Company Presentations

Partnering Opportunities Abound!

See the list of 60+ leading cell and gene therapy companies who have been selected to give presentations at the 2016 Partnering Forum! Representatives from these companies will also be available for one-on-one partnering opportunities throughout the conference.

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2016 Company Presenters

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Elizabeth Alcamo, Ph.D., Senior Director, Corporate Development
Oct. 5 | 1:30pm | La Jolla Ballroom 2
Emeryville, CA
4D Molecular Therapeutics (4DMT) is focused on the discovery and development of targeted and proprietary adeno-associated virus (AAV) gene therapy vectors and therapeutic products. The company’s robust discovery platform, termed Therapeutic Vector Evolution, empowers 4DMT to create customized gene delivery vehicles to deliver genes to any tissue or organ in the body, by optimal clinical routes of administration and with antibody evasion. 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 partnerships, while progressing internal products toward clinical trials in parallel. 4DMT partners include: Pfizer, Roche, uniQure, AGTC and Benitec.


Tim Miller, Ph.D., President and CEO
Oct. 5 | 11:15am | La Jolla Ballroom 2
Dallas, TX
Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on delivering gene therapy and plasma-based products for severe and life-threatening rare diseases. Abeona’s lead programs are ABO-101 (AAV-NAGLU) and ABO-102 (AAV-SGSH), adeno-associated virus (AAV)-based gene therapies for Sanfilippo syndrome (MPS IIIB and IIIA). The company is also developing ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease and ABO-301 (AAV-FANCC) for Fanconi anemia disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing plasma protein therapies including SDF Alpha (alpha-1 protease inhibitor) for inherited COPD using its proprietary SDF (Salt Diafiltration) ethanol-free process.


Amber Salzman, Ph.D., President and Chief Operating Officer
Oct. 6 | 2:30pm | La Jolla Ballroom 1
Menlo Park, CA
Adverum is a gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline and is leveraging its next generation adeno-associated virus (AAV)-based directed evolution platform to generate product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum’s focus on the patient is supported by clinical development expertise and core capabilities in vector optimization, process development, manufacturing and assay development.


Sue Washer, President and CEO
Oct. 5 | 2:45pm | La Jolla Ballroom 1
Alachua, FL
AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products designed to transform the lives of patients with severe diseases, with an initial focus in ophthalmology. AGTC’s lead product candidates are designed to treat inherited orphan diseases of the eye, caused by mutations in single genes that significantly affect visual function and currently lack effective medical treatments. AGTC’s product pipeline includes six named ophthalmology development programs across five targets: X-linked retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), achromatopsia, wet age-related macular degeneration and blue cone monochromacy; and two non-ophthalmology programs: alpha-1 antitrypsin deficiency and adrenoleukodystrophy. AGTC is also continuing to develop early research studies in additional indications. The company is exploring genetic defects in cells in the inner ear that lead to deafness and expects to advance several product candidates into development within the next few years.


Alex Dusek, Head of Commercial Strategy
Oct. 6 | 4:30pm | La Jolla Ballroom 1
Durham, NC
Argos Therapeutics is a company focused on developing personalized immunotherapy products for cancer and infectious disease. The company’s lead program is rocapuldencel-T (AGS-003) which completed enrollment to a Phase III study called ADAPT in front-line mRCC in October 2015 with final data expected in the first half of 2017. This study is under a Special Protocol Assessment with the U.S. Food and Drug Administration (FDA). Argos expects to obtain FDA approval through a Biologics License Application submission based upon results of the Phase III ADAPT study in the first half of 2018.


Matt Patterson, President and CEO
Oct. 5 | 3:15pm | La Jolla Ballroom 1
San Francisco, CA
Audentes Therapeutics is a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening diseases. The company has four products in development, AT132 for the treatment of X-Linked Myotubular Myopathy (XLMTM), AT342 for the treatment of Crigler-Najjar Syndrome, AT982 for the treatment of Pompe disease and AT307 for the treatment of the CASQ2 subtype of Catecholaminergic Polymorphic Ventricular Tachycardia (CASQ2-CPVT). Audentes is home to a focused, experienced and passionate team committed to forging strong, global relationships with the patient, research and medical communities.


Andrew Quick, SVP, Clinical Development
Oct. 6 | 3:30pm | La Jolla Ballroom 2
Cambridge, UK
Avita Medical is a regenerative medicine company specializing in the treatment of wounds and skin defects. Its patented and proprietary collection and application technology provides innovative treatment solutions derived from a patient’s own skin. The company’s lead product, ReCell, is used in the treatment of a wide variety of burns, plastic, reconstructive and cosmetic procedures. ReCell is patented, CE‐marked for Europe, TGA‐registered in Australia and CFDA‐cleared in China. In the U.S., ReCell is an investigational device limited by federal law to investigational use and is being evaluated in a pivotal trial in burns. Enrollment in the pivotal study was completed in January 2016 and submission of a premarket approval application is expected in Q1 2017.


Saverio La Frencesca, M.D., Executive Vice President and Chief Medical Officer
Oct. 6 | 3:15pm | La Jolla Ballroom 2
Boston, MA
Biostage is a regenerative medicine company focused on esophageal cancer, central lung cancer and complications of the trachea. The company’s novel Cellframe technology is engineered to stimulate the body’s signaling pathways and natural healing process to regenerate and restore organ function. Biostage’s Cellframe technology is based on over 20 years of scientific progress in the fields of tissue engineering, cell biology and material science. Cellframe technology combines the best attributes of a synthetic scaffold with tissue engineering and cell biology to create what may be a revolutionary method of addressing organ damage.


Jeffrey Walsh, Chief Financial and Strategy Officer
Oct. 6 | 1:15pm | La Jolla Ballroom 1
Cambridge, MA
bluebird bio has built an integrated product platform of lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities with broad potential application to severe genetic diseases and cancer. bluebird bio is studying its gene therapy programs in patients with cerebral adrenoleukodystrophy, transfusion-dependent ß-thalassemia and severe sickle cell disease. bluebird bio’s lead CAR T oncology program is being studied in patients with relapsed/refractory multiple myeloma.


