Company Presentations

View the 2021 Slate of Presenting Companies!

This year’s meeting featured 90+ presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

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2021 Company Presenters

Yuta Lee, CEO
Carlsbad, CA
Accelerated Biosciences is a regenerative medicine company sourcing the highest quality immune-privileged human Trophoblast Stem Cells (hTSCs) with no socio-ethical controversy. The company is focused on supplying well-characterized cell types from its primary hTSC source. hTS cells are pluripotent, immune-privileged, ethically derived, virus and pathogen-free, easy to manufacture, genetically stable, and GTP-compliant. The company has optimized expansion 75+ population doublings and has 28 issued patents worldwide with seven patent families in prosecution. Accelerated Biosciences has differentiated these stem cells into NK, neuronal, pancreatic, and hepatic cells banked and ready to go. The company is available for partnering and licensing opportunities.

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

Mark Womack, Chief Business Officer
Seattle, WA
Right. On Time. AGC Biologics is a leading global Contract Development and Manufacturing Organization (CDMO), providing world-class development and manufacture of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), viral vectors, and genetically engineered cells. The company’s commitment to continuous innovation fosters the technical creativity to solve clients’ most complex challenges, including specialization in fast track projects for orphan drugs and rare diseases.

Sue Washer, President and CEO
Alachua, FL
AGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. The company’s most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the company’s industry-leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology, and CNS disorders.

Hans Keirstead, Ph.D., Chairman and CEO
Irvine, CA
AIVITA Biomedical is a biotechnology company developing personalized vaccines for the prevention of COVID-19 and treatment of cancer. The company targets the seed of all cancers: tumor-initiating cells, using a unique pan-antigenic approach. By targeting all neoantigens, rather than a select few, AIVITA’s treatment overcomes cancer’s tendency to mutate over time. The company’s personalized COVID-19 vaccine can be rapidly adapted and scaled to meet any emerging viral mutations. The company has also leveraged capabilities to create a proprietary skincare line that dedicates net proceeds towards advancing revolutionary treatments for patients with cancer. AIVITA explores additional applications in human health, including vision loss. AIVITA owes these innovations to its unique expertise in stem cell growth and directed, high-purity differentiation which enables the company to develop safe, efficient, and economical manufacturing processes for therapeutic development.

David Smith, President and Chief Commercial Officer
Boca Raton, FL
Akron Biotech drives advanced therapy development and commercialization with high quality, industrial scale solutions. A regulatory compliant (ISO-certified) company, Akron’s strategic focus is manufacturing cGMP-compliant ancillary materials and providing services that enable the advancement of cell and gene therapies. These include cytokines and growth factors, human sera and purified proteins, cryopreservation solutions, plasmid DNA manufacturing and custom development services.

Jeff Galvin, CEO
Rockville, MD
American Gene Technologies (AGT) is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. AGT’s mission is to transform people’s lives through genetic medicines that rid the body of disease. AGT has received three patents for the technology used to make the AGT103-T cell product and 10 patents for its unique immuno-oncology approach to stimulate gamma-delta (γδ) T cells to destroy a variety of solid tumors. The company has developed a synthetic gene for treating Phenylketonuria (PKU), a debilitating inherited disease. AGT’s treatment for PKU has been granted Orphan Drug Designation by the Food and Drug Administration (FDA), and it is expected to reach the clinic in 2022.


Jeff Castelli, Ph.D., Chief Development Officer
Cranbury, NJ
Amicus Therapeutics is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases, including an industry leading rare disease gene therapy portfolio.

Kaare Engkilde, Ph.D., CEO
Lund, Sweden
Amniotics™ was born out of the discovery of a novel source of stem cells in full-term amniotic fluid. Based on a decade of research at the internationally recognized Lund University Stem Cell Center and Hospital, the company is pioneering the harvesting and propagation of tissue relevant neonatal quality mesenchymal stem cells (MSCs). Researchers and founders of the company, pediatrician Marcus Larsson, obstetrician Andreas Herbst, and stem cell specialist Niels-Bjarne Woods, discovered a new type of stem cells in amniotic fluid that has unique properties for applications in regenerative medicine. The company raised SEK 25M in 2020 to validate MSCs in preclinical models for lung disease as well as for the completion and process validation of the GMP manufacturing facility. With the new GMP facilities approved in October 2020, Amniotics is now looking to establish strategic partnerships with companies that are interested in developing stem cell-based therapies targeting diseases with unmet needs.

