 11:30am | BlueRock Therapeutics Ballroom
Cambridge, MA
(NASDAQ: BLUE)
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, MA headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become accessible to all those that can benefit. bluebird bio is a human company powered by human stories. We are putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma, using three gene therapy technologies: gene addition, cell therapy, and (megaTAL-enabled) gene editing. bluebird bio has nests in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina; and Zug, Switzerland. www.bluebirdbio.com

BlueRock_160x65

BLUEROCK THERAPEUTICS
Emile Nuwaysir, Ph.D., CEO
Oct. 2 | 11:45am | Cognate Bioservices Ballroom
Cambridge, MA
(Private)
BlueRock Therapeutics is an engineered cell therapy company advancing its novel CELL+GENE™ platform to develop, manufacture, and deliver an entirely new generation of authentic and engineered cell therapies across three therapeutic areas: neurology, immunology, and cardiology. BlueRock’s cell differentiation technology recapitulates the cell’s developmental biology to produce authentic cell therapies that can be further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock was founded in 2016 by Versant Ventures and capitalized with one of the largest-ever Series A financings in biotech history by Bayer AG and Versant. BlueRock’s culture is defined by scientific innovation, the highest ethical standards, and an urgency to bring transformative treatments to all who would benefit. www.bluerocktx.com

B-MOGEN BIOTECHNOLOGY
Jeff Liter, President and CEO
Oct. 4 | 9:15am | Cognate Bioservices Ballroom
Minneapolis, MN
(Private)
B-MoGen Biotechnologies is a genome engineering company that was established to address complex cell engineering challenges in order to accelerate the study of human diseases and the development of novel therapies. The B-MoGen team is equipped with proprietary tools and knowledge that have been developed into finely honed methods for efficient cellular engineering. We utilize this unique expertise to provide three distinct services that address highly complex gene delivery and gene editing. We offer a non-viral transposon-based gene delivery platform to support the T-Cell immunotherapy industry; we are the first company in the world to demonstrate genome engineering of the mitochondria genome; and we offer fee-for-service custom cell engineering of both cell lines and, uniquely, primary human lymphohematopoietic cells for cancer research, drug screening, and antibody validation. www.bmogen.com

Caladrius_130x65

CALADRIUS BIOSCIENCES
David Mazzo, Ph.D., President and CEO
Oct. 2 | 10:45am | Cognate Bioservices Ballroom
Basking Ridge, NJ
(NASDAQ: CLBS)
Caladrius is a late-stage therapeutics development biopharmaceutical company pioneering advancements of cell therapies for select cardiovascular and autoimmune diseases. Our leadership team collectively has decades of biopharmaceutical development experience and world-recognized scientific achievement in the fields of cardiovascular and autoimmune disease, among other areas. Our current product candidates include three developmental treatments for cardiovascular diseases based on our CD34+ cell therapy platform: CLBS12, recipient of a SAKIGAKE designation, in Phase II testing in Japan and eligible for early conditional approval for the treatment of critical limb ischemia; CLBS14-CMD, subject of the proof-of-concept ESCaPE-CMD clinical trial in the US for the treatment of coronary microvascular dysfunction; and CLBS14-NORDA, recipient of a RMAT designation in the US and for which we are in preparations to commence a Phase III clinical trial in no option refractory disabling angina. www.caladrius.com

CAPRICOR THERAPEUTICS
Linda Marban, Ph.D., CEO
Oct. 4 | 11:15am | Cognate Bioservices Ballroom
Los Angeles, CA
(NASDAQ: CAPR)
Capricor Therapeutics (NASDAQ: CAPR) is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor is also exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders. www.capricor.com

CARIBOU BIOSCIENCES
Timothy Herpin, Ph.D., Chief Business Officer
Oct. 3 | 11:30am | Cognate Bioservices Ballroom
Berkeley, CA
(Private)
Caribou Biosciences is a leading company in CRISPR genome editing founded by pioneers of CRISPR-Cas9 biology. We are using a next-generation gene editing technology to develop a pipeline of off-the-shelf CAR-T cell therapies for the treatment of cancer. We are also using gene editing to develop novel microbiome based therapies. Caribou’s tools and technologies provide transformative capabilities to therapeutic development, agricultural biotechnology, industrial biotechnology, and basic and applied biological research. www.cariboubio.com

CARISMA THERAPEUTICS
Steven Kelly, President and CEO
Oct. 2 | 2:30pm | Cognate Bioservices Ballroom
Philadelphia, PA
(Private)
CARISMA Therapeutics is developing a novel platform technology based on ex vivo engineered Macrophages. Initial applications are Autologous CAR Macrophages, an approach that combines powerful chimeric antigen receptor technology and adoptive cell transfer, two techniques with an established clinical track record in cancer therapy, to macrophages, cells known to infiltrate the solid tumor microenvironment. Ample in vitro and in vivo data demonstrate the ability of CAR-macrophages to traffic to solid tumors and to selectively eliminate tumor cells via phagocytosis. CAR-Macrophages will also leverage macrophage’s natural antigen presenting capability to stimulate an adaptive immune response. The first product under development is a HER2 targeted CAR-Macrophage with an anticipated IND filing in Q419. Additional CAR-Macrophages targeting other solid tumor antigens are under development. www.carismatx.com

CARTHERICS
Richard Boyd, Ph.D., Chief Scientific Officer
Oct. 4 | 12:00pm | Cognate Bioservices Ballroom
Melbourne, Australia
(Private)
Cartherics is a private Melbourne-based biotechnology company, focused on developing cutting edge technologies to empower the immune system to specifically target and ultimately eliminate cancer. The focus technology is Chimeric Antigen Receptors for T-cells (CAR-T) and NK cells (CAR-NK). The Cartherics business model is distinguished by the development of novel single or dual specific CAR-T/NK cells targeting strategically chosen cancer antigens coupled to editing out of genes that compromise killing function. The key antigen is broadly expressed by adenocarcinomas, and cutaneous T-cell lymphoma but is virtually absent in normal tissue. The primary goal of the company is to develop “off-the-shelf” allogeneic CAR-NK and CAR-T cells differentiated from iPSC derived from homozygous HLA haplotype donors, using viral-free transduction to genetically sculpture the iPSC. Genetically modified single specificity CAR-T cells are planned for near term autologous Phase I clinical trial(s). Cartherics holds patent applications for its key platforms. www.cartherics.com