David Mazzo, Ph.D., CEO
Oct. 5 | 10:30am | La Jolla Ballroom 2
Basking Ridge, NJ
Caladrius Biosciences, through its subsidiary, PCT, is a leading development and manufacturing partner to the cell therapy industry. PCT works with its clients to overcome the fundamental challenges of cell therapy manufacturing by providing a wide range of innovative services including product and process development, GMP manufacturing, engineering and automation, cell and tissue processing, logistics, storage and distribution, as well as expert consulting and regulatory support. PCT and Hitachi Chemical Co. have entered into a strategic global collaboration to accelerate the creation of a global commercial cell therapy development and manufacturing enterprise with deep engineering expertise. Around the core expertise of PCT, Caladrius strategically develops select product candidates, which currently includes an innovative therapy for type 1 diabetes based on a proprietary platform technology for immunomodulation.


Jeffrey Bartlett, Ph.D., Chief Scientific Officer
Oct. 6 | 2:15pm | La Jolla Ballroom 2
Tucson, AZ
Calimmune is a clinical-stage, international biotechnology company focused on developing novel gene therapies that have the potential to improve and protect the lives of patients by enhancing their immune system. The company was founded in 2006 from technology developed in the labs of Nobel Laureate David Baltimore, California Institute of Technology; Irvin Chen, University of California, Los Angeles; and Inder Verma, Salk Institute for Biological Studies. Calimmune’s lead product candidate for HIV, now in Phase I/II studies, is being evaluated as a one-time treatment to prevent HIV progression to AIDS. The company has labs and branch offices in Pasadena, CA and Sydney, Australia.


Linda Marbán, Ph.D., CEO
Oct. 5 | 11:45am | La Jolla Ballroom 1
Beverly Hills, CA
Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class therapeutics. Capricor has two product candidates under clinical investigation: CAP-1002, a cardiac cell therapy, and Cenderitide, a dual natriuretic peptide receptor agonist. CAP-1002 is in development for the treatment of post myocardial infarction, advanced heart failure and Duchenne muscular dystrophy-associated cardiomyopathy. Cenderitide is in development for the outpatient treatment of heart failure as well as other potential indications. In addition, Capricor is evaluating its exosomes platform technology for cardiac diseases and other therapeutic areas.


Maroun Khoury, Ph.D. Chief Scientific Officer
Oct. 5 | 5:30pm | La Jolla Ballroom 2
Santiago, Chile
Cells for Cells (C4C) is a Chilean biotech company engaged in research and development and commercialization of innovative cell therapies and technologies based on adult stem cells. The company was established in 2010 as a start-up of the Medicine School of Universidad de los Andes, Santiago, Chile. C4C’s innovative team performs novel preclinical and clinical studies using different mesenchymal stem cell (MSC) sources and/or their secreted micromolecules, targeting specific diseases. C4C’s purpose is to generate a novel and proprietary portfolio of processes, technologies and cell therapies to confront degenerative diseases. Overall, C4C’s regenerative medicine joint program with Universidad de los Andes has raised $35 million for research and development from private parties and government. The company is looking for investors, partners and collaborators who can contribute to further fund, complement, scale-up or internationalize C4C’s portfolio.


Emile Nuwaysir, Ph.D., President and Chief Operating Officer
Oct. 6 | 9:30am | La Jolla Ballroom 2
Madison, WI
Cellular Dynamics International (CDI), a FUJIFILM company, is a leading developer and manufacturer of human cells used in drug discovery, toxicity testing, stem cell banking and cell therapy development. The company partners with innovators worldwide to combine biologically relevant human cells with the newest technologies to drive advancements in medicine. CDI’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 any cell type in the human body. The company’s 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. Founded in 2004 by Dr. James Thomson, a pioneer in human pluripotent stem cell research, Cellular Dynamics is based in Madison, WI, with a second facility in Novato, CA.


Christian Homsy, M.D., CEO
Oct. 5 | 11:15am | La Jolla Ballroom 1
Mont-Saint-Guibert, Belgium
Celyad is a clinical-stage biopharmaceutical company focused on the identification, development and commercialization of specialized cell-based therapies. Celyad has a portfolio of product candidates in immuno-oncology seeking to address cancers with high unmet medical needs. Founded in 2007, Celyad is using its unique know-how in cell engineering, as well as the manufacturing and logistical capability for complex products to develop product candidates from bench to the clinic. Celyad builds its business model on partnering with prominent research institutions such as the Mayo Clinic, Dartmouth College and Institute Curie to develop innovative and breakthrough research into potential commercial applications.


Tom Isett, CEO
Oct. 5 | 2:45pm | La Jolla Ballroom 2
Sparks, MD
Commence Bio is founded on the vision that cancer and inflammatory diseases will be optimally treated with new cellular immunotherapies created using proprietary Stimulated Toll-like Receptor Technology (STaRT). STaRT is used to program adult mesenchymal stem cells (MSCs) to create two new therapeutic platforms: an anti-tumor MSC1 and an anti-inflammatory MSC2 phenotype. These breakthrough cell therapies have proven to be safe and effective in seven preclinical models of disease, including ovarian cancer, demyelinating diseases, rheumatoid arthritis, acute lung injury/ARDS, Crohn’s disease and diabetic neuropathic pain. The company’s lead indication is CMB-200, an MSC2 therapy for acute optic neuritis and multiple sclerosis to be evaluated in a multicenter Phase I/II clinical trial planned for 2017.


Mark Sawicki, Ph.D., President and Chief Commercial Officer
Oct. 5 | 5:00pm | La Jolla Ballroom 2
Irvine, CA
Cryoport is the leader in cold chain logistics supporting the regenerative therapy field and is the only partner with the capability of consolidating all chain of condition, chain of custody, validation and regulatory documentation in a single searchable data stream. The company is currently supporting more than 67 clinical programs (including 12 Phase III programs) in the regenerative therapy space. As a premier logistics company serving life sciences, Cryoport forms an essential logistics backbone for research, development, manufacturing and trade; ensuring that life sciences materials are stored and delivered, reliably and efficiently. Cryoport provides 24/7/365 live logistics and monitoring services with specialized knowledge in the shipment of life sciences material requiring cryogenic temperatures.