Stefano Baila, Ph.D., Director of Operations
Gerenzano, Italy
Anemocyte is a Biotech Manufacturing Organization (BMO), a biotech company active in the field of cell and gene therapies (CGTs) that addresses CGT needs proactively by offering one-stop shop solutions and fostering exciting innovations. The company’s business offers process development and GMP capabilities (somatic cells, non-viral modified cells) and plasmids for viral vector manufacturing. Anemocyte’s experience includes more than 15 years of GMP manufacturing of CGTs and biological drugs; and more than 60 years of contract manufacturing within Nine Trees Group S.p.A.

Hendrik Jan Leonard Ankersmit, M.D., CEO
Vienna, Austria
Aposcience AG is a spin-off of the Medical University of Vienna developing a novel drug product manufactured from stressed white blood cells (PBMCs). The company was founded in 2008 and is funded by private investors. Aposcience has successfully completed preclinical studies, development of a GMP production facility, and CMC and toxicology studies for the company’s product Aposec. Aposec is a cell-free product with double (orthogonal) viral clearance that contains proteins, lipids, and exosomes. The lead product, APO-2 (Aposec for the treatment of hard-healing wounds), is currently tested in a multinational randomized, placebo-controlled, double-blind clinical Phase I/II trial (MARSYAS II, Identifier: NCT04277598). MARSYAS II is the first clinical trial for a secretome-based product ever, and Aposec (being classified as a biologic) is a new substance class in regenerative medicine.

Will Chou, M.D., CEO
New York, NY
Aruvant Sciences is a clinical-stage biopharmaceutical company focused on developing and commercializing gene therapies for the treatment of rare diseases. The company has a talented team with extensive experience in the development, manufacturing, and commercialization of gene therapy products. Aruvant has an active research program with a lead product candidate, ARU-1801, in clinical development for individuals suffering from sickle cell disease (SCD).


David Venables, Ph.D., President, AskBio Europe
Research Triangle Park, NC
AskBio, a wholly owned, independently operated subsidiary of Bayer AG, is a fully integrated AAV gene therapy company dedicated to developing life-saving medicines that cure genetic diseases. The company maintains a portfolio of clinical programs across neuromuscular, CNS, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for Pompe disease, Parkinson’s disease, and congestive heart failure, as well as out-licensed clinical indications for hemophilia and Duchenne muscular dystrophy. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive AAV capsid and promoter library. With global headquarters in Research Triangle Park, NC, and European headquarters in Edinburgh, UK, AskBio has generated hundreds of proprietary third-generation AAV capsids and promoters, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production and chimeric and self-complementary capsids.

Tamer Mohamed, CEO
Vancouver, Canada
Aspect Biosystems is a privately held biotechnology company combining the power of microfluidics and 3D bioprinting to fuel medical research and the development of bioprinted therapeutics. By adopting Aspect’s microfluidic 3D bioprinting platform and collaborating within Aspect’s network, researchers worldwide are accelerating the development and commercialization of 3D bioprinted tissues. Internally, Aspect is advancing multiple regenerative medicine programs, initially focused on metabolic, pancreas, and liver diseases and strategically partnering with global biopharma leaders to bring bioprinted therapeutics to the clinic.

Yoshitsugu Shitaka, Ph.D., President, Astellas Institute for Regenerative Medicine
Tokyo, Japan
Astellas Pharma is an innovative pharmaceutical company with global headquarters in Tokyo, Japan, conducting business in more than 70 countries around the world. Astellas is promoting the focus area approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on biology and modality. Furthermore, the company is also looking beyond its foundational Rx focus to create Rx+® healthcare solutions that combine the company’s expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients.

