Company Presentations

Partnering Opportunities Abound!

The 8th Annual Partnering Forum at the Cell & Gene Meeting on the Mesa includes a pipeline loaded with partnering opportunities. Collectively, the initial slate of 60+ 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 following companies – register for the conference today!

2018 Company Presenters

blue bird bio



Carsten Thiel, Ph.D., CEO
New York, NY
Abeona Therapeutics is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. Abeona’s lead programs include EB-101 for recessive dystrophic epidermolysis bullosa, ABO-102, an adeno-associated virus (AAV)-based gene therapy for Sanfilippo syndrome type A and ABO-101, an adeno-associated virus based gene therapy for Sanfilippo syndrome type B. Abeona is also developing ABO-201 gene therapy for CLN3 disease, ABO-202 for treatment of CLN1 disease, EB-201 for epidermolysis bullosa, ABO-301 for Fanconi anemia disorder and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing a proprietary vector platform, AIM, for next generation product candidates.


Adrian Rawcliffe, Chief Financial Officer
Philadelphia, PA
Adaptimmune, a leader in T-cell therapy, has clinical trials ongoing for multiple proprietary SPEAR T-cells in solid tumor indications. The company also has a T-cell program partnered with GSK, which has already shown preliminary evidence of tumor reduction in patients with synovial sarcoma and multiple myeloma. The company’s proprietary SPEAR T-cell platform has generated a strong pipeline of affinity-enhanced T-cell therapies, with multiple investigational new drugs (INDs) open. Adaptimmune uses these therapies to harness the body’s own immune system to find and destroy diseased cells. The company’s SPEAR TCR therapies offer promise to patients that often have no other options.


Leone Patterson, Interim President, CEO and Chief Financial Officer
Menlo Park, CA
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin deficiency and Hereditary Angioedema as well as wet age-related macular degeneration. Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop and commercialize gene therapy products for ophthalmic diseases, and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development.

Shelley Hartman, CEO
Miami, FL
Aegle Therapeutics is a regenerative medicine company isolating extracellular vesicles (EV) from allogeneic bone marrow derived mesenchymal stem cells to treat severe dermatological conditions including burns, scarring and epidermolysis bullosa. In April of 2018 the U.S. Food and Drug Administration (FDA) cleared the company’s first Investigational New Drug (IND) application to begin clinical trials in burn patients. Aegle plans to file an additional IND for the treatment of EB later this year.

Markus Peters, Ph.D., Chief Commercial Officer
Lynnfield, MA
Agilis Biotherapeutics is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system.


Sue Washer, President and CEO
Alachua, FL
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 retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD) and otology. The clinical-stage XLRS and XLRP programs, the discovery program in ALD and two additional ophthalmology programs are being developed in collaboration with Biogen. 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.


David Pauza, Ph.D., Chief Scientific Officer
Rockville, MD
American Gene Technologies International (AGT) is an advanced gene and cell therapeutics company with proprietary technologies having broad applications including immuno-oncology, infectious disease and monogenic disorders. AGT has received two patents for its novel immuno-oncology approach of stimulating gamma-delta (γδ) T-cells to attack a variety of epithelial cancers. Preclinical animal studies demonstrate high efficacy in primary tumors and an abscopal effect in remote tumors. A proof of concept is expected in liver cancer (HCC) in 2020 with AGT collaborator Dean Felsher of the Stanford Medical School. AGT will take its HIV cure candidate into the clinic by the end of this year. This cure has been preliminarily confirmed during pilot runs of HIV patient blood. AGT has also developed a synthetic gene that is capable of expressing therapeutic levels of human phenylalanine hydroxylase (PAH) to cure Phenylketonuria (PKU) that is expected to reach the clinic in 2019.

Derrell Porter, M.D., SVP, Global Commercial Head
San Francisco, CA
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 most advanced T-cell immunotherapy, tabelecleucel, or tab-cel™, is in Phase III development for the treatment of patients with Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (EBV+ PTLD). Tab-cel™ is also being studied in other EBV associated hematologic and solid tumors, including nasopharyngeal carcinoma (NPC). Atara is also developing off-the-shelf, allogeneic ATA188 and autologous ATA190 T-cell immunotherapies using a complementary targeted antigen recognition technology for specific EBV antigens believed to be important for the potential treatment of multiple sclerosis (MS).

Gil Van Bokkelen, Ph.D., Chairman and CEO
Cleveland, OH
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 heavily focused on the regenerative medicine area through the development of MultiStem cell therapy, a patented, adult-derived “off-the-shelf” stem cell product. The company’s mission is to treat serious or life-threatening diseases and conditions where there is substantial unmet medical need. The company is especially focused on disease indications in the neurological, cardiovascular and inflammatory and immune disease areas, and other critical care indications. They have several ongoing clinical trials, including their most advanced program, a Phase III clinical trial for the treatment of ischemic stroke (MASTERS-2), which is being conducted under a Special Protocol Assessment from the U.S. Food and Drug Administration. This program has received Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations. Their partner, Healios KK, is also conducting a parallel registrational trial in Japan.