Timothy Schroeder, Founder and CEO
Oct. 5 | 4:30pm | La Jolla Ballroom 2
Cincinnati, OH
CTI Clinical Trial & Consulting Services is a global, privately-held, full service contract research organization, delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides pharmaceutical, biotechnology and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine/gene therapy, immunology, transplantation, nephrology, hematology/oncology, neurology, infectious diseases, hepatology, cardiopulmonary and pediatric populations. CTI also offers a fully integrated multispecialty clinical research site that conducts Phase I-IV trials. CTI has a passion for helping life-changing therapies succeed in chronically and critically ill patient populations. With clinical trial experience across six continents, CTI partners with research sites, patients and sponsors to fulfill unmet medical needs. CTI has operations across North America, Europe, Latin America and Asia-Pacific.


Ross Macdonald, Ph.D., CEO
Oct. 6 | 10:45am | La Jolla Ballroom 2
Melbourne, Australia
Cynata Therapeutics is an Australian stem cell and regenerative medicine company that is developing a therapeutic mesenchymal stem cell (MSC) platform technology, Cymerus, using discoveries made by Professor James Thompson’s group at the University of Wisconsin-Madison (UWM). Cynata’s proprietary technology utilizes iPSCs originating from an adult donor as the starting material for manufacturing the allogenic MSC therapeutic product. Cynata is the only company able to manufacture consistent, robust therapeutic MSCs without reliance upon multiple stem cell donors. The company currently plans to commence a Phase I clinical trial in GvHD in the UK during the second half of 2016. Cynata recently announced a partnership with German company Apceth to investigate genetically engineered Cymerus MSCs in cancer.


Andy Hu, M.D., Head of Business Development
Oct. 6 | 2:00pm | La Jolla Ballroom 1
Cambridge, MA
Dimension Therapeutics is the leader in discovering and developing new therapeutic products for people living with devastating rare diseases associated with the liver, based on the most advanced, mammalian adeno-associated virus (AAV) gene delivery technology. Dimension is actively progressing its broad pipeline, which features programs addressing unmet needs for patients suffering from inherited metabolic diseases, including OTC deficiency, GSDIa, citrullinemia type 1, PKU and Wilson disease, as well as a collaboration with Bayer in hemophilia A, and a wholly-owned clinical program in hemophilia B. The company targets diseases with readily identifiable patient populations, highly predictive preclinical models and well-described and often clinically validated, biomarkers.


Robert Wynalek, COO
Oct. 6 | 3:45pm | La Jolla Ballroom 2
Salt Lake City, UT
DiscGenics is a biotechnology company developing advanced spinal therapeutics to treat patients with diseases of the intervertebral disc. DiscGenics is the only company to utilize therapeutic progenitor cells derived from the intervertebral disc to treat the disc. The company’s allogeneic methodology allows DiscGenics to derive many doses from each adult tissue donation. The company’s first product, Injectable Discogenic Cell Therapy (IDCT), is in preclinical development for the treatment of moderate degenerative disc disease. This cellular treatment offers a cost effective and non-surgical solution to patients that currently have few treatment options. Furthermore, the treatment may delay the progression towards costly and often ineffective surgical interventions necessary at the late stages of degeneration. Follow-on products utilizing the same cell population with different scaffold carriers are being explored for additional disc-related indications.


Deborah Palestrant, Ph.D., Senior Director, Business Development and Strategy
Oct. 6 | 1:30pm | La Jolla Ballroom 1
Cambridge, MA
Editas Medicine is a leading genome editing company dedicated to treating patients with genetically defined diseases by correcting their disease-causing genes. While genetic defects are now recognized as the cause of many diseases, the vast majority of these diseases lack effective treatments. Editas’ product development strategy is to target genetically defined diseases with an initial focus on debilitating illnesses where there are no approved treatments and where the genetic basis of disease is well understood. The company is developing a proprietary genome editing technology based on CRISPR (clustered, regularly interspaced short palindromic repeats)/Cas9 (CRISPR associated protein 9), that has the potential to achieve precise, directed changes in DNA. Editas was founded by world leaders in genome editing, and its mission is to translate the promise of genome editing science into a broad class of transformative genomic medicines to benefit the greatest number of patients.


John Maslowski, SVP, Scientific Affairs
Oct. 6 | 9:15am | La Jolla Ballroom 2
Exton, PA
Fibrocell Science 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 gene therapy product candidate, FCX-007, has begun a Phase I/II trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in preclinical development of FCX-013, its gene therapy product candidate for the treatment of linear scleroderma. In addition, Fibrocell has a third gene therapy program in the research phase for the treatment of arthritis and related conditions. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation, a leader in synthetic biology.


Jean-Philippe Combal, Ph.D., Pharm.D., Chief Operating Officer
Oct. 5 | 3:00pm | La Jolla Ballroom 1
Paris, France
GenSight Biologics is a clinical-stage biotechnology company discovering and developing novel therapies for neurodegenerative retinal diseases and diseases of the central nervous system. GenSight Biologics’ pipeline leverages two core technology platforms, Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from severe degenerative retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber’s Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible vision loss in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to offer patients a sustainable, functional visual recovery with a single treatment to each eye through an intravitreal injection.


Doug Brough, Ph.D., Chief Scientific Officer
Oct. 5 | 11:00am | La Jolla Ballroom 2
Gaithersburg, MD
GenVec is a clinical-stage gene delivery company developing a pipeline of cutting-edge therapeutics using its proprietary AdenoVerse gene delivery platform. The platform provides assets and know-how for commercial product development across a range of applications, including immunotherapy, gene editing and cell and gene therapy. GenVec has extensive expertise in translating innovative product concepts from the laboratory to the clinic. Examples include the company’s lead product candidate, CGF166, partnered with Novartis, in a first-in-man Phase I/II clinical study for the treatment of hearing loss as well as the collaborative approach combining TheraBiologics’ neural stem cells with commercializable AdenoVerse technology to treat cancer. GenVec is particularly excited to share new T-cell transduction data, generated using proprietary vectors that are particularly well suited for efficient delivery of large therapeutic payloads to genetically engineer T-cells. GenVec believes its AdenoVerse platform and translational expertise can contribute significantly to collaborations with partners developing novel gene-based medicines.


Hardy Kagimoto, M.D., Ph.D., President and CEO
Oct. 6 | 11:00am | La Jolla Ballroom 2
Tokyo, Japan
(4593: Tokyo)
Healios is a biotechnology venture leading the field in developing iPS cell-based products for regenerative medicine. The company was founded in 2011 and listed on the stock exchange in 2015. Helios is developing a treatment for age-related macular degeneration (AMD) and organ bud transplantation therapy involving a technology to create functional human organs as an iPSC pipeline. The company is conducting a Phase II/III study in Japan for ischemic stroke as a somatic stem cell pipeline.