CCRM
Mitch Sivilotti, Chief Operating Officer
Oct. 4 | 10:15am | Cognate Bioservices Ballroom
Toronto, ON
(Private)
CCRM, a Canadian not-for-profit funded by the Government of Canada, the Province of Ontario, and leading academic and industry partners, supports the development of regenerative medicines and associated enabling technologies, with a specific focus on cell and gene therapy. A network of academic researchers, leading companies, strategic investors, and entrepreneurs, CCRM aims to accelerate the translation of scientific discovery into new companies and marketable products for patients, with specialized teams, funding and infrastructure. CCRM sources and evaluates IP from around the globe, offers various consulting services, conducts development projects with partners, and establishes new companies built around strategic bundles of intellectual property. CCRM has a 40,000 square foot space dedicated to advanced cell manufacturing that includes a fully resourced process development facility and a Good Manufacturing Practices facility. CCRM is hosted by the University of Toronto and was launched in Toronto’s Discovery District in June 2011. www.ccrm.ca

CELLECT BIOTECHNOLOGY
Shai Yarkoni, M.D., Ph.D., CEO
Oct. 2 | 11:30am | Cognate Bioservices Ballroom
Kfar 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 stem cell selection technology platform, the ApoGraftTM; 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 has broad potential application, enabling the stem-cell based industry to produce safe, robust, and affordable stem cells for any usage, in any indication. The first ApoGraftTM application in clinical development is the prevention of Graft versus Host Disease (GvHD) in bone marrow transplantation recipients. Early results from Phase I/II study show good engraftment, with perfect safety score and no ApoGraftTM-related toxicity. Cellect’s pipeline also includes purification and expansion of mesenchymal stem cells and preclinical programs including CAR-T. Cellect’s technology is protected by eight patent families. www.cellect.co

CELLS FOR CELLS
Roberto Bobadilla, Ph.D., CEO
Oct. 3 | 10:45am | Cognate Bioservices Ballroom
Santiago, 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

CELSENSE
Eric Ahrens, Ph.D., Founder and Chief Scientific Officer
Oct. 3 | 10:00am | Cognate Bioservices Ballroom
Pittsburgh, PA
(Private)
Celsense offers MRI tracer agents used to visualize the trafficking of transplanted cells in vivo. The company’s lead product CS-1000 is the subject of Drug Master Files at FDA and Health Canada and authorized for use in clinical trials. A new formulation called FETRIS is the subject of a pending DMF and offers increased sensitivity. www.celsense.com

COVANCE
Steve Anderson, Ph.D., Chief Scientific Officer
Oct. 3 | 5:15pm | Cognate Bioservices Ballroom
Princeton, NJ
(NYSE: LH)
Covance is the drug development business of Laboratory Corporation of America Holdings (LabCorp). 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 and its subsidiaries around the world. www.covance.com

Cynata_140x65

CYNATA THERAPEUTICS
Ross Macdonald, Ph.D., CEO
Oct. 3 | 5:00pm | Cognate Bioservices Ballroom
Melbourne, Australia
(ASX:CYP)
Cynata Therapeutics is an Australian clinical stage stem cell and regenerative medicine company that is developing a therapeutic stem cell 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 mesenchymal stem cells (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 was recently completed, meeting all safety and efficacy endpoints. The company expects to commence three Phase II trials during 2019. Cynata has a strategic partnership with Fujifilm, which is one of the company’s larger shareholders. www.cynata.com

DiscGenics_165x65

DISCGENICS
Flagg Flanagan, Chairman and CEO
Oct 3. | 3:15pm | Cognate Bioservices Ballroom
Salt Lake City, UT
(Private)
DiscGenics is a privately-held, clinical stage biotechnology company focused on developing regenerative cell-based therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. DiscGenics’ first product candidate, IDCT, is a homologous, allogeneic, injectable cell therapy that utilizes biomedically engineered progenitor cells derived from intervertebral disc tissue to treat degenerative disc disease. The company is currently conducting two regulator-allowed, prospective, randomized, double-blind clinical studies in the U.S. and Japan to evaluate the safety and preliminary efficacy of IDCT as a non-surgical, potentially regenerative treatment for patients with mild to moderate degenerative disc disease. www.discgenics.com

 

EXCELLTHERA
Guy Sauvageau, M.D, Ph.D., CEO
Oct. 2 | 1:45pm | Cognate Bioservices Ballroom
Montreal, Quebec
(Private)
ExCellThera is an advanced clinical stage biotechnology company delivering molecules and bioengineering solutions to expand stem and immune cells for use in novel one time curative therapies for patients with hematologic malignancies, autoimmune, and other diseases. ExCellThera’s lead solution combines a proprietary small molecule, UM171, and an optimized culture system. In pursuit of better treatments for patients, the company is building out its portfolio of products, as well as supporting best-in-class clinical trials. www.excellthera.com

