Company Presenters

Slate of Presenting Companies Announced!

See the list of 30+ companies presenting at this year’s Cell & Gene Therapy Investor Day who will also be available for one-on-one partnering at the program. Collectively, these leading 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 – indicate your interest in attending the conference today!

2019 Company Presenters



4D Molecular Therapeutics
David Kirn, M.D., Chairman and CEO
Emeryville, CA
4D Molecular Therapeutics (4DMT) is focused on the discovery and development of targeted, customized and proprietary next-generation AAV gene therapy products for use in patients with severe genetic diseases with high unmet medical need. The company’s robust discovery platform, termed Therapeutic Vector Evolution, empowers 4DMT to create customized gene delivery vehicles to deliver genes specifically to any tissue or organ in the body, by optimal clinical routes of administration, at lower doses, and with resistance to pre-existing antibodies. These proprietary and targeted products allow the company to treat both rare genetic diseases and complex large market diseases. 4DMT is creating a diverse and deep product pipeline through its own internal 4D products, as well as partnered programs.


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 retinitis pigmentosa (XLRP), achromatopsia (ACHM CNGB3 & 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.


Jeff Galvin, CEO
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 four 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 Dr. Dean Felsher of the Stanford Medical School. AGT will take its HIV cure candidate into the clinic around year’s end. 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), which is expected to reach the clinic in 2019.


Birgitte Volck, M.D., Ph.D., President of Research and Development
Cambridge, MA
AVROBIO is a Phase II clinical-stage gene therapy company developing gene therapies to potentially cure rare diseases with a single dose. AVROBIO’s lentiviral-based gene therapies employ hematopoietic stem cells that are collected from the patient and then modified with a lentiviral vector to insert a functional copy of the gene that is defective in the target disease. AVROBIO is focused on the development of its gene therapy, 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. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON.

Pavan Cheruvu, M.D., CEO
New York, NY
Axovant Sciences is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases. The company’s current pipeline of gene therapy candidates targets GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases, Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. 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.

Jim McGorry, CEO
Holliston, MA
Biostage is a biotech company developing bioengineered organ implants to treat life-threatening conditions of the esophagus, trachea and bronchus. Biostage is looking to file an IND in esophageal disease in Q2 2019. Biostage is also focusing on the major unmet clinical need of esophageal atresia where 1 of 3,000 children are born with a defect where the upper and lower esophagus does not meet. Biostage’s Cellspan implant technology is intended to stimulate the regeneration of a structurally intact living biological conduit in EA patients. Preclinical studies show great promise. Additionally, Biostage was recently awarded a $1.1M SBIR NIH fast-track grant to address the unmet need in esophageal atresia. Biostage has a real opportunity to advance the field of tissue engineering, regenerative medicine and stem cell therapy by transitioning into the clinic in 2019.

Brian Culley, CEO
Alameda, CA
BioTime is a clinical-stage biotechnology company focused on the development and commercialization of novel therapies for the treatment of degenerative diseases. BioTime’s pipeline is based on two platform technologies which encompass cell replacement and cell/drug delivery. BioTime’s lead cell replacement product candidate is OpRegen®, a retinal pigment epithelium transplant therapy in Phase II development for the treatment of dry age-related macular degeneration, the leading cause of blindness in the developed world. BioTime’s lead cell delivery clinical program is Renevia®, an investigational medical device being developed as an alternative for whole adipose tissue transfer procedures. BioTime common stock is traded on the NYSE American and TASE under the symbol BTX.

Jeffrey Walsh, Chief Financial and Strategy Officer
Cambridge, MA
bluebird bio is pioneering gene therapy with purpose. The company is 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 bluebird’s labs, the company is 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. The company is putting 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, MA; Seattle, WA; Durham, NC and Zug, Switzerland.

Jeff Liter, CEO
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. B-Mogen offers a non-viral transposon-based gene delivery platform to support the T-Cell immunotherapy industry; it is the first company in the world to demonstrate genome engineering of the mitochondria genome; and the company offers fee-for-service custom cell engineering of both cell lines and, uniquely, primary human lymphohematopoietic cells for cancer research, drug screening, and antibody validation.