Nerissa Kreher, M.D., Chief Medical Officer
Cambridge, MA
AVROBIO, a leader in lentiviral-based gene therapies, is a clinical-stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose.

Jim Knight, SVP, Head of Corporate Development
Alameda, CA
BioTime is a clinical-stage biotechnology company focused on degenerative diseases. Its clinical programs are based on two platform technologies: cell replacement and cell/drug delivery. With its cell replacement platform, BioTime is producing new cells and tissues with its proprietary pluripotent cell technologies. These cells and tissues are developed to replace those that are either rendered dysfunctional or lost due to degenerative diseases or injuries. BioTime’s cell/drug delivery programs are based upon its proprietary HyStem® cell and drug delivery matrix technology. HyStem® was designed, in part, to provide for the transfer, retention and/or engraftment of cellular replacement therapies.


Jeffrey Walsh, Chief Financial and Strategy Officer
Cambridge, MA
With its lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin BB305 product candidate for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T-cell engineering. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR-T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.


Emile Nuwaysir, Ph.D., President and CEO
Cambridge, MA
Driven by a vision to liberate patients from the burden of degenerative disease, BlueRock Therapeutics is ushering in a new era of cell-based medicine that repairs the body when it cannot repair itself. Founded in 2016 through one of the largest Series A financings in biotech history, BlueRock and its team of preeminent scientists are pioneering cell therapies that replace dead, damaged or dysfunctional cells to restore critical natural functions in the body. BlueRock’s induced pluripotent stem cell (iPSC) platform can be applied to multiple diseases with great unmet need. BlueRock is initially targeting severe brain and heart conditions with the goal of altering the course of disease and drastically improving quality of life. BlueRock’s culture is defined by scientific innovation, high ethical standards and an urgency to bring transformative treatments to all.


David Hermanson, Ph.D., Director of Technical Operations
Minneapolis, MN
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. The company utilizes this unique expertise to provide three distinct services that address highly complex gene delivery and gene editing. They offer a non-viral transposon-based gene delivery platform to support the T-cell immunotherapy industry; they are the first company in the world to demonstrate genome engineering of the mitochondria genome; and they 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.


Douglas Losordo, M.D., Chief Medical Officer
Basking Ridge, NJ
Caladrius Biosciences is a clinical-stage development company with cell therapy products in development based on multiple technology platforms and targeting cardiovascular and autoimmune indications. Its late stage cardiovascular cell therapy targeting refractory angina recently received Regenerative Medicine Advanced Therapy (RMAT) designation and its critical limb ischemia program received Sakigake status in Japan.

Linda Marban, Ph.D., President and CEO
Los Angeles, CA
Capricor Therapeutics is a clinical-stage biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases. Capricor’s lead candidate, CAP-1002, is a cell-based candidate currently in clinical development for the treatment of DMD. Capricor is also exploring the potential of CAP-2003, a cell-free, extracellular vesicle-based candidate, to treat a variety of disorders.

Rachel Haurwitz, Ph.D., President and CEO
Berkeley, CA
Caribou Biosciences is a leading company in CRISPR genome editing founded by pioneers of CRISPR/Cas9 biology and using a next-generation gene editing technology to develop a pipeline of “off-the-shelf” CAR-T candidates and microbiome-based therapies. Caribou’s tools and technologies provide transformative capabilities to therapeutic development, agricultural biotechnology, industrial biotechnology and basic and applied biological research.

Steve Kelly, President and CEO
Philadelphia, PA
CARISMA Therapeutics is pioneering the development of CAR macrophages, a disruptive approach to immunotherapy. The company’s technology leverages advances in macrophage biology, chimeric antigen receptor engineering and adoptive cellular therapy for the treatment of human disease.

Ajan Reginald, Founder and CEO
Stratford-upon-Avon, UK
Celixir invents and translates novel biotechnology into best-in-class medicines to treat incurable or poorly treated diseases within the oncology, heart and musculoskeletal fields. Currently Celixir has seven products in development of which two are in Phase IIb/Phase III trials and five are in pre-Investigational New Drug (IND) development. Celixir has built a sustainable, high-output innovation engine with 92 patents for 15 novel medicines having been filed. The company also has rapid translation of IP into high value medicines through expert in-house development, as well as a high clinical trial and regulatory filing success rate.

Ram Mandalam, Ph.D., President and CEO
San Carlos, CA
Cellerant Therapeutics is a clinical-stage company developing innovative cell- and antibody-based immunotherapies for hematologic malignancies and other blood-related disorders. We have two products in development. Our lead product, romyelocel-L, is a universal cell therapy to prevent infections during prolonged neutropenia. We plan to initiate a Phase III trial for romyelocel-L in 2019, and the U.S. Food & Drug Administration (FDA) has granted romyelocel-L Regenerative Medicine Advanced Therapy (RMAT) designation. Our second product, CLT030 is an antibody-drug conjugate in preclinical development to treat AML by targeting and killing leukemic stem cells, which are believed to be the cause of relapse in patients. CLT030 is directed at the C-type lectin-like molecule-1 (CLL1), which is highly expressed on leukemic stem cells, but not on normal hematopoietic stem or progenitor cells.