FATE THERAPEUTICS
Dan Shoemaker, Ph.D., Chief Scientific Officer
Oct. 3 | 5:15pm | BlueRock Therapeutics Ballroom
San Diego, CA
(NASDAQ: FATE)
Fate Therapeutics is a clinical stage biopharmaceutical company dedicated to the development of next-generation cellular immunotherapies for cancer and immune disorders. Our first-in-class cell therapy product candidates undergoing clinical development today utilize healthy donor cells, which we modify ex vivo using pharmacologic modulators, such as small molecules, to improve the cells’ biological properties and therapeutic function. We are also pioneering a revolutionary approach to cell therapy using renewable master induced pluripotent stem cell (iPSC) lines generated from our proprietary iPSC platform to derive cell therapy product candidates that can be delivered off-the-shelf for the treatment of a large number of patients. Our cell therapy candidate pipeline is comprised of immuno-oncology programs, including off-the-shelf NK- and T-cell product candidates derived from master iPSC lines, and immuno-regulatory programs, including product candidates to prevent life-threatening complications in patients undergoing hematopoietic cell transplantation and to promote immune tolerance in patients with autoimmune disease. www.fatetherapeutics.com

Fibrocell_165x65

FIBROCELL SCIENCE
John Maslowski, President and CEO
Oct. 3 | 2:00pm | Cognate Bioservices Ballroom
Exton, PA
(NASDAQ: FCSC)
Fibrocell is a cell and gene therapy company focused on improving the lives of people with rare diseases of the skin and connective tissue. The company is utilizing its proprietary autologous fibroblast technology to develop personalized biologics that target the underlying cause of disease. Fibrocell’s pipeline of localized gene therapy candidates include FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa, or RDEB, a life-threatening genetic disorder diagnosed in infancy with no cure or treatment approved by the U.S. Food and Drug Administration. The company is also developing FCX-013 for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation, a leader in synthetic biology. FCX-007 is being co-developed in collaboration with Castle Creek Pharmaceuticals. www.fibrocell.com

FLEXION THERAPEUTICS
Adam Muzikant, Ph.D., SVP, Business Development
Oct. 2 | 2:30pm | BlueRock Therapeutics Ballroom
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 U.S. Food and Drug Administration-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 pain relief and disease modification in patients with knee OA. FX201 is designed to durably stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present in the joint. FX201 has completed GLP toxicology studies and the manufacturing process has been transferred to a US CMO. An IND filing and initiation of clinical trials with FX201 in the US is expected in 2019. www.flexiontherapeutics.com

FUJIFILM CELLULAR DYNAMICS
Nicholas Manusos, Chief Operating Officer
Oct. 2 | 5:00pm | Cognate Bioservices Ballroom
Madison, WI
(Private)
FUJIFILM Cellular Dynamics Inc. (FCDI) is a leading developer and supplier of human cells used in discovery, toxicity testing, stem cell banking, and cell therapy development. The company partners with innovators from around the world to combine biologically relevant human cells with the newest technologies to drive advancements in medicine and healthier living. FCDI’s technology offers the potential to create induced pluripotent stem cells (iPSCs) from anyone, starting with a standard blood draw, and followed by the powerful capability to develop into virtually any cell type in the human body. Our proprietary manufacturing system produces billions of cells daily, resulting in inventoried iCell® Products and donor-specific MyCell® Products in the quantity, quality, purity, and reproducibility required for drug and cell therapy development. www.fujifilmcdi.com

GAMIDA CELL
Tom Klima, Chief Commercial Officer
Oct. 2 | 11:45am | BlueRock Therapeutics Ballroom
Boston, MA
(NASDAQ: GMDA)
Gamida Cell is a clinical stage biopharmaceutical company committed to developing advanced cell therapies with the potential to cure blood cancers and rare, serious hematologic diseases. We are leveraging our proprietary nicotinamide-based, or NAM-based, cell expansion technology to develop product candidates designed to address the limitations of cell therapies. www.gamida-cell.com

GINER LIFE SCIENCES
Linda Tempelman, Ph.D., CEO
Oct. 4 | 10:00am | Cognate Bioservices Ballroom
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 technology includes the control, power and recharging system for a fully implantable oxygen generator. GLS has partnered with a leading membrane technology for a system with oxygen-enabled cell capsules. 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 type 1 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

GYROSCOPE THERAPEUTICS
Susan Hill, Ph.D., Chief Business Officer
Oct. 3 | 4:15pm | Cognate Bioservices Ballroom
London, UK
(Private)
Gyroscope: Vision for Life Gyroscope Therapeutics is developing gene therapy beyond rare disease and using it to treat a leading cause of blindness, dry age-related macular degeneration (dry AMD). Currently, 35M people are affected by dry AMD around the world and there are no approved treatments. Our investigational therapy, GT005, is designed to restore balance to a part of our immune system called the complement system. The goal is to slow, or possibly stop the progression of dry AMD. Patients in our Phase I/II clinical trial receive a single dose therapy through a one time procedure into the back of 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, we are building a pipeline of medicines to preserve sight and fight the devastating impact of blindness. www.gyroscopetx.com

Healios_150x65

HEALIOS
Richard Kincaid, Executive Officer and Chief Finance Officer
Oct. 2 | 4:30apm | Cognate Bioservices Ballroom
Tokyo, Japan
(JPX 4593.T)
Healios is a Tokyo-based, clinical stage world leader in regenerative medicine and cell therapy. After its founding in 2011, Healios listed on the Tokyo Stock Exchange in 2015 and currently has more than 130 people based in its Japan and US offices. Healios leverages the attractive Japanese regulatory framework for regenerative medicine to rapidly and efficiently deliver results for patients, its partners, and stakeholders. It is currently running clinical trials for ischemic stroke (Phase II/III) and acute respiratory distress syndrome (Phase II) using bone marrow-derived allogeneic adult stem cells. At the same time, Healios is developing a next generation gene edited iPSC platform to remove the need for immunosuppressants, improve scalability, and enhance effectiveness. Its iPSC programs seek to address multiple areas of unmet medical need in the ophthalmology, liver disease, and immuno-oncology fields, and are supported by manufacturing platform development undertaken by Healios and its industry and academic partners. www.healios.co.jp/en

HOMOLOGY MEDICINES
Arthur Tzianabos, Ph.D., President and CEO
Oct. 2 | 2:15pm | BlueRock Therapeutics Ballroom
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