Adam Gridley, President and CEO
Oct. 5 | 5:15pm | La Jolla Ballroom 1
Boston, MA
Histogenics is a leading regenerative medicine company developing and commercializing products in the musculoskeletal segment of the marketplace. Histogenics’ regenerative medicine platform combines expertise in cell processing, scaffolding, tissue engineering, bioadhesives and growth factors to provide solutions to treat musculoskeletal-related conditions. Histogenics’ first investigational product candidate, NeoCart, is currently in Phase III clinical development. NeoCart is an autologous cell therapy designed to treat cartilage defects in the knee using the patient’s own cells. Knee cartilage defects represent a significant opportunity in the United States, with an estimated 500,000 or more applicable procedures each year. NeoCart is designed to exhibit characteristics of articular, hyaline cartilage prior to and upon implantation into the knee and therefore does not rely on the body to make new cartilage; characteristics not exhibited in other current treatment options. The company is also developing next generation allogeneic therapies with Intrexon Corporation.


Matthew Scholz, CEO
Oct. 6 | 2:30pm | La Jolla Ballroom 2
Seattle, WA
Immusoft’s mission is the development of a new breakthrough platform for treating a variety of genetic diseases through a process called Immune System Programming (ISP). ISP uses a patient’s immune cells to cure disease. Cells that are reprogrammed using ISP become miniature drug factories that are expected to survive in the patient for many years. Cell therapy with ISP is a better way to deliver biologic drug therapies using a safe, non-viral vector, the Sleeping Beauty (SB) transposon system. ISP replaces a lifetime of infusions by instructing cells to constantly secrete therapeutic enzymes, proteins or antibodies, minimizes the risk that a patient’s immune system may target a treatment and eliminates damage to veins and injection site reactions. The first clinical application is MPS I, a rare genetic lysosomal storage disease.



John Grant, Ph.D., Director, Business Development
Oct. 6 | 1:45pm | La Jolla Ballroom 1
Cambridge, MA
Intellia Therapeutics is building a fully integrated, product-driven biotechnology company that is focused on developing and commercializing potentially curative CRISPR/Cas9 based gene editing treatments. Intellia’s approach to advancing the broad potential of gene editing includes: focus on strategically identified “sentinel indications” that enable the company to fully develop the potential of the CRISPR/Cas9 system with a risk mitigated approach; aggressively pursue in vivo liver indications to develop therapeutics rapidly with existing delivery technology; continue to develop and expand ex vivo therapeutic programs through the company’s eXtellia division; leverage strategic partnerships, including existing collaborations with Novartis and Regeneron, to accelerate clinical development; and grow Intellia’s leadership position in the field of gene editing. A combination of deep scientific expertise and clinical development experience, along with the company’s leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products.


Tom Ulich, M.D., Chief Scientific Officer
Oct. 6 | 4:00pm | La Jolla Ballroom 2
Cambridge, MA
InVivo Therapeutics is a research and clinical-stage biomaterials and biotechnology company with a focus on treatment of spinal cord injuries. The company was founded in 2005 with proprietary technology co-invented by Robert Langer, Professor at Massachusetts Institute of Technology, and Joseph Vacanti, who was then at Boston Children’s Hospital and is now affiliated with Massachusetts General Hospital. In 2011, the company earned the David S. Apple Award from the American Spinal Injury Association for its outstanding contribution to spinal cord injury medicine. In 2015, the company’s investigational Neuro-Spinal Scaffold received the 2015 Becker’s Healthcare Spine Device Award.


Marlin Frechette, Director, Quality Systems and Regulatory Affairs
Oct. 5 | 4:00pm | La Jolla Ballroom 2
Santa Ana, CA
Irvine Scientific, a member of JX Group, is a worldwide leader in the innovation and manufacture of cell culture media, reagents and medical devices for researchers and clinicians. The company provides unrivalled service and quality to scientists working in cell therapy and regenerative medicine, assisted reproductive technology and cytogenetics, and industrial cell culture for the large-scale production of biotherapeutics and vaccines. Irvine Scientific adheres to both ISO and FDA regulations and operates dual cGMP manufacturing facilities in California and Tokyo, Japan. The company’s consultative philosophy combined with expertise in cell culture and compliance provides customers with unique capabilities and support. For over 40 years Irvine Scientific has remained uniquely flexible and focused on media while becoming a strategic global leader in media products and services.


Rahul Aras, Ph.D., President and CEO
Oct. 5 | 11:45am | La Jolla Ballroom 2
Cleveland, OH
Juventas Therapeutics is a private, clinical-stage biotechnology company developing novel non-viral gene therapies that activate natural processes to repair the body. The company’s product candidate, JVS-100, is a non-viral gene therapy that expresses stromal cell-derived factor-1, or SDF-1, a naturally occurring signaling protein that has been shown to recruit the body’s own stem cells and promote tissue repair in a broad range of disease states. Juventas’ therapeutic approach is based on research originating at the Cleveland Clinic. Juventas is currently enrolling a Phase IIb study in patients with advanced peripheral artery disease.


Jurjen Velthuis, Ph.D., VP of Chemistry, Manufacturing and Controls (CMC)
Oct. 6 | 4:45pm | La Jolla Ballroom 1
Amsterdam, The Netherlands
Kiadis Pharma uses T-cell based immunotherapies as an adjunctive treatment to haploidentical stem cell transplantation in hematological cancers and non-malignant diseases of the blood. The company is initiating a randomized transatlantic Phase III study under IND in 3Q, 2016 and had announced the decision to file a Marketing Authorisation Application (MAA) within the next 6-8 months in Europe. The company recently presented Phase II data at the American Society of Hematology (ASH) and European Society for Blood and Marrow Transplantation (EBMT) meetings.