Stefanos Theoharis, Ph.D., SVP, Corporate Development and Partnering
London, UK
Cell Medica is committed to improving patients’ lives through the significant therapeutic potential of cellular immunotherapy. The company’s approach is to apply innovative technologies with the aim of improving the treatment of cancer and immune reconstitution following hematopoietic stem cell transplant. Cell Medica has developed a business platform that has positioned the company as a leader in the manufacture and commercialization of cellular products. The company collaborates with leading academic institutions to turn early stage innovations into medicinal products. Cell Medica’s highly dedicated team embraces the challenges that the field of cellular therapies presents. With patient benefits at the forefront of their activities, the company has overcome the considerable challenges through collaboration and the will to succeed.

Roberto Bobadilla, Ph.D., CEO
Santiago, Chile
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 Cells for Cells’ labs with GMP standards. Duly certified specialists apply the company’s therapies.

Robert Hariri, M.D., Ph.D., Founder, Chairman and CEO
Warren, NJ
Celularity is a biotechnology company that harnesses the power of placenta-derived cells and tissue to create cancer immunotherapies and regenerative health solutions. Celularity’s intellectual property and research portfolio consists of 800+ patents as well as preclinical and clinical assets, including CAR constructs for allogeneic CAR-T/NK products, and licenses to 100+ immunotherapy assets.


Ross Macdonald, Ph.D., CEO
Melbourne, Australia
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 utilizes 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. Patient enrollment was recently completed in a Phase I clinical trial of the company’s first product in graft-versus-host disease; initial results are very promising. Cynata has a strategic partnership with Fujifilm, which is one of the company’s larger shareholders.


Flagg Flanagan, Chairman and CEO
Salt Lake City, UT
DiscGenics is a privately held, clinical-stage regenerative medicine company focused on developing cell therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. DiscGenics is harnessing the restorative potential of intervertebral disc cells to develop therapies the company believes will have a profound impact on millions suffering from the debilitating effects of back pain. DiscGenics’ first product candidate, IDCT, is a homologous, allogeneic, “off-the-shelf” injectable cell therapy for the treatment of patients with degenerative disc disease (DDD).


John Maslowski, President and CEO
Exton, PA
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase I/II clinical trial for the treatment of recessive dystrophic epidermolysis bullosa. Fibrocell is also developing FCX-013, the company’s product candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Precigen, a wholly owned subsidiary of Intrexon Corporation, a leader in synthetic biology.

Adam Muzikant, Ph.D., VP, Business Development
Burlington, MA
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). In October 2017, the U.S. Food and Drug Administration (FDA) approved the company’s first product ZILRETTA (triamcinolone acetonide extended-release injectable suspension), as the first and only extended-release, intra-articular (in the joint) injection for the management of OA knee pain. Flexion’s pipeline includes FX201, a preclinical-stage, intra-articular gene therapy being developed for pain relief and disease modification in patients with knee OA. FX201 is designed to stimulate the production of an anti-inflammatory protein, interleukin-1 receptor antagonist (IL-1Ra), whenever inflammation is present within the joint. Based on strong preclinical data, a single injection of FX201 could potentially enable expression of IL-1Ra in the joint for at least a year. Flexion held a pre-Investigational New Drug (IND) meeting with the FDA in 2018 and anticipates initiating first-in-human trials in knee OA in 2019.

Chris Loose, Ph.D., Co-Founder and CSO
Woburn, MA
Frequency Therapeutics develops small molecule drugs that activate progenitor cells within the body to restore healthy tissue. Progenitor cells are the natural sources of tissues throughout the body and remain present through adulthood. Through the transitory activation of these progenitor cells, Frequency enables disease modification without the complexity of genetic engineering. The company’s therapies use proprietary combinations of small molecule drugs to cause dormant progenitor cells to multiply and create new cells.

Nicholas Manusos, SVP, Strategy and Business Development
Madison, WI
Cellular Dynamics International (CDI), a FUJIFILM company, produces human cells for basic, translational and pharmaceutical discovery and regenerative medicine applications. The company’s iPSC-derived iCell and MyCell products provide immediate access to healthy and diseased cellular models, such as neurons, cardiomyocytes and hepatocytes, to meet small- and large-scale needs.


Hardy Kagimoto, M.D., Ph.D., CEO
Tokyo, Japan
(JPX 4593.T)
Healios is a biotechnology venture leading the field of developing iPS cell-based products for regenerative medicine. The company was founded in 2011 and listed on the stock exchange in 2015. Healios is developing a product for the treatment of age-related macular degeneration jointly with Suimitomo Dainippon Pharma under the plan of obtaining approval of its manufacture and distribution in 2020. The company has also started research and development of products capable of creating functional human organs jointly with Yokohama City University.