Pascal Touchon, President and CEO
San Francisco, CA
Atara Biotherapeutics is a pioneer in T-cell immunotherapy, leveraging its novel allogeneic EBV T-cell platform to develop transformative therapies for patients with serious diseases including solid tumors, hematologic cancers, and autoimmune disease. With the company’s lead program in Phase III clinical development, Atara is the most advanced allogeneic T-cell immunotherapy company and intends to rapidly deliver off-the-shelf treatments to patients with high unmet medical need. The company’s platform leverages the unique biology of EBV T cells and has the capability to treat a wide range of EBV-associated diseases, or other serious diseases through incorporation of engineered CARs or TCRs. Atara is applying this one platform to create a robust pipeline including: tab-cel® (tabelecleucel) in Phase III development for Epstein-Barr virus-driven post-transplant lymphoproliferative disease; ATA188, a T-cell immunotherapy targeting EBV antigens as a potential treatment for multiple sclerosis; and multiple next-generation chimeric antigen receptor T-cell immunotherapies for both solid tumors and hematologic malignancies.

Chris Vann, Chief Operating Officer
London, UK
Autolus is a leader in T-cell programming technologies and believes its leadership will provide a competitive advantage as it looks to develop future generations of T-cell therapies targeting both hematological cancers and solid tumors.

Michael Maguire, Ph.D., CEO
Co. Kildare, Ireland
Avectas is a cell engineering company developing SOLUPORE, a patented non-viral cell engineering technology addressing challenges in intracellular delivery and manufacturing in the immuno-oncology space including CAR-T and gene editing.

Geoff MacKay, President and CEO
Cambridge, MA
AVROBIO strives to give people with lysosomal disorders freedom for life through gene therapies designed to halt or reverse disease with a single dose. The company’s goal is simple and powerful: aim to enable individuals with genetic disease to live longer free from disease progression, free from painful and debilitating symptoms, and free from the burden of chronic treatment regimens. AVROBIO’s initial focus is on lysosomal disorders, where the standard of care does not halt disease progression or adequately address many symptoms, particularly in the brain and nervous system. The company’s investigational therapies are currently being studied in clinical trials in Fabry disease, Gaucher disease, and cystinosis. AVROBIO is also advancing a program in Pompe disease.

Haro Hartounian, Ph.D., SVP and General Manager
Newark, NJ
BioCentriq is a full service Contract Development and Manufacturing Organization (CDMO) for cell and gene therapy process development and clinical manufacturing with a BSL-2 certified process development and ISO-7 certified GMP clinical manufacturing facility. The company manufactures autologous and allogeneic cell therapies, gene therapies, and provides analytical and quality services across several cell types. BioCentriq also conducts viral vector production, cell and viral banking, and upstream and downstream processing. Additionally, the company has the capabilities to manufacture immunotherapies, including monoclonal antibodies and proteins, and vaccines. BioCentriq allows customer access to work in hybrid teams from tech transfer through GMP clinical production to accelerate scale-up and translation activities. In addition, to help the industry overcome the skills gap, the company offers pre-defined and custom workforce development programs as well as full access to BioCentriq’s centers during projects in a unique collaborative approach with our expert scientists to ensure knowledge transfer.

Samantha Vieira, Executive Director, Product Development and Commercial Lead
Cambridge, MA
At Biogen, our mission is clear: we are pioneers in neuroscience. Since our founding in 1978 as one of the world’s first global biotechnology companies, Biogen has led innovative scientific research with the goal over the last decade to defeat devastating neurological diseases.

Seth Ettenberg, Ph.D., Chief Scientific Officer
Cambridge, MA
BlueRock Therapeutics is using its unique cell and gene platform to direct cellular differentiation and genetically engineer cells to create an entirely new generation of cellular medicines in the areas of neurology, cardiology, and immunology.