David Mazzo, Ph.D., President and CEO
Basking Ridge, NJ
Caladrius Biosciences is a clinical-stage biopharmaceutical company committed to the development of innovative products in cardiovascular and autoimmune disease that have the potential to restore the health of people with chronic illnesses. The company’s leadership team collectively has decades of drug development experience and globally-recognized scientific achievement. Caladrius’ goal is to build a broad portfolio of novel and versatile products that address important unmet medical needs.

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. The company is using a next-generation gene editing technology to develop a pipeline of off-the-shelf CAR-T cell therapies for the treatment of cancer. Caribou is 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.

Steve Kelly, President and CEO
Philadelphia, PA
Carisma Therapeutics leverages advances in macrophage biology, chimeric antigen receptor engineering and adoptive cellular therapy to develop disruptive immunotherapy approaches for the treatment of human disease. Carisma’s discovery and development efforts are focused on a proprietary CAR-Macrophage platform – the first technology to combine antigen recognition with the effector function of macrophages. The proposed dual mechanism of action includes the ability to directly kill targeted cells and the ability to leverage antigen-presenting cell biology to mount an adaptive immune response. Initial applications for therapeutic candidates will be in the treatment of solid tumors. Other potential applications include the disruption of protein aggregates in multiple disease states. The company anticipates initiating clinical development in 2019.

Ross Macdonald, Ph.D., CEO
Melbourne, Australia
Cynata Therapeutics Limited 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.


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 RDEB. Fibrocell is also developing FCX-013, the company’s clinical-stage candidate 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.


Julien Pham, M.D., President and Chief Operating Officer
Austin, TX
Genprex is a clinical-stage biopharmaceutical company developing novel gene therapies to improve outcomes for cancer patients receiving promising targeted and immunotherapies, as well as to expand therapeutic benefits to a greater number of patients. The company’s unique approach to cancer treatment is grounded in its proprietary gene therapy platform with broad application to utilize multiple tumor suppressor genes, provide multiple synergies with targeted drug treatments, and address a range of cancer indications. Genprex’s lead product candidate, Oncoprex™, has been developed on this cutting-edge platform and combines the features of gene therapy and immunotherapy to provide a multimodal approach to treating cancer.


Richard Pascoe, Chairman and CEO
San Diego, CA
Histogen is a regenerative medicine company developing patented technologies that stimulate the body’s stem cells to regenerate tissues and restore youthful function. The company’s lead therapeutic product has induced hair growth in multiple clinical trials, addressing a multi-billion dollar global market. Additional applications of Histogen’s technology span multiple therapeutic and aesthetic areas including skin care, orthopedics, wound healing, and dermal fillers.


Albert Seymour, Ph.D., Chief Scientific Officer
Bedford, MA
Homology Medicines is a genetic medicines company dedicated to transforming the lives of patients suffering from rare genetic diseases 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 and manufacturing capabilities position it as a leader in the development of genetic medicines.

Maria Fardis, Ph.D., President and CEO
San Carlos, CA
Iovance is commercializing tumor infiltrating lymphocyte (TIL) therapy that amplifies the body’s own immune response in order to eradicate solid tumors. Iovance is conducting multiple Phase II trials and has reported clinically meaningful responses in disease areas or populations where other approaches are not effective, notably in patients who have progressed on checkpoint inhibitors and other therapies.

Arthur Lahr, Ph.D., CEO
Amsterdam, Netherlands
(Euronext Amsterdam and Brussels: KDS)
Kiadis is developing potentially life saving therapies for patients with late-stage blood cancers, who are in need of a transplant, an area of significant need. Kiadis is building a fully integrated biopharmaceutical company to maximize the potential of ATIR, the company’s proprietary cell-based immunotherapy platform. Kiadis’ lead program, ATIR101, is a patient-specific T-cell product delivered following a haploidentical hematopoietic stem cell transplant in order to support the patient’s newly transplanted immune system. The U.S. FDA granted ATIR101 the Regenerative Medicine Advanced Therapy designation. ATIR101 has been granted Orphan Drug Designations in the U.S. and Europe.