Adam Gridley, President and CEO
Waltham, MA
Histogenics is a leader in the development of restorative cell therapies that may offer rapid-onset pain relief and restored function. Histogenics’ lead investigational product, NeoCart, is designed to rebuild a patient’s knee cartilage to treat pain at the source and possibly prevent the progression to osteoarthritis. NeoCart is one of the most rigorously studied restorative cell therapies for orthopedic use. Histogenics completed enrollment of its NeoCart Phase III clinical trial and expects to report top-line, one-year superiority data in Q3 of this year. NeoCart is designed to perform like articular hyaline cartilage at the time of treatment, and as a result, may provide patients with more rapid pain relief and accelerated recovery as compared to the current standard of care. Histogenics’ technology platform has the potential to be used for a broad range of additional restorative cell therapy indications.


Arthur Tzianabos, Ph.D., President and CEO
Bedford, MA
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.

Maria Fardis, Ph.D., President and CEO
San Carlos, CA
Iovance Biotherapeutics is working to treat cancer by developing immuno-oncology Tumor Infiltrating Lymphocytes (TIL) therapies that harness a patient’s own immune system. Iovance is focused on the development and commercialization of autologous cellular immunotherapies optimizing personalized TIL for the treatment of solid tumors. The company’s TIL technology is designed to address the various obstacles that reduce the natural anti-tumor immune response in the body. The company is conducting four Phase II clinical trials to assess the efficacy and safety of TILs for treatment of patients with metastatic melanoma, squamous cell carcinoma of the head and heck, metastatic non-small cell lung cancer (NSCLC) and cervical cancer. Iovance is also studying TIL therapy in ovarian cancer and sarcomas. The company aims to change the cancer treatment paradigm by developing and optimizing TIL therapy to help support a patient’s own immune system in fighting various cancers.

Suma Krishnan, Founder and Chief Operating Officer
Pittsburgh, PA
Krystal Biotech has developed a proprietary gene therapy platform, the Skin TARgeted Delivery platform, or STAR-D platform, that consists of a patent pending engineered viral vector and skin-optimized gene transfer technology, to develop “off-the-shelf” treatments for dermatological diseases for which there are no known effective treatments. The company’s modified HSV-1 is a replication-deficient, non-integrating viral vector that can efficiently penetrate a broad range of skin cells. Its high payload capacity to accommodate large or multiple genes and low immunogenicity makes it a suitable choice for direct and repeat delivery to the skin.

Joshua Hare, M.D., Co-Founder and Chief Scientific Officer
Miami, FL
Longeveron is a clinical-stage cellular medicine company developing a proprietary allogeneic stem cell product. The company has four Investigational New Drugs (INDs), four actively enrolling clinical trials and a treatment registry specifically for Aging Frailty patients. The company’s primary therapeutic focus is aging-related diseases and life-threatening conditions, for which there are no available therapies. The lead investigational product is a mesenchymal stem cell being tested in a Phase IIb trial in Aging Frailty patients. Longeveron has the most advanced Aging Frailty clinical program in the world. Frailty affects approximately 12-15% of all individuals over the age of 65 and represents a substantial cost burden to countries with large elderly demographics. Longeveron has also completed its PMDA meetings in Japan and is preparing to initiate a Phase II trial in Japanese Aging Frailty patients. Longeveron has a cGMP-compliant cell processing facility where it produces its clinical supply using a proprietary manufacturing process.


Karen Aiach, CEO
Neuilly-sur-Seine, France
Lysogene is a gene therapy company focused on the treatment of lysosomal diseases and other genetic disorders of the central nervous system (CNS). The company has built a unique capability using a well-known, safe neurosurgical procedure delivering the gene therapy direct to the CNS to maximize therapeutic benefit. A pivotal Phase II-III clinical trial in MPS IIIA is expected to start in 2018, a Phase I-II clinical trial in GM1 Gangliosidosis in 2019, while the company is currently collaborating with a major partner to define the strategy of development for the treatment of Fragile X syndrome, a genetic disease related to autism.


Debra Bowes, Chief Business Officer, CARMA Cell Therapies
Gaithersburg, MD
Maxcyte is an early stage drug development company. MaxCyte has developed a novel and proprietary platform for next-generation chimeric antigen receptor (CAR)-engineered cell therapies. In addition, the company is advancing its first CAR therapy into the clinic this year.

Zandy Forbes, Ph.D., CEO
New York, NY
MeiraGTx is a vertically integrated, clinical-stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.