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

Steven Nichtberger, M.D., President and CEO
Philadelphia, PA
Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T-cell therapies, and exploring their potential to provide a deep and durable, perhaps curative, treatment, for patients with B cell-mediated autoimmune diseases. The Cabaletta approach to selective B-cell Ablation (CABA) platform, in combination with Cabaletta’s proprietary technology, utilizes Chimeric AutoAntibody Receptor (CAAR) T-cells that are designed to selectively bind and eliminate only specific autoantibody-producing B cells while sparing normal antibody-producing B cells, which are essential for human health. The company’s lead product candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ phase I clinical trial as a potential treatment for patients with mucosal pemphigus vulgaris. The FDA has granted Fast Track Designation for DSG3-CAART. The company’s lead preclinical product candidate, MuSK-CAART, is designed as a potential treatment for patients with MuSK-associated myasthenia gravis. An IND submission for MuSK-CAART is anticipated in 2H21.

Rachel Haurwitz, Ph.D., President and CEO
Berkeley, CA
Caribou Biosciences is a leading clinical-stage CRISPR genome editing biotechnology company founded by pioneers of CRISPR biology. The company is developing an internal pipeline of off-the-shelf genome-edited CAR-T and natural killer (NK) cell therapies. CB-010, Caribou’s lead allogeneic CAR-T cell program, targets CD19 and has been cleared by the FDA for clinical evaluation. CB-011, Caribou’s second allogeneic CAR-T cell therapy, targets BCMA. Through its next-generation CRISPR genome editing technologies, Caribou implements multiple strategies to boost CAR-T cell persistence to overcome T-cell exhaustion and to prevent rapid immune-mediated clearance.

Bernd Leistler, Ph.D., VP, Production
Freiburg, Germany
CellGenix is a leading global supplier of high quality raw materials for the expanding cell and gene therapy and regenerative medicine space. The company develops, manufactures, and market human cytokines and growth factors in preclinical and GMP quality along with GMP serum-free media for further manufacturing of ATMPs. Our products are used by academia and industry partners in clinical trials and commercial manufacturing throughout the world. With 25 years of experience we are experts in the GMP manufacturing of raw materials for the cell and gene therapy space. As a former ATMP developer and manufacturer, we gained in-depth cell culture knowledge and superior regulatory expertise. With this unique background we understand the high requirements our customers face during product development and the regulatory approval process. By offering expert technical and regulatory support we can help simplify raw material qualification and validation efforts.

Nabiha Saklayen, Ph.D., Co-Founder and CEO
Cambridge, MA
Cellino’s vision is to make personalized regenerative medicines viable at large scale. Cellino’s platform combines label-free imaging and high-speed laser editing with machine learning to automate cell reprogramming, expansion, and differentiation in a closed cassette format, enabling thousands of patient samples to be processed in parallel in a single facility.

Filippo Petti, CEO
Mont-Saint-Guibert, Belgium
Celyad Oncology is a clinical-stage biotechnology company focused on the discovery and development of chimeric antigen receptor T-cell (CAR-T) therapies for cancer. The company is developing a diversified pipeline of allogeneic and autologous CAR-T cell therapy candidates for cancer patients with hematological malignancies and solid tumors.

Adrienne Farid, Ph.D., Chief Development Officer
Philadelphia, PA
Century Therapeutics is a leading off-the-shelf induced pluripotent stem cell (iPSC)-based cell therapy company developing genetically engineered allogeneic cell products for oncology, that overcome many limitations of first-generation cell therapies. The company’s unique and proprietary iPSC platform generates potent CAR-T and CAR NK products that are designed to target hematologic and solid tumor cancers, avoid host rejection, and provide durable responses for patients, with on demand availability at any hospital or clinic.

Nicole Faust, Ph.D., CEO
Koeln, Germany
CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company’s product portfolio comprises platform technologies for gene therapy viral vectors (AAV, adenoviral vectors, lentiviral vectors) vaccines, and complex recombinant proteins. With the ELEVECTA® Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any transfection reagents, cGMP plasmids or helper virus. CEVEC’s CAP® Technology based on human suspension cells is the ideal production platform for RCA-free adenoviral vectors, lentiviral vectors, oncolytic viruses, viral vaccines, and exosomes.