Frederic Chereau, President and CEO
Cambridge, MA
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in 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 promotorless approach by relying on the native process of homologous recombination to drive lifelong expression. LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

Joshua Hare, M.D., Chief Scientific Officer
Miami, FL
Longeveron is a clinical-stage regenerative medicine company developing cell-based therapeutics for chronic, aging-related and life-threatening diseases with no current available treatments. The company’s primary focus is to use the basic building blocks of life, healthy living adult stem cells, to heal, repair and regenerate tissue, thereby improving and extending the quality of life of elderly individuals (enhancing healthspan) and reducing the cost burden placed on the healthcare system by treating age-related disorders. Longeveron’s lead investigational product candidate has a mechanism of action that is based on harnessing the pro-regenerative and therapeutic properties of specialized cells known as mesenchymal stem cells. Longeveron Mesenchymal Stem Cells (LMSCs) are derived from the bone marrow of healthy young adult donors. The company’s allogeneic, off-the-shelf product is manufactured as part of an FDA authorized IND and being tested in clinical investigations for chronic conditions of the elderly, and other life-threatening diseases, without current treatments.

Douglas Doerfler, President and CEO
Gaithersburg, MD
MaxCyte is a global cell-based medicines and life sciences company applying its proprietary cell engineering technology platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA™ therapies for its own pipeline. CARMA is MaxCyte’s mRNA-based proprietary therapeutic platform for rapid autologous cell therapy for the treatment of solid cancers. In addition, through its core 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 flow electroporation instruments worldwide, with all of the top ten global biopharmaceutical companies, has more than 70 partnered program licenses in cell therapy including more than 35 licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products.


Josh Muntner, Chief Financial Officer
Melbourne, Australia
Mesoblast Limited, world leader in the development and commercialization of cellular medicines, has leveraged its proprietary technology platform to establish a broad portfolio of late-stage allogeneic off-the-shelf product candidates with three product candidates in Phase III trials – acute graft versus host disease, chronic heart failure and chronic low back pain due to degenerative disc disease. Through a proprietary process, Mesoblast selects rare 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. Mesoblast has facilities in Melbourne, New York, Singapore and Texas.

Jeff Ross, Ph.D., CEO
Eden Prairie, MN
Miromatrix is a leading regenerative medicine company bioengineering advanced tissues and transplantable organs, including livers, kidneys, hearts and lungs based on its patented perfusion decellularization and recellularization technologies. Miromatrix’ ultimate mission is to save and drastically improve patients’ lives by eliminating the organ transplant waiting list. The company has also successfully developed, manufactured and commercialized the only porcine liver-derived biological products including MIROMESH® (soft tissue reinforcement) and MIRODERM® (advanced wound care) to provide unique product solutions to the market and a proven foundation for the engineering of whole organs. Miromatrix’ perfusion decellularization and recellularization technology is protected by a series of issued patents and pending patent applications in all major markets worldwide.


Kourous Rezaei, M.D., Chief Medical Officer
New York, NY
Ophthotech is a dedicated retinal disease company with unique in-house expertise and experience in retinal drug development. Ophthotech is anchored by a pipeline of gene therapy and therapeutic programs that reach across multiple retinal diseases. The company’s goal is to employ cutting-edge science to develop new therapies for retinal diseases with unmet medical need, improve patients’ lives and to create value for shareholders.


Susan Hill, Ph.D., CEO
Ambler, PA
Orbit Biomedical is a specialist medical device company, operating at the intersection of biomedical engineering, surgeon training and curative therapeutics. Orbit Biomedical’s mission is to revolutionize gene and cell therapy treatment by setting a new standard for precise, targeted surgical delivery. The company’s current focus is delivery or one-time therapies to the sub-retinal space for the treatment of retinal disease. Orbit Biomedical’s 510k approved microcannula accesses the subretinal space without the need to remove the vitreous (the gel-like substance in the centre of the eye) or to pierce the retina itself. Located in London, UK and Ambler, PA, Orbit Biomedical was founded in 2018 with Series A funding from Syncona, a leading life sciences investor.