Madhusudan Peshwa, Ph.D., Chief Scientific Officer; EVP, Cellular Therapies
Oct. 5 | 1:15pm | La Jolla Ballroom 2
Gaithersburg, MD
MaxCyte is an established, revenue generating U.S.-based developer and supplier of cell engineering technology to biotechnology and pharmaceutical firms engaged in cell therapy, drug discovery and development, biomanufacturing, gene editing and immuno-oncology. The company’s patented flow electroporation technology enables its products to deliver fast, reliable and scalable cell engineering to drive the research and clinical development of a new generation of cell-based medicines. MaxCyte’s high performance platform allows transfection with any molecule or multiple molecules with a high degree of consistency and minimal cell disturbance, thereby facilitating rapid, large scale, commercial and clinical grade cell engineering in a non-viral system and with low toxicity concerns. The company’s cell engineering technology platform is CE-marked and FDA-accredited, providing MaxCyte’s customers with an established regulatory path. MaxCyte is developing CARMA, its proprietary platform in immuno-oncology, to deliver a validated non-viral approach to CAR therapies in a number of cancer indications, including solid tumors.


Antonio Lee, Ph.D., CEO
Oct. 6 | 1:45pm | La Jolla Ballroom 2
Rockville, MD
MEDIPOST’s research and development focuses on allogeneic Umbilical Cord Blood-derived Mesenchymal Stem Cells (hUCB-MSCs) in areas of unmet medical need, such as Osteoarthritis (OA), Broncho-pulmonary Dysplasia (BPD) and Alzheimer’s disease (AD). MEDIPOST’s flagship product, CARTISTEM for OA has been approved by the regulatory agency in Korea as an allogeneic stem cell product in 2012 and to date over 3,500 patients have been treated on the market with an excellent long-term (5+ years) safety and efficacy profile, including 615 Post Market Surveillance (PMS) registries in Korea. CARTISTEM has also successfully completed Phase I/IIa trials in the U.S. PNEUMOSTEM, a therapy for the prevention of BPD in premature infants, completed a first-in-human Phase I trial, and a 70 subject Phase II RCT in Korea. PNEUMOSTEM has completed a Phase I/II clinical trial in the U.S. with Orphan Drug designations from Korea, the U.S. and EU. MEDIPOST America is a wholly-owned U.S. subsidiary of MEDIPOST Co.


Donna Skerrett, M.D., Chief Medical Officer
Oct. 5 | 11:30am | La Jolla Ballroom 1
Melbourne, Australia
Mesoblast is a world leader in cell-based medicines. The company has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells (MLCs), to establish a broad portfolio of late-stage product candidates. These allogeneic, off-the-shelf cell product candidates target advanced stages of diseases with high, unmet medical needs including cardiovascular diseases, orthopedic disorders, oncology and hematology diseases, and immune-mediated and inflammatory conditions. Mesoblast believes the company is well positioned to have the first industrially manufactured allogeneic stem cell product approved in the U.S. Mesoblast’s licensee in Japan has launched its mesenchymal stem cell-based product, which is the first allogeneic cell-based product to receive full approval in Japan. The majority of the company’s time and resources are focused on Tier 1 product candidates. Additionally, the company has a strong pipeline of products for follow-on indications.


Luca Alberici, Ph.D., Director, Business Development
Oct. 6 | 4:15pm | La Jolla Ballroom 1
Milan, Italy
MolMed is a medical biotechnology company focused on research, development and clinical validation of novel anticancer therapies. MolMed’s pipeline includes anti-tumor therapeutics in clinical and preclinical development. Zalmoxis (TK) is a cell-based therapy enabling bone marrow transplants from partially compatible donors, in the absence of post-transplant immune-suppression prophylaxis. It is currently in a Phase III trial in high-risk acute leukemia and was granted a positive opinion by the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) for a Conditional Marketing Authorization. CAR-CD44v6 is an immuno-gene therapy project potentially effective for many hematological malignancies and several epithelial tumors. MolMed also offers top-level expertise in cell and gene therapy to third parties to develop, conduct and validate projects from preclinical to Phase III trials, including scale-up and cGMP production of clinical-grade viral vectors, and manufacturing of patient-specific genetically engineered cells.


Colleen Delaney, M.D., Scientific Founder and Chief Medical Officer
Oct. 5 | 10:45am | La Jolla Ballroom 2
Seattle, WA
Nohla Therapeutics is a cellular therapy company focused on progressing the development and commercialization of a range of products from a unique ex vivo stem cell expansion platform developed at the Fred Hutchinson Cancer Research Center. Nohla’s lead product is a universal donor cell therapy product, NLA-101, a truly off-the-shelf cellular therapy, developed to enhance hematopoietic recovery, especially the myeloid lineage, associated with treatment for hematologic malignancies, and is being extended to treat a range of other medical indications. Nohla Therapeutics is working on extending this expansion platform to direct lineage specific differentiation and growth of other cell types such as NK cells or pre T-cells that can then be developed into other novel cell therapies.


Petter Bjorquist, Ph.D., CEO
Oct. 5 | 11:30am | La Jolla Ballroom 2
Goteborg, Sweden
NovaHep is a biotechnology company founded as a spin-off from the Karolinska Institute with a unique, proprietary technology in the field of regenerative medicine. The company is developing the next generation of tissue-engineered products for replacement therapy. NovaHep’s pipeline consists of high quality, biocompatible products that are individualized with the patient’s own stem cells, and the company’s first product in the area of vascular disease has already been tested in humans. NovaHep holds a proprietary technology for de- and re-cellularization of tissues and organs. In short, a donated tissue is de-cellularized followed by a process when this scaffold is seeded with the patient´s own cells, resulting in an allogeneic tissue becoming autologous. This addresses the two major shortcomings in transplantation surgery of today: availability of suitable donor tissue and the severe side effects following lifelong immunosuppressive treatment. The unique technique is covered by patent applications and trade secrets.


Nicolas Koebel, SVP, Business Operations
Oct. 6 | 1:00pm | La Jolla Ballroom 2
London, UK
Orchard Therapeutics is a clinical-stage biotechnology company with operations in London and the U.S., and is dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases.


Keith Murphy, Chairman and CEO
Oct. 5 | 5:00pm | La Jolla Ballroom 1
San Diego, CA
Organovo designs and creates functional, three-dimensional (3D) human tissues for use in medical research and therapeutic applications. The company develops 3D human disease models through internal development and in collaboration with pharmaceutical and academic partners. Organovo’s 3D human tissues have the potential to accelerate the drug discovery process, enabling treatments to be developed faster and at a lower cost. The company recently launched exVive3D Human Liver Tissue for use in toxicology and other preclinical drug testing, with the anticipated release of the exVive3D Human Kidney Tissue scheduled for Q3 2016. The company also actively conducts early research on specific tissues for therapeutic use in direct surgical applications. Organovo is changing the shape of medical research and practice.