Donna Skerrett, M.D., Chief Medical Officer
Melbourne, Australia
Mesoblast Limited is a global leader in developing innovative cell-based medicines. Through a proprietary process, Mesoblast selects highly purified mesenchymal lineage precursor and stem cells from the bone marrow of healthy adults, and creates master cell banks which can be industrially expanded to produce thousands of doses from each donor that meet stringent release criteria, have lot-to-lot consistency, and can be used “off-the-shelf” without the need for tissue matching. The company has leveraged its proprietary technology platform to establish a broad portfolio of late stage product candidates. Mesoblast’s allogeneic, “off-the-shelf” cell product candidates are being evaluated in their ability to target advanced stages of diseases with high, unmet medical needs including cardiovascular conditions, orthopedic disorders, immunologic and inflammatory disorders and oncologic/hematologic conditions.

Gloria Matthews, Ph.D., SVP
Marietta, GA
MiMedx is a biopharmaceutical company developing and marketing regenerative biologics utilizing human placental tissue allografts and patent protected processes for multiple sectors of healthcare.

Jeff Ross, Ph.D., CEO
Eden Prairie, MN
Miromatrix is a leading regenerative medicine company bioengineering transplantable organs including livers, kidneys, hearts and lungs based on its patented perfusion decellularization and recellularization technology to address the immense need for transplantable whole organs. In addition to whole organ development, Miromatrix has successfully developed, manufactured and commercialized a series of whole organ-derived biological products. MIROMESH is the only liver-derived biologic mesh for soft tissue reinforcement, and MIRODERM is the only liver-derived wound care product. Miromatrix is utilizing the expertise and knowledge gained through the commercialization of the company’s acellular products, with their recellularization technology, to fully address the transplantable organ shortage and provide new options for thousands of patients.


Luca Alberici, Ph.D., Chief Business Officer
Milan, Italy
MolMed S.p.A. is a medical biotechnology company focused on research, development and clinical validation of novel cell and gene therapy products. MolMed’s pipeline main products are Zalmoxis (TK), a cell-based therapy in Phase III for high-risk acute leukemia and CAR-CD44v6, an immune gene therapy project potentially effective for many hematological malignancies and several epithelial tumors currently in preclinical development. MolMed also offers top-level expertise in cell and gene therapy to third parties to develop and validate projects from preclinical to Phase III trials, including scale-up and cGMP manufacturing of retroviral and lentiviral vectors and patient-specific genetically engineered CD34+ or T-cells. MolMed is headquartered in Milan and has two GMP manufacturing facilities in the Milan area. MolMed has been listed on the Milan Stock Exchange since 2008.

Colin Kealey, M.D., Chief Medical Officer
Los Angeles, CA
MonarchBio is a life sciences company focused on commercializing next generation cell therapy and regenerative medicine products based on its Thin Film Nitinol (TFN) technology platform. MonarchBio’s lead product, developed in collaboration with the Fred Hutchinson Cancer Research Center, is a unique platform for local delivery of cell therapies treating solid tumors. The so-called Engineered Lymph Node (ELN) uses a bioactive thin film mesh to deliver cell therapies directly to the site of the tumor. MonarchBio believes that this approach solves some of the key challenges for cell therapies in solid tumors by delivering ultra high cell densities directly to the site of the tumor, and then providing a stimulatory microenvironment to encourage proliferation of the engineered cells and sustained killing of the tumor. Pre-clinical testing of this approach at the Fred Hutchinson Cancer Research Center has demonstrated significant efficacy advantages in five different animal models of solid tumors.

Many Litchman, M.D., CEO
New York, NY
Mustang Bio, a Fortress Biotech company, is a clinical-stage biopharmaceutical company focused on the development and commercialization of novel cancer immunotherapy products designed to leverage the patient’s own immune system to eliminate cancer cells. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development and to out-license or bring the technologies to market. Mustang has partnered with the City of Hope National Medical Center and the Fred Hutchinson Cancer Research Center to develop proprietary chimeric antigen receptor engineered T-cell (CAR-T) therapies across many cancers, and with Harvard Medical School’s Beth Israel Deaconess Medical Center and the Harvard Stem Cell Institute for the development of CRISPR/Cas9-enhanced CAR-T therapies in hematologic malignancies and solid tumors.

David Fellows, CEO
London, UK and Waltham, MA
Nightstar is a leading clinical-stage gene therapy company focused on novel one-time treatments for patients suffering from rare inherited retinal diseases. Nightstar’s lead product candidate, NSR-REP1, an AAV2 vector designed to produce REP1 inside the eye, is currently in Phase III development for the treatment of patients with choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people. Positive results from Phase I/II trials of NSR-REP1 were published in 2014 and 2016. In data from 32 patients treated with NSR-REP1 across four open-label Phase I/II clinical trials, over 90% of treated patients maintained or improved their visual acuity over a two year follow-up period. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a clinical trial known as the XIRIUS trial for the treatment of patients with X-linked retinitis pigmentosa.