Laurent Picard, Ph.D., Global Director, Strategy and Business Development
Corning, NY
As a leading developer, manufacturer, and global supplier of laboratory products for over 100 years, Corning Life Sciences works with researchers and drug manufacturers seeking to increase efficiencies, reduce costs, and compress timelines. Using unique expertise in the fields of materials science, polymer surface science, cell culture, and biology, Corning Life Sciences provides innovative solutions that improve productivity and enable breakthrough research. Corning’s Life Sciences products include consumables (such as plastic vessels, specialty surfaces, cell culture media, and serum), as well as general labware and equipment that are used for advanced cell culture research, bioprocessing, genomics, drug discovery, microbiology, and chemistry.

Jayanthi Grebin, Senior Business Development Manager, Cell and Gene Therapy
Roseville, MN
CPC – Colder Products Company is the leader in single-use connection technology offering a wide variety of cell and gene therapy connection solutions that ensure ease of use and a robust connection. Single-use technologies (SUT) saves time and labor during set-up and between cycles, by eliminating steps, costs of cleaning, sterilization, and eliminating cross-contaminations between batches. CPC connectors have been tested and validated to comply with accepted industry standards for mechanical strength, extractables and leachables, bacterial ingress. and others.

Eric Unrau, VP of Operations, Europe
Basel, Switzerland
CRB is a leading provider of engineering, architecture, construction, and consulting solutions to the global life sciences and advanced technology industries, with an emphasis on cell and gene therapy. Our more than 1,400 employees provide best-in-class solutions that drive success and positive change for our clients, our people, and our communities. CRB is a privately held company with a rich history of serving clients throughout the world, consistently striving for the highest standard of technical knowledge, creativity, and execution.

Mark Sawicki, Ph.D., CEO
Nashville, TN
Cryoport Systems is the trusted global provider of temperature-controlled supply chain solutions for the life sciences industry, serving the biopharmaceutical market with leading-edge logistics solutions for biologic materials, including immunotherapies, stem cells, and CAR-T cells. Cryoport Systems’ proprietary Cryoport Express® Shippers, Cryoportal® Logistics Management Platform, leading-edge Smartpak II® Condition Monitoring System and geo-sensing technology, paired with unparalleled logistics expertise and 24/7/365 client support, make Cryoport Systems the end-to-end temperature controlled logistics partner that the industry trusts. In most recent news, Cryoport Systems is currently supporting over 500 active clinical trials and has been selected by Novartis, Kite, and bluebird bio to support the commercialization of their advanced therapies.

Laurence Reid, Ph.D., CEO
Boston, MA
Decibel Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing transformative treatments to restore and improve hearing and balance, one of the largest areas of unmet need in medicine. Decibel has built a proprietary platform that integrates single-cell genomics and bioinformatic analyses, precision gene therapy technologies, and expertise in inner ear biology. Decibel is leveraging its platform to advance gene therapies designed to selectively replace genes for the treatment of congenital, monogenic hearing loss and to regenerate inner ear hair cells for the treatment of acquired hearing and balance disorders. Decibel’s pipeline, including its lead gene therapy program, DB-OTO, to treat congenital, monogenic hearing loss, is designed to deliver on our vision of a world in which the privileges of hearing and balance are available to all.

Adam Muzikant, Ph.D., Chief Business Officer
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). The company’s first product, ZILRETTA® (triamcinolone acetonide extended-release injectable suspension), is the first and only FDA-approved extended-release, intra-articular (in the joint) injection for OA knee pain. Flexion’s pipeline includes FX201, an intra-articular gene therapy candidate being developed for symptomatic relief and disease modification in patients with knee OA. FX201 is designed to durably stimulate the production of an anti-inflammatory protein, IL-1Ra, whenever inflammation is present in the joint. Enrollment in a Phase I clinical trial with FX201 in patients with knee OA began in Q1 2020. The company is also developing FX301, a locally delivered NaV1.7 inhibitor formulated for extended release from a thermosensitive hydrogel, for control of post-surgical pain. FX301 is expected to enter clinical trials in 2021.

Timothy Miller, Ph.D., Co-Founder, President and CEO
Grove City, OH
Forge Biologics is a gene therapy manufacturing and development company helping clients achieve their cGMP program goals. Forge enables AAV vector production at 1-50L research and 50-500L cGMP grade scales, including process and analytical development. The company’s mission is to enable access to life changing gene therapies and help bring them from idea into reality.