IMMUSOFT CORPORATION
Sean Ainsworth, CEO
Oct. 4 | 11:45am | Cognate Bioservices Ballroom
Seattle, WA
(Private)
Immune System Programming (ISP™) is a proprietary ex vivo culture system developed by Immusoft to expand and differentiated human B cells into gene-modified plasmablasts (ISP™ cells). Plasmablasts are the cell type responsible for long-term humoral immunity, are the bodies’ natural protein-producing cell type, and have the ability to become long-lived in the body where they can engraft and survive for years to decades. ISP™ cells can be an ideal cell type for delivery of therapeutics since they continuously secrete very high levels of protein and can migrate to and occupy diverse tissues, including bone marrow, spleen, mucosal tissues, and at sites of inflammation. Immusoft’s autologous ISP™ cell product(s) have been reprogrammed to produce a variety of protein therapeutic drugs, including antibodies, enzymes, and blood clotting factors for hemophilia – treatments which are currently only available through intravenous infusion. www.immusoft.com

IOVANCE BIOTHERAPEUTICS
Maria Fardis, Ph.D., CEO
Oct. 3 | 5:30pm | BlueRock Therapeutics Ballroom
San Carlos, CA
(NASDAQ: IOVA)
Iovance Biotherapeutics intends to commercialize autologous cell therapy products that amplify the body’s own immune response to eradicate solid tumors or attack blood cancers. The company is currently conducting the pivotal study innovaTIL-01 in patients with metastatic melanoma. In addition, the company’s tumor infiltrating lymphocyte (TIL) therapies are being investigated for the treatment of patients with locally advanced, recurrent or metastatic cancers including cervical, head and neck, and non-small cell lung cancer. www.iovance.com

IVERIC BIO
Keith Westby, Chief Operating Officer
Oct. 2 | 5:30pm | Cognate Bioservices Ballroom
New York, NY
(NASDAQ: ISEE)
IVERIC bio is a biotechnology company with a focus on the discovery and development of novel gene therapy solutions to treat orphan inherited retinal diseases with unmet medical needs. Vision is our mission. www.ivericbio.com

KYTOPEN
Paulo Garcia , Ph.D., Co-Founder and CEO
Oct. 3 | 4:45pm | Cognate Bioservices Ballroom
Cambridge, MA
(Private)
Kytopen is a spin out of MIT developing proprietary platforms for non-viral delivery of payloads for ex vivo cell therapy discovery and manufacturing. The scalable solution combines microfluidics and automation to make transfection easier, faster, and more cost-effective than the state-of-the art solutions. Kytopen is integrating their platforms with automated liquid handling systems for processing small volumes and also developing a large volume platform for therapeutic applications. The non-viral solution will accelerate the time-to-market of next-generation cell and gene therapies by enabling discovery of therapeutic targets and reducing the cell therapy manufacturing costs. Kytopen recently closed a $3.6M seed funding round and is the recipient of a Phase II SBIR grant from the National Science Foundation (NSF). www.kytopen.com

LINEAGE CELL THERAPEUTICS
Brian Culley, CEO
Oct. 3 | 5:00pm | BlueRock Therapeutics Ballroom
Alameda, CA
(NYSE: LCTX)
Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs. Lineage’s programs are based on its proprietary cell-based therapy platform and associated development and manufacturing capabilities. With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials. These differentiated cells are developed either to replace or support cells that are dysfunctional or absent due to degenerative disease or traumatic injury or administered as a means of helping the body mount an effective immune response to cancer. Lineage’s clinical assets include (i) OpRegen®, a retinal pigment epithelium transplant therapy in Phase I/IIa development for the treatment of dry age-related macular degeneration, a leading cause of blindness in the developed world; (ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase I/IIa development for the treatment of acute spinal cord injuries; and (iii) VAC2, an allogeneic cancer immunotherapy of antigen-presenting dendritic cells currently in Phase I development for the treatment of non-small cell lung cancer. www.lineagecell.com

LOGICBIO
Fred Chereau, CEO
Oct. 2 | 5:45pm | BlueRock Therapeutics Ballroom
Cambridge, 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. Headquartered in Cambridge, MA, LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families. www.logicbio.com

MAGENTA THERAPEUTICS
Jason Gardner, Ph.D., President and CEO
Oct. 3 | 4:45pm | BlueRock Therapeutics Ballroom
Cambridge, MA
(NASDAQ: MGTA)
Headquartered in Cambridge, MA, Magenta Therapeutics is a clinical stage biotechnology company developing novel medicines for patients with autoimmune diseases, blood cancers and genetic diseases. By creating a platform focused on critical areas of unmet need, Magenta Therapeutics is pioneering an integrated approach to allow more patients to receive one-time, curative therapies by making the process more effective, safer, and easier. www.magentatx.com

MAXCYTE
Douglas Doerfler, President and CEO
Oct. 2 | 2:15pm | Cognate Bioservices Ballroom
Gaithersburg, MD
(LON: MXCT)
MaxCyte is a global clinical stage cell-based therapies and life sciences company applying its proprietary cell engineering platform to deliver the advances of cell-based therapy to patients with high unmet medical needs. MaxCyte is developing novel CARMA™ therapies for its own pipeline, with its first drug candidate in Phase I. CARMA is MaxCyte’s mRNA-based proprietary therapeutic platform for autologous cell therapy for the treatment of solid cancers. In addition, through its life sciences business, MaxCyte leverages its Flow Electroporation® Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its instruments worldwide, with all top ten global biopharmaceutical companies. The company now has 70+ partnered program licenses in cell therapy with 35+ licensed for clinical use, including four announced commercial licenses covering potentially 30+ products. MaxCyte helps its partners to unlock the full potential of their products. www.maxcyte.com