Katie Fanning, CEO
Seattle, WA
Nohla Therapeutics is a leading developer of “off-the-shelf” cell therapies for patients with cancer and other critical diseases. Nohla’s proprietary notch ligand technology platform serves as the foundation for its ongoing clinical, preclinical and discovery programs. The company’s lead product candidate, dilanubicel, is currently being evaluated in two ongoing Phase II trials for patients with hematologic malignancies undergoing a myeloablative allogeneic transplant, and acute myeloid leukemia (AML) patients with chemotherapy-induced myelosuppression following high-dose chemotherapy. Nohla is also pursuing multiple preclinical and discovery programs in the areas of immune tolerance and other diseases.


Larry Couture, Ph.D., CEO
Galway, Ireland
Orbsen Therapeutics is a clinical-stage stromal cell immunotherapy company based in Galway, Ireland. The company’s proprietary GMP-compliant and scalable positive-selection purification strategies provide for a well characterized stromal cell population. Stromal cell immunotherapy holds great promise as an alternative to drugs and surgical procedures for treating a wide range of medical conditions including acute inflammatory disease, arterial disease of the limbs, diabetes complications, arthritis and other inflammatory conditions. The therapeutic potential is linked to these cells’ natural capacity to modulate the immune system, limit inflammation and promote healing and regeneration of damaged tissues. The cells can be selected at high yield to near 100% purity from umbilical cord, bone marrow or adipose from single or pooled donors, and expanded and frozen to generate significant numbers of “off-the-shelf” allogeneic products. Clinical trials in patients with diabetic foot ulcers, autoimmune liver disease, diabetic kidney disease and acute respiratory distress syndrome are underway.


Mark Rothera, President and CEO
London, UK
Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to transforming the lives of patients with rare disorders through innovative gene therapies. The company’s programs will use the potential of ex-vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in severe and life-threatening inherited disorders. The company works in partnership with the world’s leading research centers to harness the life-giving potential of gene therapy. Orchard’s mission is to transform the lives of patients with rare disorders through innovative gene therapies. The company’s vision is to become the leader in gene therapy for primary immune deficiencies and inherited metabolic disorders.

Taylor Crouch, President and CEO
San Diego, CA
Organovo is a biotech platform company that has developed a leadership position with its revolutionary ability to 3-D bioprint tissues with human functionality. The 3-D bioprinting platform enables the creation of durable, multi‐cellular tissues with complex architecture that replicates key aspects of native biology to gain a better understanding of disease processes, the discovery of novel therapeutics and the safety assessment of drugs in a disease‐relevant background. Organovo has the potential to provide customers with what is essentially a lab to human translation, which could greatly improve the effectiveness of drug research. Organovo is also pursuing multiple Investigational New Drug (IND)-track programs to develop its NovoTissues transplantable tissues to address a number of serious unmet medical needs in adult and pediatric populations, initially focusing on liver disease.

Eric Halioua, President and CEO
Liege, Belgium
Founded in 2014 as a spin-off of the French Blood Bank (EFS), PDC*line Pharma is a Belgian biotech company that develops an innovative class of active immunotherapies for cancers, based on an allogeneic antigen presenting cell line (PDC*line). PDC*line is a new, potent and scalable therapeutic cancer vaccine based on a proprietary allogeneic cell line of Plasmacytoid Dendritic Cells. PDC*line is much more potent to prime and boost antitumor antigen including neoantigens, specific cytotoxic T-cells than conventional vaccines and improves the response to checkpoint inhibitors. The technology can be applied for any type of cancer.


Michael Dombeck, VP, Business Development
Durham, NC
Precision BioSciences is a biotechnology company dedicated to improving life with its next generation gene editing technology, ARCUS, a proprietary, wholly-owned non-CRISPR editing platform with applications in gene therapy, cell therapy and agriculture. Precision’s allogeneic cell therapy pipeline includes a broad collection of engineered T-cells, with the company’s lead CD19 CAR-T on schedule to initiate clinical testing by 2019. Precision’s gene therapy pipeline includes multiple in-vivo liver, eye and central nervous system gene editing therapeutic candidates, based on the ability of ARCUS nucleases to efficiently edit desired target sites in-vivo, with systemic therapeutic efficacy demonstrated in non-human primates. The value of Precision’s ARCUS editing platform is enhanced by the company’s extensive patent portfolio, established freedom to operate and significant partnerships with industry leaders, positioning Precision to lead the development and commercialization of advanced gene editing products that solve important problems in oncology, genetic disease, agriculture and beyond.

Jane Lebkowski, Ph.D., President, Research and Development
Menlo Park, CA
Regenerative Patch Technologies (RPT) is a company developing a stem cell-based implant technology for the treatment of retinal diseases. CPCB-RPE1 is RPT’s lead product which is in clinical development in patients with geographic atrophy. CPCB-RPE1 consists of pluripotent stem cell derived polarized retinal-pigmented epithelial cells on an ultrathin parylene membrane that mimics the diffusion characteristics of the Bruch’s membrane. The technology to produce the CPCB-RPE1 implant is exclusively licensed to RPT from the University of Southern California, the California Institute of Technology and UC Santa Barbara.