Thomas Tradler, Ph.D., Head of Business Development
Leipzig, Germany
Representing a member of Europe’s largest organization for applied research, the Fraunhofer Institute for Cell Therapy and Immunology IZI investigates and develops solutions to specific problems at the interfaces of medicine, life sciences, and engineering.

Dennis Powers, VP, Business Development and Sales Engineering
College Station, TX
G-CON Manufacturing designs, produces, and installs prefabricated cleanrooms called PODs. G-CON is the global leader of cleanroom design and construction with a POD portfolio that encompasses a variety of different dimensions and purposes, from aseptic fill/finish and laboratory environments to advanced therapies and production process platforms. PODs are unique from traditional cleanroom structures due to the ease of scalability, mobility, and the ability to repurpose the PODs once the production process reaches the end of its lifecycle.

Angela Columbano, Ph.D., Head, Business Development and Partnership
Évry, France
Genethon’s mission is to design gene therapy products for rare diseases, and to ensure their preclinical and clinical development in order to provide patients with access to these innovative treatments.

Michael Redman, EVP and Chief Operating Officer
Austin, TX
Genprex is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also been shown to block mechanisms that create drug resistance.

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

Jennifer Guzman, Senior Director, Medical Affairs Strategy
Seoul, Korea
(KOSDAQ: 084990)
Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals to tackle previously untreated diseases, and is listed on KOSDAQ. The company has an extensive gene therapy pipeline, including a CAR-T program targeting several different types of solid tumors and an AAV vector program targeting neuromuscular diseases. Engensis (VM202), the most advanced pipeline candidate, is a plasmid DNA therapy being studied for DPN, diabetic foot ulcers, claudication, amyotrophic lateral sclerosis (Phase II beginning in late 2020), coronary artery disease, and Charcot-Marie-Tooth disease.

David Hodl, Head, Business Development, Cell Therapy
Melbourne, Australia
Invetech helps cell and gene therapy developers to visualize, strategize, and manage the future. With proven processes, expert insights and full-spectrum services, the company swiftly accelerates life-changing therapies from clinical to commercial-scale manufacturing. With configurable technology platforms and automated production systems, Invetech enables predictable, reproducible products of the highest quality and efficacy. The company’s integrated approach brings together biological scientists, engineers, designers, and program managers to deliver successful market offerings to more people globally. Working in close collaboration with early-stage and mature life science companies, Invetech is committed to advancing the next generation of vital, emerging therapies to revolutionize healthcare and precision medicine.

Maria Fardis, Ph.D., President and CEO
San Carlos, CA
Iovance Biotherapeutics aims to improve patient care by making T cell-based immunotherapies broadly accessible for the treatment of patients with solid tumors and blood cancers. Tumor infiltrating lymphocyte (TIL) therapy uses a patient’s own immune cells to attack cancer. TIL cells are extracted from a patient’s own tumor tissue, expanded through a proprietary process, and infused back into the patient. Upon infusion, TIL reach tumor tissue, where they attack cancer cells. The company has completed dosing in the pivotal study in patients with metastatic melanoma and cervical cancer. In addition, the company’s TIL therapy is being investigated for the treatment of patients with locally advanced, recurrent, or metastatic cancers including head and neck and non-small cell lung cancer. A clinical study to investigate Iovance T cell therapy for blood cancers called peripheral blood lymphocyte (PBL) therapy is open to enrollment.

Arthur Lahr, CEO
Amsterdam, The Netherlands
(Euronext Amsterdam: KDS)
Kiadis’ NK-cell programs consist of off-the-shelf and haplo donor cell-based medicines to treat liquid and solid tumors as adjunctive and stand-alone therapies and infectious diseases. The company’s NK-cell PM21 particle technology enables improved ex vivo expansion and activation of anti-cancer cytotoxic NK-cells supporting multiple high-dose infusions. Kiadis’ proprietary off-the-shelf NK-cell platform uses NK-cells from unique universal donors. NK-cell based products can be produced rapidly and economically for a broad patient population across a potentially wide range of indications. Kiadis is clinically developing K-NK003 for the treatment of relapse/refractory acute myeloid leukemia. The company is also developing K-NK002, which is administered as an adjunctive immunotherapeutic on top of HSCT and provides functional, mature, and potent NK-cells from a haploidentical family member. Furthermore, Kiadis is developing K-NK-ID101 for the treatment of Covid-19. In addition, the company has preclinical programs evaluating NK-cell based medicines for the treatment of solid tumors and infectious diseases.