Jason Slingsby, Ph.D., Chief Business Officer
Oxford, UK
Using Oxford BioMedica’s unique LentiVector® delivery platform, the company has created a valuable portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. Oxford BioMedica has strong partnerships with Novartis, Bioverativ (part of the Sanofi group), Immune Design, Boehringer Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium and Orchard Therapeutics, providing these partners with access to Oxford BioMedica’s intellectual property, state-of-the-art production facilities and expertise, and, in addition, the company has licensed products and technology rights to Sanofi and Axovant. These partnerships provide Oxford BioMedica with multiple income streams, consisting of upfront milestone payments, development and production fees and potential royalties on future product sales. Oxford BioMedica plans to progress the company’s wholly-owned products via spin-outs and out-licensing opportunities, while continuing to invest in the LentiVector® platform. The company plans to continue preclinical research and development to discover new potential products.

Olav Hellebo, CEO
Pencoed, UK
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, ReNeuron has therapeutic candidates in clinical development for disability as a result of stroke and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that would otherwise be unable to reach their site of action.


Gaurav Shah, M.D., President and CEO
New York, NY
Rocket Pharmaceuticals is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket’s lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Preclinical studies of additional bone marrow-derived disorders are ongoing and target Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket’s first AAV-based gene therapy program targets Danon disease, a rare neuromuscular and cardiovascular disease.

Sandy Macrae, Ph.D., CEO
Richmond, CA
Sangamo Therapeutics is focused on translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using the company’s platform technologies in genome editing, gene therapy, gene regulation and cell therapy.


Tim Lu, Ph.D., Co-Founder and CEO
San Francisco, CA
Senti Bio is the next generation therapeutics company that designs gene circuits and programs cells for tremendous therapeutic value. The company’s mission is to outsmart complex diseases with more intelligent medicines that will transform people’s lives.


Brian Miller, CEO
Lexington, MA
Sentien Biotechnologies is a privately-owned, clinical-stage company developing novel cell therapies that restore balance to the immune system and transform treatment options for patients with systemic inflammatory diseases. The company’s innovative blood conditioning approach has been proven to significantly enhance exposure and extend therapeutic activity of Mesenchymal Stromal Cells (MSCs). By immobilizing MSCs in an extracorporeal device, Sentien’s flagship SBI-101 product enables sustained, controlled dosage of secreted anti-inflammatory and regenerative properties that are unattainable by direct injection or intravenous infusion.

Rogerio Vivaldi, M.D., President and CEO
Cambridge, MA
Sigilon Therapeutics is a biopharmaceutical company creating functional cures for chronic diseases using Shielded Living Therapeutics. The Shielded Living Therapeutics product platform consists of novel engineered human cells that are encased in a proprietary immune-shielding matrix and placed in the body. These Shielded Living Therapeutics then produce therapeutic proteins in a programmable and durable fashion, without generating fibrosis or immune rejection. The company’s pipeline of products in hemophilia, lysosomal storage diseases, and, in collaboration with Eli Lilly, type 1 diabetes, are rapidly advancing toward the clinic. Sigilon was founded and created by Flagship Pioneering in conjunction with Daniel Anderson and Robert Langer of the Massachusetts Institute of Technology.

Ilan Ganot, Founder, President and CEO
Cambridge, MA
Solid Biosciences is a clinical-stage biotechnology 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. Solid is progressing programs across four scientific platforms: corrective therapies, disease-modifying therapies, disease understanding and assistive devices. Solid’s lead asset is SGT-001, a microdystrophin gene therapy currently in a Phase I/II clinical trial called IGNITE DMD.

David Venables, Ph.D., CEO
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. Through the application of PromPT the company is able to design, build and test synthetic promoters that regulate gene expression in a highly specific manner. For instance, Synpromics is able to create promoters that control expression in a particular cell type or tissue, or in response to environmental, biological or chemical stimuli.

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.

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