Marcie Glickman, Ph.D., Chief Scientific Officer
Oct. 6 | 2:00pm | La Jolla Ballroom 2
Boston, MA
ORIG3N is transforming the future of health through Nobel Prize-winning regenerative medicine technology to produce individual-specific induced pluripotent stem cells (iPSCs) – grown from blood, not embryos – which can be used to grow different cell types including neurons, heart cells and liver cells. These cells offer a powerful tool for accurate disease modeling outside of the body.


Alla Danilkovitch, Ph.D., Chief Scientific Officer
Oct. 5 | 5:30pm | La Jolla Ballroom 1
Columbia, MD
Osiris Therapeutics is a leader in researching, developing and marketing cellular regenerative medicine products that improve the health and lives of patients and lower overall healthcare costs. Having developed the world’s first approved stem cell drug, Osiris works to further advance the medical field. Osiris’ research and development in biotechnology focuses on innovation in regenerative medicine – including stem cell research, bioengineering and viable tissue-based products. Osiris has achieved commercial success with products in orthopedics, sports medicine and wound care, including BIO4, a viable bone matrix; Cartiform, a viable osteochondral allograft; Grafix, a cryopreserved placental membrane; TruSkin, a viable human skin allograft; and Stravix, a durable placental allograft.


Jason Slingsby, Ph.D., Head of Business Development
Oct. 5 | 2:30pm | La Jolla Ballroom 1
Oxford, UK
Oxford BioMedica is a leading gene and cell therapy company focused on developing life changing treatments for serious diseases. Oxford BioMedica has built a sector leading lentiviral vector delivery platform (LentiVector) through which it develops in vivo and ex vivo products both in-house and with partners. The company has created a valuable proprietary portfolio of gene and cell therapy product candidates including OXB-102 for Parkinson’s disease, OXB-202 for corneal graft rejection and OXB-302, a CAR T program targeting the solid tumor antigen 5T4. The company has also entered into a number of partnerships, including with Novartis, Sanofi, GSK, Immune Design and Green Cross LabCell.


Zami Aberman, Chairman and CEO
Oct. 6 | 11:15am | La Jolla Ballroom 2
Haifa, Israel
Pluristem Therapeutics is a leading developer of placenta-based cell therapy products. The company has reported robust clinical trial data in multiple indications for its patented PLX (P Lacental eXpanded) cells. The cells release a range of therapeutic proteins in response to inflammation, ischemia, hematological disorders and radiation damage. PLX cell products are grown using the company’s proprietary three-dimensional expansion technology. They are off-the-shelf, requiring no tissue matching prior to administration. Pluristem has a strong intellectual property position; company-owned and operated GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.


Michael Dombeck, VP, Business Development
Oct. 5 | 2:15pm | La Jolla Ballroom 2
Durham, NC
At Precision Biosciences, the company utilizes a proprietary genome editing method called ARCUS, with a team of some of the leading minds and pioneers in genome editing. Precision’s goal is to overcome cancers, cure genetic diseases and develop safer and more productive food sources.


Torsten Hombeck, Ph.D., Chief Commercial and Strategy Officer
Oct. 6 | 4:30pm | La Jolla Ballroom 2
Brussels, Belgium
Promethera is a clinical-stage biopharmaceutical company as well as the global leader and most advanced cell therapy and regenerative medicine company for the treatment of inborn and acquired liver diseases with no effective therapeutic cure. Promethera uses allogeneic progenitor cells, stem cells and mature hepatocytes harvested and purified from non-transplantable, healthy human livers (Heterologous Human Adult Liver-derived Progenitor Cells, HHALPC; and Heterologous Human Liver Cells, HHLivC). Promethera develops therapeutic options for the treatment of a broad variety of liver diseases ranging from rare inborn metabolic diseases including urea cycle disorders (UCD) and inherited diseases such a1at deficiency (α1-antitrypsin) and hemophilia to large indications such as acute on chronic liver failure (ACLF), fibrosis and nonalcoholic steatohepatitis (NASH).


Tom Okarma, M.D., Ph.D., President and CEO
Oct. 5 | 5:15pm | La Jolla Ballroom 2
Los Altos, CA
Regencor was formed to commercially develop the Epicapatch, a resorbable collagen membrane containing modified human recombinant follistatin-like 1 (fstl1) applied to the epicardial surface overlying the infarct zone. When applied after infarction to rodents and large pigs, the patch delivers fstl1 over 4-6 weeks directly to the infarcted myocardium resulting in new blood vessel formation, new functional myocardium, reduced scar and improved LVEF and fractional shortening. The degree of regenerated myocardium, scar reduction and LV functional improvement are unprecedented. Regencor has secured IP protection and freedom to operate through two patent applications and three exclusive license agreements. The company is developing a proprietary cannula for closed chest patch delivery which will be ready for the first-in-human clinical trial, planned for 2018 at The University Medical Center, Utrecht.


Olav Hellebo, CEO
Oct. 5 | 2:15pm | La Jolla Ballroom 1
Guildford, UK
ReNeuron is a leading, clinical-stage cell therapy development business. Based in the UK, its primary objective is the development of novel cell-based therapies targeting areas of significant unmet or poorly met medical need. ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant diseases where the cells can be readily administered off-the-shelf to any eligible patient without the need for additional immunosuppressive drug treatments. The company’s therapeutic candidates for stroke disability and critical limb ischemia are in clinical development and its cell-based treatment for the blindness-causing disease, retinitis pigmentosa, is about to enter the clinic in the U.S. ReNeuron is also advancing a proprietary platform technology to exploit nanoparticles (exosomes) secreted by stem cells as potential new drug candidates targeting a range of cancers.