MEDEOR THERAPEUTICS
Karen Smith, M.D., Ph.D., CEO
Oct. 2 | 11:15am | Cognate Bioservices Ballroom
San Fransisco, CA
(Private)
Medeor Therapeutics is a clinical stage biotechnology company dedicated to discovery, development, and commercialization of personalized cellular immunotherapies to improve outcomes in organ transplant recipients and patients that suffer from hematologic conditions. Medeor’s product candidates each comprise a unique composition of different types of hematopoietic donor-derived cells, based on breakthrough technologies discovered at Stanford University and exclusively licensed by Medeor. These product candidates are designed to produce mixed chimerism, the co-existence of both recipient and donor blood and immune cells in the recipient. For organ transplant patients, mixed chimerism can induce donor-specific immune tolerance, which reduces the likelihood of transplant organ loss, reduces or eliminates chronic immunosuppressive drug therapy, and thereby improves patient outcomes. For hematologic conditions, mixed chimerism has the potential to reduce or eliminate disease symptoms. www.medeortx.com

MIROMATRIX MEDICAL
Jeff Ross, Ph.D., CEO
Oct. 3 | 4:00pm | Cognate Bioservices Ballroom
Eden Prairie, MN
(Private)
Miromatrix is a biotechnology company on a mission to save and drastically improve patients’ lives by eliminating the organ transplant waiting list, including livers, kidneys, and lungs based on its patented perfusion decellularization and recellularization technologies. Our approach has the potential to access these critically underserved markets and our progress has been recently recognized by winning a KidneyX prize and being named Buzz of BIO for Pipelines of Promise. www.miromatrix.com

MolMed_130x65

MOLMED
Luca Alberici, Ph.D., Chief Business Officer
Oct. 2 | 3:15pm | Cognate Bioservices Ballroom
Milan, Italy
(MLMD.MI)
MolMed is a biotech company focused on research, development, manufacturing, and clinical validation of innovative CellandGene based therapies. MolMed built an original dual business model, based on RandD of proprietary products and third parties GMP services. MolMed’s pipeline includes anti-tumour therapeutics in clinical and preclinical development: Zalmoxis® (TK), a cell-based therapy currently in Phase III in high-risk acute leukaemia; Zalmoxis® obtained a CMA from the European Commission and reimbursement conditions in Italy and Germany. CAR-T CD44v6, an immune gene therapy recently authorized for human clinical trials in onco-hematologic indications. MolMed offers a wide range of CellandGene therapy activities for leading EU/US companies, including RL/LV vectors manufacturing and cells transduction, supporting clinical trials and commercial supply. MolMed is headquartered in Milan (Italy) at the San Raffaele DIBIT. MolMed has an operating unit at OpenZone in Bresso and is listed on the main market (MTA) of the Milan stock exchange. www.molmed.com

NOVADIP BIOSCIENCES
Jeff Abbey, CEO
Oct. 3 | 9:15am | Cognate Bioservices Ballroom
Belgium
(Private)
Novadip Biosciences is a pioneer in the development of 3D tissue products derived from adipose stem cells, addressing the need for critical hard and soft tissue reconstruction in patients who currently have limited or no effective treatment options. www.novadip.com

OPSIS THERAPEUTICS
Carter Cliff, Co-Founder
Oct. 2 | 5:45pm | Cognate Bioservices Ballroom
Madison, WI
(Private)
Opsis Therapeutics is developing advanced medicines for patients suffering from inherited retinal diseases (IRD) and age-related macular degeneration (AMD). The company is focused on a pipeline of induced-pluripotent stem cell (iPSC) therapies for direct repair of damaged retina, including retinal pigment epithelium (RPE), photoreceptor (PRP), and an engineered tissue containing both cell types (RPE+PRP). www.opsistx.com

ORCHARD THERAPEUTICS
Mark Rothera, President and CEO
Oct. 2 | 5:00pm | BlueRock Therapeutics Ballroom
London, UK
(NASDAQ: ORTX)
Orchard Therapeutics is a leading global fully integrated commercial stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of ex vivo autologous gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first ex vivo autologous gene therapy approved by the EMA in 2016, three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT), and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele. www.orchard-tx.com

ORGANOVO
Taylor Crouch, President and CEO
Oct. 3 | 5:45pm | Cognate Bioservices Ballroom
San Diego, CA
(NASDAQ: ONVO)
Organovo is a biotech platform company that has developed a leadership position with its revolutionary ability to 3D bioprint tissues with human functionality. The company is pursuing IND-track programs to develop its NovoTissues® to address a number of serious unmet medical needs, initially focusing on liver disease. Organovo’s program for Alpha-1-antitrypsin deficiency received orphan drug designation from the FDA in 2017. The company is also providing access to its ExVive™ in vitro tissue platform to facilitate high value drug discovery and development collaborations. Organovo’s wholly-owned subsidiary, Samsara Sciences, provides the company and its clients with high quality human liver cells for research applications. Organovo is changing the shape of life science research and transforming medical care. www.organovo.com

OXFORD BIOMEDICA
Jason Slingsby, Ph.D., Chief Business Officer
Oct. 2 | 5:15pm | BlueRock Therapeutics Ballroom
Oxford, UK
(LON: OXB)
Using our unique LentiVector® delivery platform, we have created a valuable portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, and CNS disorders. We have strong partnerships with Novartis, Bioverativ (part of the Sanofi Group), Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, and Imperial Innovations, Immune Design, and Orchard Therapeutics, providing them with access to our intellectual property, state-of-the-art production facilities and expertise, and, in addition, we have licensed products and technology rights to Boehringer Ingelheim, Sanofi, and Axovant. These partnerships provide us with multiple income streams, consisting of upfront milestone payments, development and production fees, and potential royalties on future product sales. We plan to progress our wholly-owned products via spin-outs and out-licensing opportunities, while continuing to invest in our LentiVector® platform. We plan to continue our preclinical RandD to discover new potential products. www.oxb.com