Suzanne Ildstad, M.D., CEO
Louisville, KY
Regenerex is an innovative, late clinical-stage cellular therapy company developing its FCRx platform technology with the goal of eliminating the burden of chronic immunosuppression in select organ transplant and severe autoimmune conditions. In an ongoing Phase II study, the company’s proprietary allogeneic cell therapy product, FCR001, has been shown to induce tolerance in recipients of solid organ transplants from unmatched and unrelated donors, allowing living donor kidney transplant recipients to wean completely off immunosuppression one year after transplant. FCR001 Phase II subjects achieved these results utilizing low myeloablative conditioning that minimize toxicities typically associated with a standard Hematopoietic Stem Cells (HSC) transplant. The U.S. Food and Drug Administration (FDA) has granted Regenerex clearance to proceed with a pivotal Phase III study in Living Donor Kidney Transplant. The FDA has also granted FCR001 Orphan Drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation.


Olav Hellebo, CEO
Pencoed, South Wales
ReNeuron is a leading, clinical-stage cell therapy development company and 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.

Sandy Macrae, Ph.D., CEO
Richmond, CA
Sangamo Therapeutics is focused on translating groundbreaking science into genomic therapies that transform patients’ lives using the company’s platform technologies in genome editing, gene therapy, gene regulation and cell therapy.


BG Rhee, Ph.D., CEO
Incheon, Republic of Korea
SCM Lifescience is a research-based biopharmaceutical company focused on developing next-generation clonal mesenchymal stem cell therapeutics using its proprietary technology platform, Subfractionation Culturing Method (SCM). With over a decade of research and development, the company has obtained intellectual property protection for its high-purity stem cell isolation and manufacturing technologies in major markets including Korea, the U.S., Europe, China and Japan. The company’s products are allogeneic, bone marrow-derived clonal mesenchymal stem cells (cMSCs) targeting various inflammatory immune diseases. The company’s current clinical pipeline includes chronic and acute steroid refractory graft-versus-host diseases, acute pancreatitis, atopic dermatitis and liver cirrhosis. In order to meet the needs of patients, the company’s products are manufactured as fresh and frozen formulations – fresh for chronic and frozen for acute patients. Currently, SCM Lifescience is running four clinical trials with two additional trials scheduled to initiate by 2019. In addition to the company’s pipeline, SCM Lifescience is open to innovative collaborations in the field of regenerative medicine.

Felicia Pagliuca, Ph.D., VP, Cell Biology Research and Development
Cambridge, MA
Semma Therapeutics is pioneering the new treatment paradigm of using cells as medicine. Semma’s new class of regenerative medicine therapies couples its breakthrough stem cell technologies with proprietary delivery systems designed to protect cells from the immune system. Ongoing research at Semma Therapeutics is focused on combining these highly-differentiated active cells with a state-of-the-art device to provide a functional cure for patients with diabetes. Semma Therapeutics is working to bring new therapeutic options to the clinic and improve the lives of patients. The company is headquartered in Cambridge, MA and has operations in Providence, RI.

Brian Miller, CEO
Lexington, MA
Sentien is a clinical-stage, venture-backed company developing cell-based treatments for systemic inflammatory disorders. The company’s lead product, SBI-101, utilizes mesenchymal stromal cells (MSCs) in a hollow fiber device to provide controlled, sustained exposure of the MSC secreted factors to patient’s blood. Sentien is currently enrolling in a Phase I/II clinical trial for the treatment of dialysis dependent acute kidney injury with additional preclinical programs in the acute, systemic inflammatory space.


Devyn Smith, Ph.D., Chief Strategy Officer and Head of Operations
Cambridge, MA
The Afibromer biomaterials of Sigilon’s platform avoid the fibrotic response, allowing engineered cells implanted in the body to deliver therapeutic molecules for extended periods of time, acting as living therapeutics. These engineered cells can either deliver molecules at a constant rate or be programmed using the techniques of synthetic biology to respond to changing conditions, such as intermittent flare ups of disease symptoms or fluctuating blood levels of specific factors like glucose. Synthetic biology tools and programmable genetic circuits create new possibilities for the treatment of complex diseases, and can be programmed to sense changing conditions and respond appropriately. This approach is unlike gene therapies or RNA interference, which cannot be adjusted once they are introduced into the body.

Jennifer Ziolkowski, Chief Financial Officer
Cambridge, MA
Solid Biosciences is a life science company focused solely on finding meaningful therapies for Duchenne muscular dystrophy (DMD). Founded by those touched by the disease, Solid is a center of excellence for DMD, bringing together experts in science, technology and care to drive forward a portfolio of candidates that have life-changing potential. Currently, Solid is progressing programs across four scientific platforms: Corrective Therapies, Disease-Modifying Therapies, Disease Understanding and Assistive Devices.