Sandy Macrae, Ph.D., President and CEO
Oct. 6 | 1:00pm | La Jolla Ballroom 1
Richmond, CA
Sangamo BioSciences is a clinical-stage genome editing company focused on Engineering Genetic Cures for monogenic and infectious diseases. Sangamo is developing novel ZFP Therapeutics for therapeutic genome editing and gene regulation based on its proprietary zinc finger DNA-binding protein (ZFP) technology platform. The company has an ongoing Phase II clinical study to evaluate the safety and efficacy of a novel T-cell based ZFP Therapeutic for the treatment of HIV/AIDS (SB-728), and an ongoing Phase I clinical trial of this same approach in hematopoietic stem cells (HSCs). Both strategies are designed to genetically engineer an HIV-resistant immune system that can achieve functional control of the virus. Sangamo is developing additional stem cell applications for monogenic diseases such as sickle cell disease and beta-thalassemia, and in vivo approaches for indications including hemophilia A and B, lysosomal storage disorders and Huntington’s disease.


Sun Song, Ph.D., CEO
Oct. 6 | 4:45pm | La Jolla Ballroom 2
Incheon, South Korea
SCM Lifescience was established in 2014 with the vision of developing treatment options for difficult to treat diseases to provide a new source of hope for patients and contribute to the welfare of humanity as a whole using proprietary stem cell isolation and production technologies. Through over 10 years of research and development, SCM Lifescience has obtained intellectual property protection for its high-purity stem cell isolation technology and high-purity stem cell production technology. Based on these technical breakthroughs, high-purity stem cell therapy will treat various immunological conditions such as graft-versus-host disease, acute pancreatitis, liver cirrhosis, atopic dermatitis, diabetes and asthma. With the company’s proprietary technology, SCM will continue to pursue excellence in the fields of stem cell and regenerative medicine to become an internationally recognized leader in these fields.


John Campbell, Ph.D., Professor and Associate Director, Research Development and Innovation
Oct. 6 | 4:15pm | La Jolla Ballroom 2
Edinburgh, UK
The Scottish National Blood Transfusion Service (SNBTS) is the national blood, blood product and tissue provider for Scotland. It makes up a Strategic Business Unit of NHS National Services Scotland. SNBTS has nearly a 20 year track record in cellular therapy research and manufacturing. SNBTS has a very strong record in full GMP translation of near-the-clinic products for use in clinical trials. Cellular therapy development and manufacturing is carried out at SNBTS’ dedicated MHRA-licensed facility based within the Scottish Center for Regenerative Medicine in Edinburgh.


Felicia Pagliuca, Ph.D., VP, Cell Biology Research and Development
Oct. 6 | 1:30pm | La Jolla Ballroom 2
Cambridge, MA
Semma Therapeutics was founded to develop transformative therapies for Type 1 diabetes patients. Work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing islet cells in the laboratory. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of- the-art device to provide a true replacement for the missing islet cells in a diabetic patient without immunosuppression. Semma Therapeutics is working to bring new therapeutic options to the clinic and improve the lives of patients with diabetes.


Robert Hutchens, President and CEO
Oct. 5 | 4:15pm | La Jolla Ballroom 2
San Antonio, TX
StemBioSys is a privately-held biomedical company focused on enabling optimized capture, growth and delivery of adult stem cells for research, therapeutic or drug discovery applications. Its patented technology platforms, licensed from the University of Texas Board of Regents, overcomes key obstacles to creating clinically useful stem cell therapies. The centerpiece of these enabling technologies is the High Performance Micro Environment (HPME) platform. This cell derived three-dimensional microenvironment allows a variety of stem cells to replicate more rapidly, maintain a small cell size and express markers indicative of retained stem cell potency beyond that seen with more traditional tissue culture substrates. StemBioSys’ first commercial product BM-HPME, is currently available to the stem cell research market. With directed collaborations and further research, StemBioSys hopes to extend the disruptive technology presented by the company’s HPME platform to drug discovery and therapeutic applications.


Norio Nakatsuji, Ph.D., Director and Chief Advisor
Oct. 5 | 5:45pm | La Jolla Ballroom 2
Kyoto, Japan
Stem Cell & Device Laboratory advances research and innovative technology through the integration of stem cell technology and nanotechnology/micro-engineering, with a focus on human pluripotent stem cells (ES/iPS cells). Through the advancement of these technologies to practical applications, the company will create novel stem-cell-based products and contribute to the industry and society. The company develops and supplies high quality cell and micro-tissue products, starting from cardiomyocyte products, which can be used for drug screening and drug safety assay.


Michael Roberts Ph.D., Founder and Chief Scientific Officer
Oct. 5 | 1:45pm | La Jolla Ballroom 2
Edinburgh, UK
Synpromics gives researchers, developers and manufacturers unprecedented control of gene expression through proprietary technology that yields highly specific and active synthetic promoters. The company’s patentable synthetic promoters are designed to regulate genes in a customized manner and help facilitate a competitive IP position for Synpromics’ partners. Given that these promoters greatly improve upon the viral and natural promoters on which the cell and gene therapy industry currently relies, there is enormous potential for the technology to help drive the industry forward and transform the entire sector.


David Perez, President and CEO; Chairman, Blood Management Division, Terumo Corporation
Oct. 5 | 3:15pm | La Jolla Ballroom 2
Denver, CO
Terumo BCT, a global leader in blood component and cellular technologies, is the only company with the unique combination of apheresis collections, manual and automated whole blood processing, and pathogen reduction coupled with leading technologies in therapeutic apheresis and cell processing. The company believes in the potential of blood to do even more for patients than it does today. This belief inspires Terumo BCT’s innovation and strengthens the company’s collaboration with customers.


Bryan Poltilove, General Manager, Cell Therapy, BioSciences Division, Thermo Fisher Scientific
Oct. 6 | 10:00am | La Jolla Ballroom 2
Waltham, MA
Thermo Fisher Scientific is the world leader in serving science which uniquely positions the company to partner with customers to provide end-to-end cell therapy solutions. Thermo Fisher Scientific understands that cell therapy is a dynamic industry with rapidly evolving technologies and processes and are committed to helping cell therapy customers transition from discovery to clinical research and commercial manufacturing. Thermo Fisher Scientific’s portfolio of trusted and recognized products and services combined with over 50 years of cell culture knowledge and experience allows the company to provide comprehensive solutions to support every step of the cell therapy process – from cell collection through manufacture to clinical site delivery. Through Thermo Scientific, Applied BioSystems, Invitrogen, Fisher Scientific, Fisher BioServices, Unity Lab Services, and Gibco Cell Therapy Systems (CTS) brands, the company offers an unmatched combination of innovative technologies, purchasing convenience and comprehensive support.