PDC*LINE PHARMA
Eric Halioua, President and CEO
Oct. 2 | 1:30pm | Cognate Bioservices Ballroom
Belgium
(Private)
PDC*line Pharma is a clinical stage biotech company that develops an innovative class of cancer vaccine, based on a GMP-grade allogeneic therapeutic cell line of plasmacytoid dendritic cells (PDC*line). PDC*line is much more potent than conventional Dendritic Cell-based vaccines in priming and boosting antitumor antigen-specific cytotoxic T-cells, including T-cells specific for neoantigens, and is synergistic with checkpoint inhibitors.Following a first-in-human phase I feasibility study in melanoma, PDC*line Pharma focuses on the development of PDC*lung01, a candidate for non-small-cell lung cancer and neoantigens (PDC*Neo). The company has so far raised $19.3M including $8.6M in equity and loans from Belgian investors in addition to $10.5M of non-dilutive funding. The company granted an exclusive license to LG Chem Life Sciences Company in South Korea with exclusive option in other Asian countries, for the development and commercialization of the PDC*lung01. The total deal value is $123M plus tiered royalties on net sales in Asia. www.pdc-line-pharma.com

POLARITYTE
Nikolai Sopko, M.D., Ph.D., Chief Scientific Officer and VP, Research and Development
Oct. 3 | 9:45am | Cognate Bioservices Ballroom
Salt Lake City, UT
(NASDAQ: PTE)
PolarityTE is a commercial stage biotechnology and regenerative biomaterials company focused on transforming the lives of patients by discovering, designing and developing regenerative tissue products and biomaterials for medicine, biomedical engineering and material sciences. The PolarityTE® platform technology begins with a small piece of the patient’s own, or autologous, healthy tissue, rather than artificially manipulated individual cells. From this small piece of healthy autologous tissue, the company creates an easily deployable, dynamic and self-propagating product designed to enhance and stimulate the patient’s own cells to regenerate the target tissues. SkinTE™ is commercially available and uses this technology to regenerate full-thickness, functional, hair-bearing skin using a small piece of the patient’s own skin to treat full-thickness burns, chronic wounds, and large cutaneous defects. Its ease of use and one-time application allows its use in outpatient and resource-limited settings while reducing treatment costs and, most importantly, providing the patient with full thickness skin. www.polarityte.com

POSEIDA THERAPEUTICS
Matthew Spears, M.D., Chief Medical Officer
Oct. 2 | 2:45pm | Cognate Bioservices Ballroom
San Diego, CA
(Private)
Poseida Therapeutics is a clinical stage biotechnology company translating next generation gene engineering technologies into lifesaving treatments for patients with high unmet medical need. The company is currently developing a wholly-owned pipeline of cell and gene therapy candidates including autologous and allogeneic CAR-Ts. www.poseida.com

Precision-Biosciences_145x65

PRECISION BIOSCIENCES
Michael Dombeck, SVP, Corporate Development
Oct. 3 | 4:15pm | BlueRock Therapeutics Ballroom
Durham, NC
(NASDAQ: DTIL)
Precision BioSciences is dedicated to improving life through its proprietary genome editing platform, ARCUS. Precision BioSciences 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 BioSciences is actively developing product candidates in three innovative areas: allogeneic CAR-T immunotherapy, in vivo gene correction, and food. www.precisionbiosciences.com

REGENERATIVE PATCH TECHNOLOGIES
Jane Lebkowski, Ph.D., President
Oct. 3 | 11:15am | Cognate Bioservices Ballroom
Menlo Park, CA
(Private)
Regenerative Patch Technologies (RPT) is a California biotechnology company developing a pluripotent stem cell-based implant for the treatment of retinal diseases. CPCB-RPE1 is RPT’s lead product and is composed of stem cell-derived retinal pigmented epithelial cells on an ultrathin parylene membrane that mimics the function of the Bruch’s membrane, CPCB-RPE1 is in clinical trials for the treatment of the dry form of age-related macular degeneration, an disease that effects over one million people in the United States RPT was founded by Drs. Mark Humayun and David R. Hinton from the University of Southern California and Dr. Dennis O. Clegg from UC Santa Barbara. The company has completed enrollment in a Phase I/IIa clinical trial of of CPCB-RPE1 in patients with geographic atrophy, the advanced form of dry age-related macular degeneration. The company is actively planning a Phase IIb clinical trial. www.regenerativepatch.com

REGENXBIO
Olivier Danos, Ph.D., SVP and Chief Scientific Officer
Oct. 2 | 3:00pm | Cognate Bioservices Ballroom
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 licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. REGENXBIO’s approach to gene therapy focuses on developing single administration therapies that target genetic defects or drive cellular production of therapeutic proteins. Its NAV Technology Platform serves as the backbone of gene therapy research and is a widely-utilized gene therapy platform across diverse disease applications. REGENXBIO’s scientific advancements give renewed and meaningful hope to patients with severe, pervasive, and currently incurable diseases. More than 200 subjects to date have been treated in clinical trials using our NAV Technology. www.regenxbio.com

ReNeuron_135x65

RENEURON
Michael Hunt, Chief Financial Officer
Oct. 3 | 11:00am | Cognate Bioservices Ballroom
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. ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L. www.reneuron.com

SANGAMO THERAPEUTICS
Stéphane Boissel, EVP, Corporate Strategy
Oct. 2 | 3:15pm | BlueRock Therapeutics Ballroom
San Francisco, 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
Oct. 3 | 5:30pm | Cognate Bioservices Ballroom
Incheon, Korea
(Private)
SCM Lifescience is a clinical stage biopharmaceutical company focused in development of next-generation stem cell therapeutics for inflammatory-immune diseases with high unmet medical needs. Through over 17 years of research, SCM has obtained intellectual property protection for its high-purity stem cell isolation technology, subfaractionation culturing method, in major markets and serves to provide patients with homogeneous and effective stem cell therapies. SCM Lifescience carries a robust pipeline with three indicaitons in clinical stages. The leading pipeline is SCM-CGH, a bone marrow-derived clonal mesenchymal stem cell therapy for steroid-refractory, chronic graft-versus-host disease(cGVHD). SCM-AGH is being studied for the treatments of severe acute pancreatitis and moderate-to-severe atopic dermatitis. Our research and development efforts are not solely restricted to MSCs. Through open innovation, we are not only seeking technologies that may work synergistically but also a brand new arm in the field of regenerative medicine including DCs, iPSCs, exosomes, and others. www.scmlifescience.com