Robert Hutchens, President and CEO
San Antonio, TX
StemBioSys is focused on enhancing the growth of adult stem cells for research, therapeutic or drug discovery applications. The company’s patented technology platforms overcome obstacles to creating clinically useful stem cell therapies. The company’s technologies represent the next evolution in cell research and are branded to the research market under the CELLvo label. The centerpiece of these enabling technologies is the company’s CELLvo Matrix. This cell-derived 3-D microenvironment allows a variety of stem cell types to replicate more rapidly, maintain a small cell size and express markers indicative of retained stem cell potency beyond that seen with traditional tissue culture substrates. The company recently announced a Xeno-free version of the company’s technology. StemBioSys also has several cell products for the research market, and all cell products are isolated and expanded on the company’s CELLvo Matrix.

Sarah Haecker Meeks, Ph.D., VP, Business Development
Edinburgh, UK
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, including broad applications in cell- and gene-based medicine, biologics manufacturing and viral vector bioprocessing. Synpromics designs and develops highly specific promoters to drive gene expression at the desired level and specificity in any cell type, tissue or environmental condition. Current partners include Audentes, BioMarin, uniQure, AGTC, Solid Biosciences, Homology Medicines, Adverum, GE Healthcare and Sartorius-Stedim Cellca, as well as numerous undisclosed partners in the pharmaceutical sector.


Antoinette Gawin, EVP, Global Commercial
Lakewood, CO
As a global leader in blood component, therapeutic apheresis, cellular and autologous biologic technologies, Terumo BCT believes in the potential of cells to do even more for patients than they do currently. As innovators for over 40 years in cell separation, the company is committed to providing autologous biologic technologies that deliver consistently reproducible results that clinicians and healthcare organizations trust. Through Terumo’s service and support, the company enables physicians to use cutting-edge technologies, increase patient access to autologous therapies and continue to advance next-generation technology.


Stéphane Boissel, CEO
Sophia-Antipolis, France
TxCell is a biotechnology company that develops platforms for innovative, personalized T-cell immunotherapies for the treatment of severe inflammatory and autoimmune diseases with high unmet medical need. TxCell is targeting transplantation as well as a range of autoimmune diseases (both T-cell and B-cell-mediated), such as multiple sclerosis, rheumatoid arthritis, inflammatory bowel diseases or inflammatory skin diseases. TxCell’s cellular immunotherapies are based on regulatory T lymphocytes (Tregs). Tregs are a T-cell population for which anti-inflammatory properties have been demonstrated. Contrary to conventional approaches based on non-specific polyclonal Tregs, TxCell is exclusively developing engineered antigen-specific Tregs, where the antigen specificity is brought by a Chimeric Antigen Receptor.

Daniel Gau, Ph.D., Chief Operating Officer
Oberdorf, Switzerland
Unicyte is a leading expert on nano-extracellular vesicles and human liver stem cells with focus on kidney and liver disorders, diabetes and oncology. Unicyte builds on a 15 year public-private partnership with Professor Camussi at the University of Torino, Italy and a very strong patent estate with 15 patent families and more than 130 granted patents. The company has built a broad therapeutic pipeline for internal development and licensing. Unicyte currently runs nine preclinical programs using extracellular vesicles, human liver stem cells or derived organoids. Unicyte is an independent affiliate of Fresenius Medical Care, the world’s largest provider of products and services for individuals with renal diseases.

Jon Garen, Chief Business Officer
Lexington, MA
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 have 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.


Nick Colangelo, President and CEO
Cambridge, MA
Vericel is an autologous cell therapy company marketing autologous cell therapy products in the U.S. MACI is the company’s product for cartilage repair and has a market of more than $700M per year. The company also markets Epicel for skin repair for burn victims. The Epicel market is approximately a $100M annually. Vericel’s first quarter revenues of $18.1M represent a 55% year over year increase.


Paul Laikind, Ph.D., President and CEO
San Diego, CA
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte has two products in clinical development. The PEC-Direct product candidate delivers stem cell-derived PEC-01 pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic liability and/or recurrent severe hypoglycemic episodes. The PEC-Encap (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is funded in part by the California Institute for Regenerative Medicine and JDRF.

Miguel Forte, M.D., Ph.D., CEO
Oslo, Norway
Zelluna Immunotherapy is developing innovative T-cell receptor (TCR)-based cancer therapies for patients with a high unmet medical need. The company’s core asset and unique competitive advantage is a large portfolio of TCRs isolated from long-term surviving patients from peptide vaccine trials. The company’s TCRs are directed against epitopes from the known tumor associated antigens TGFβRII, hTERT and K-Ras and have the potential to be used in the treatment of a broad range of solid tumors. Zelluna has a long-term collaborative research and development agreement (CRADA) with the Department of Cell Therapy at the Oslo University Hospital and is in the process of setting up cell manufacturing capabilities in collaboration with MaSTherCell. Zelluna aims to start its first clinical trial in 2019-2020.