Miguel Mulet, Director, Strategy and New Projects
Oct. 6 | 11:00am | La Jolla Ballroom 2
Leuven, Belgium
TiGenix is an advanced biopharmaceutical company focused on developing and commercializing novel therapeutics from its proprietary platforms of allogeneic, or donor-derived, expanded stem cells. Two products from the adipose-derived stem cell technology platform are currently in clinical development. Cx601 is in Phase III for the treatment of complex perianal fistulas in Crohn’s disease patients. Cx611 has completed a Phase I sepsis challenge trial and a Phase I/II trial in rheumatoid arthritis. In 2015 TiGenix acquired Coretherapix, whose lead cellular product, AlloCSC-01, is currently in a Phase II clinical trial in acute myocardial infarction (AMI). In addition, the second product candidate from the cardiac stem cell-based platform acquired from Coretherapix, AlloCSC-02, is being developed in a chronic indication. In July 2016 TiGenix entered into a licensing agreement with Takeda, a large pharmaceutical company active in gastroenterology, under which Takeda acquired the exclusive right to commercialize Cx601 for complex perianal fistulas outside the U.S. TiGenix is headquartered in Leuven, Belgium and has operations in Madrid, Spain.


Jongmoon Kim, CEO
Oct. 5 | 2:00pm | La Jolla Ballroom 2
Seoul, Korea
(KONEX: 199800)
ToolGen is a publicly traded biotechnology company focused on the development and application of genome editing technologies. It creates, and holds intellectual property rights for, essential tools and technologies for editing the genetic information in microbial, plant, animal and human cells. ToolGen’s mission is to translate the potential of the company’s innovative platform technology into transformative products for biomedicine and agriculture.


Jon Curley, Chief Technology Officer
Oct. 6 | 9:45am | La Jolla Ballroom 2
Cardiff, UK
TrakCel was established in 2012 to meet a need within the cell therapy industry for managing complex supply chains. The company’s technology platform provides a unique way of controlling and coordinating complex supply chain models while maintaining regulatory compliant reporting. TrakCel is more than just a technology provider, it assists its clients with operational deployment and maintenance, acceptance testing and validation. The company provides around-the-clock customer support for users of its technology.


Stephane Boissel, CEO
Oct. 5 | 3:00pm | La Jolla Ballroom 2
Valbonne, France
TxCell is a publicly listed biotechnology company that develops platforms for innovative, personalized T-cell immunotherapies for the treatment of severe chronic inflammatory and autoimmune diseases with high unmet medical need. TxCell is the only clinical-stage cellular therapy company dedicated to the science of regulatory T lymphocytes (Tregs). Tregs are a recently discovered T-cell population for which anti-inflammatory properties have been demonstrated. Ovasave, TxCell’s lead product candidate, is currently in a Phase IIb clinical trial in refractory Crohn’s disease patients. Col-Treg, its second product candidate, is in preclinical development for the treatment of autoimmune uveitis.


Claudia Mitchell, Ph.D., CEO
Oct. 5 | 2:30pm | La Jolla Ballroom 2
Seattle, WA
Universal Cells is a therapeutic genome editing company leveraging its nuclease-free, adeno-associated virus (AAV)-mediated genome editing technology to eliminate major barriers to the commercialization and safety of cell and gene therapies. The company’s gene therapy program focuses on safe and accurate ex vivo correction of mutated genes for patients with rare hematological disorders. The Universal Donor Cell program focuses on innovative strategies to completely control HLA molecules to create allogeneic cell therapy products that are resistant to host T-cells, NK cells and antibodies. The company has recently established two major strategic partnerships to help advance the Universal Donor Cell Program to the clinic. By combining the safest gene editing approach with a deep understanding of stem cell immunology, the company is positioned to play a central role in the commercialization of pluripotent cell-based therapies.


Nick Colangelo, President and CEO
Oct. 5 | 5:45pm | La Jolla Ballroom 1
Cambridge, MA
Vericel Corporation is a leader in developing patient-specific expanded cellular therapies for use in the treatment of patients with severe diseases and conditions. The company markets two autologous cell therapy products in the U.S.: Carticel (autologous cultured chondrocytes), an autologous chondrocyte implant for the treatment of cartilage defects in the knee; and Epicel (cultured epidermal autografts), a permanent skin replacement for the treatment of patients with deep-dermal or full-thickness burns comprising greater than or equal to 30% of total body surface area. Vericel is also developing MACI, a third-generation autologous chondrocyte implant for the treatment of cartilage defects in the knee, and ixmyelocel-T, a patient-specific multicellular therapy for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy.


Paul Laikind, Ph.D., President and CEO
Oct. 6 | 1:15pm | La Jolla Ballroom 2
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 development based on PEC-01 pancreatic progenitor cells differentiated from pluripotent stem cells. PEC-Direct product candidate delivers the pancreatic progenitor cells in a device that does not provide immune protection and is being developed for type 1 diabetes patients that have a high risk of acute complications. PEC-EnCap (formerly VC-01) product candidate delivers pancreatic progenitor cells in an immunoprotective device. PEC-EnCap is currently being evaluated in a Phase I/II trial in patients with type 1 diabetes who have minimal to no insulin-producing beta cell function. The company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.


Amy DuRoss, Co-Founder
Oct. 5 | 4:45pm | La Jolla Ballroom 2
Menlo Park, CA
Led by a hybrid of proven life science and software development experts, Vitruvian Networks will focus on enabling life changing autologous therapies, supporting standards and developing infrastructure that will expand patient access through expedited discovery, delivery and regulation in the field. Vitruvian Networks is committed to scaling and digitizing cell and gene therapies through cloud-ready software systems and designed-for-purpose manufacturing services.


Jeff Goater, Chief Financial Officer
Oct. 6 | 2:15pm | La Jolla Ballroom 1
Cambridge, MA
Voyager Therapeutics is a clinical-stage gene therapy company developing life-changing treatments for severe diseases of the central nervous system (CNS). Gene therapy has the potential to transform the treatment of CNS and other diseases by providing a one-time therapy that dramatically improves patients’ lives. The company is committed to advancing the field of adeno-associated virus (AAV) gene therapy through innovation and investment in vector optimization and engineering and dosing techniques, as well as process development and production.