SENTI BIOSCIENCES
Tim Lu, M.D, Ph.D, Co-Founder and CEO
Oct. 2 | 1:15pm | Cognate Bioservices Ballroom
San Francisco, CA
(Private)
Senti Biosciences is a Series A next-generation biotechnology company that designs gene circuits and programs cells for tremendous therapeutic value. Senti’s mission is to engineer an entirely new class of medicine that will transform people’s lives by curing the most complex diseases. Backed by a syndicate of leading biotech and tech investors including NEA, 8VC, Lux, Pear, Menlo Ventures, and Amgen Ventures, and led by an experienced team with deep translational knowledge of advanced cell therapies, the company is leveraging its proprietary gene circuit repertoire to solve the most pressing unmet needs in oncology and other difficult disease areas. www.sentibio.com

SENTIEN BIOTECHNOLOGIES
Brian Miller, Co-Founder and CEO
Oct. 2 | 4:00pm | Cognate Bioservices Ballroom
Lexington, MA
(Private)
Sentien Biotechnologies is a privately-owned, 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, encapsulates allogeneic mesenchymal stromal cells (MSCs) within an extracorporeal, hollow-fiber device. This design allows for extended 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 currently being evaluated in a Phase Ib/IIa study in subjects with dialysis-requiring acute kidney injury. An interim readout from the study supports safety and provides early evidence of anti-inflammatory effects consistent with the SBI-101 therapeutic hypothesis. Sentien plans to expand its pipeline to include additional systemic inflammatory indications where single-factor agents have not been effective, in both the acute and chronic disease spaces. www.sentienbiotech.com

Sigilon_110x65

SIGILON THERAPEUTICS
Rogerio Vivaldi, M.D., President and CEO
Oct. 3 | 1:30pm | Cognate Bioservices Ballroom
Cambridge, MA
(Private)
Sigilon Therapeutics’ mission is to create immune-protected, engineered human cells that restore normal physiology in a wide range of diseases without generating fibrosis or immune rejection, liberating patients from challenges associated with serious chronic diseases. Treatments based on the Sigilon Therapeutics platform combine advanced cell engineering with cutting-edge innovations in biocompatible materials to pioneer a new class of therapeutics, Shielded Living Therapeutics™ (SLTx), that provide durable, controllable, and safe treatment for serious chronic diseases. In April 2018, Sigilon and Eli Lilly announced a global collaboration to develop encapsulated cell therapies for the potential treatment of type 1 diabetes. www.sigilon.com

SIRION BIOTECH
Christian Thirion, Ph.D., CEO
Oct. 2 | 4:45pm | Cognate Bioservices Ballroom
Martinsried, Germany
(Private)
SIRION Biotech was founded in 2005 to lead the next generation of viral vector technologies for gene and cell therapy as well as vaccine development. SIRION Biotech evolves novel therapeutic viral vectors and technology platforms based on lenti-, adeno-, and adeno-associated viruses, which expedite gene therapy research and advances drug development of its partners. SIRION Biotech’s proprietary LentiBOOST™ technology improves transduction efficiency of lentiviral and retroviral vectors in cell therapy and is currently used in a number of clinical trials in the U.S. and in Europe. A first approved CD34+-based cell product was manufactured with LentiBOOST™ . In the field of gene therapy, SIRION Biotech develops AAV vectors and technology, addressing vector specificity and efficacy with directed evolution, and process development for GMP production. SIRION Biotech pursues a collaborative strategy with multiple academic and clinical development partners, e.g. in the field of ophthalmology and neurodegenerative diseases. www.sirion-biotech.com

StemBioSys_170x65

STEMBIOSYS
Robert Hutchens, President and CEO
Oct. 4 | 11:30am | Cognate Bioservices Ballroom
San Antonio, TX
(Private)
StemBioSys is a privately-held, San Antonio-based biomedical company that manufactures and develops innovative cell and matrix technologies to support a broad range of research applications. The company markets its products globally to the research community under the CELLvo™ brand name. The company also partners with leading researchers around the world to develop new applications using its proprietary technologies. www.stembiosys.com

SYNPROMICS
David Venables, Ph.D., CEO
Oct. 3 | 11:45am | Cognate Bioservices Ballroom
Edinburgh, UK
(Private)
Synpromics is the leader in gene control, improving human health by enabling safer, more effective cell and gene medicines through proprietary genomics, bioinformatics, and intelligent data-driven design. The company has developed PromPT®, its multi-dimensional bioinformatics database, that enables product-specific promoter design and selection empowering the next generation of cell and gene based medicines and bioprocessing applications. The company operates in a diverse range of fields, with focus on broad applications in cell and gene-based medicine, biologics manufacturing, and viral vector bioprocessing. Partners include Audentes, Biomarin, uniQure, AGTC, Solid Biosciences, Regeneron, Takeda, Lonza, Oxford Biomedica, and Sartorius-Stedim Cellca, as well as numerous undisclosed partners in the pharmaceutical sector. www.synpromics.com

TALARIS THERAPEUTICS
Suzanne Ildstad, M.D., Chief Scientific Officer
Oct. 2 | 4:15pm | Cognate Bioservices Ballroom
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 as well as induce durable remissions in patients with severe auto-immune and immune-mediated disorders. FCR001 is an investigational, allogeneic cell therapy that is being developed to induce or restore patients’ immune tolerance by establishing a stable, chimeric immune system comprised of both donor and recipient cells. Phase II data have shown that FCR001 can durably free a significant proportion of LDKT recipients from all chronic immunosuppression by about 12 months after their transplant, without rejection of their transplanted organ. Additionally, a subset of seven patients with an underlying autoimmune kidney disease who were able to withdraw from immunosuppression therapy had no recurrence of their disease. Talaris will initiate a Phase III trial in LDKT later this year. www.talaristx.com

TERUMO BCT
Delara Motlagh, Ph.D., VP, Cellular Therapy Technologies
Oct. 3 | 3:30pm | Cognate Bioservices Ballroom
Lakewood, CO
(Private)
Terumo BCT, a global leader in blood component, therapeutic apheresis and cellular therapy technologies, believes in the potential of blood and cells to do even more for patients than they do today. Terumo BCT’s Cell Therapy Technologies business enables researchers, developers, and manufacturers to create next-generation cell and gene therapies. We do this through flexible, automated solutions that help meet your evolving needs for reproducibility, quality, and scale through the phases of development, from translational research to current good manufacturing practice (cGMP) for commercial manufacturing. One example, the Quantum® Cell Expansion System, automates and optimizes cell culture to meet the needs of a variety of cell types in a consistent, controlled, and functionally closed environment. We are also looking further downstream in the process to provide innovative solutions, like the Finia® Fill and Finish System, to support your needs into the future. www.terumobct.com

TMUNITY THERAPEUTICS
Usman Azam, M.D., President and CEO
Oct. 2 | 2:00pm | Cognate Bioservices Ballroom
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 $156M since inception and has 54 employees. www.tmunity.com

UNIQURE
Jon Garen, Chief Business Officer
Oct. 2 | 2:45pm | BlueRock Therapeutics Ballroom
Lexington, MA
(NASDAQ: QURE)
uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We have developed a modular technology platform to rapidly bring new disease-modifying therapies to patients with severe genetic diseases. We are advancing a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in our lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Through recent collaborations and our strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, we have taken the next steps toward developing gene therapies targeting chronic and degenerative diseases that affect larger populations. www.uniqure.com

VERIGRAFT
Petter Bjorquist, Ph.D., CEO
Oct. 3 | 9:30am | Cognate Bioservices Ballroom
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_160x65

VIACYTE
Paul Laikind, Ph.D., President and CEO
Oct. 3 | 1:45pm | Cognate Bioservices Ballroom
San Diego, CA
(Private)
ViaCyte is a regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments. ViaCyte manufactures pancreatic progenitor cells from pluripotent stem cells, which are then implanted in durable and retrievable cell delivery devices. Following implant, the cells mature into human islets, including beta cells, which secrete insulin and other pancreatic hormones in response to changes in blood glucose. ViaCyte has two product candidates in clinical stage development. The PEC-Direct™ product candidate delivers the cells in a non-immunoprotective device and is being developed for highest-risk type 1 diabetes patients, while the PEC-Encap™ product candidate utilizes an immunoprotective device and is being developed for all patients with diabetes who use insulin. ViaCyte is also developing immune-evasive stem cell lines, which will further broaden the availability of cell therapy for diabetes and other potential indications. ViaCyte is funded in part by the California Institute for Regenerative Medicine and JDRF. www.viacyte.com

VIVET THERAPEUTICS
Jean-Philippe Combal, Ph.D., CEO
Oct. 3 | 3:45pm | Cognate Bioservices Ballroom
Paris, France
(Private)
Vivet Therapeutics is an emerging biotechnology company, headquartered in Paris, developing novel gene therapy treatments for rare, inherited metabolic diseases. Its RandD affiliate is located in Spain. Vivet is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies developed through its partnerships with, and exclusive licenses from, the Fundación para la Investigación Médica Aplicada (FIMA), a not-for-profit foundation at the Centro de Investigación Medica Aplicada (CIMA), University of Navarra based in Pamplona, Spain. Vivet’s lead program, VTX-801, is a novel investigational gene therapy for Wilson disease which has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) and the European Commission (EC). This rare genetic disorder is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels causing severe hepatic damage, neurologic symptoms, and potentially death. www.vivet-therapeutics.com/en

WINDMIL THERAPEUTICS
Don Hayden, Chairman and CEO
Oct. 3 | 4:30pm | Cognate Bioservices Ballroom
Baltimore, MD
(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 cellular therapeutics emanating from bone marrow, WindMIL translates novel insights in bone marrow immunology into life-saving cancer immunotherapeutics for patients. The company’s proprietary process to extract, activate, and expand these cells offers unique immunotherapeutic advantages, including broad antigen specificity, high cytotoxic potential, and long persistence. www.windmiltherapeutics.com

ZELLUNA IMMUNOTHERAPY
Miguel Forte, M.D., Ph.D., CEO
Oct. 2 | 3:00pm | Cognate Bioservices Ballroom
Oslo, Norway
(Private)
Zelluna Immunotherapy is a biopharma company developing innovative TCR-based cell therapies, both autologous and allogeneic, for the treatment of solid cancers. Zelluna’s TCRs originate from a biobank of long-term surviving patients from peptide vaccine trials, target epitopes from the known tumor associated antigens TGFβRII, hTERT and K-Ras, and have a distinct mechanism of action through the HLA class II restriction. Zelluna portfolio includes a leading autologous TCR product, ZI-H04, targeting hTERT planned to enter the clinic in the second half of 2020. At preclinical stage, Zelluna’s allogeneic adoptive cell therapy takes advantage of the proprietary platform approach of TCR-NK. Zelluna, in addition to internal competences, has a long-term agreement with the Department for Cell Therapy at Oslo University Hospital and other organizations, namely MasTherCell, ensuring comprehensive capabilities of TCR development, ranging from lead discovery to clinical translation. www.zelluna.com

By using this website you agree to accept our Privacy Policy and Terms & Conditions