Krish Krishnan, Chairman and CEO
Pittsburgh, PA
Krystal Biotech is a pivotal-stage gene therapy company leveraging its novel delivery platform to develop corrective, redosable therapies to address serious rare skin and lung diseases.

Paulo Garcia, Ph.D., CEO
Cambridge, MA
Kytopen is a spin-out of MIT developing proprietary platforms for non-viral delivery of payloads for ex vivo cell therapy discovery and manufacturing. The scalable solution combines fluid handling and automation to make transfection easier, faster, and more cost-effective than the state-of-the art solutions. Kytopen is integrating their platforms with commercial automated liquid handling systems for processing small volumes and has developed a large volume platform for therapeutic applications. These non-viral solutions will accelerate the time-to-market of next-generation cell and gene therapies by enabling discovery of therapeutic targets and reducing the cell therapy manufacturing costs. Kytopen recently released a beta version of their large scale manufacturing system and is actively engaging with potential partners.

Lisa Stehno-Bittel, Ph.D., Founder and President
Kansas City, MO
Likarda is a biotech company developing cell therapies and delivery systems for pets and people. Likarda’s cell delivery platform enables better encapsulation of cells for therapies, and can be made in durable or degradable formats. The company is able to create microencapsulants using slow-gelling, advanced, biocompatible hydrogels including PEG, hyaluronic acid, and various other materials. This is achieved in a safe, cyto-compatible, biocompatible manner that can be produced at scale. Likarda is developing this platform for use in diabetes, osteoarthritis, joint pain, and inflammation. Likarda’s platform will enable the next generation of cell therapies to succeed for both pets and people.

Jim Burns, Ph.D., CEO
San Diego, CA
Locanabio is the global leader in developing a new class of genetic medicines. The company’s unique and multi-dimensional approach uses gene therapy to deliver RNA binding protein-based systems to correct the message of disease-causing RNA and thereby change the lives of patients with devastating genetic diseases. These broad capabilities delivered via gene therapy enable Locanabio to potentially address a wide range of severe diseases with a single administration. The company is currently advancing programs in neurodegenerative, neuromuscular, and retinal diseases.

Fred Chereau, President and CEO
Lexington, MA
LogicBio is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly-owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome. LogicBio is also developing a next generation capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples.

Matthew Hewitt, Ph.D., Head of Clinical Development
Basel, Switzerland
In Lonza Pharma, Biotech & Nutrition, we work to improve health by supporting our customers to deliver innovative solutions to prevent, treat or even cure disease. The segment provides a wide range of services and products from early phase discovery to custom development and manufacturing of active pharmaceutical ingredients to innovative dosage forms for the pharma and consumer health and nutrition industries. Our extensive track record includes commercialization of pioneering therapies and manufacturing of a wide variety of biological and chemical drugs. We continuously invest to solve not just the current, but also the future challenges. Together, let’s bring your next medicine to life.

Sarah Meeks, Ph.D., VP of Business Development
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 leverages its Flow Electroporation® Technology to enable its biopharmaceutical partners to advance development of innovative medicines, particularly in cell therapy, including the use of gene editing tools in treatment of genetic diseases and immuno-oncology approaches to cancer treatments. MaxCyte has placed its cutting-edge instruments worldwide, with all of the top ten global biopharmaceutical companies, and has 140+ cell therapy programs and 100+ licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its biopharmaceutical industry partners to unlock the full potential of their products.

Ann Woolfrey, M.D., Medical Director
Cincinnati, OH
Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical, and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages regulatory and therapeutic expertise across all major areas including advanced therapies, oncology, cardiology, metabolic disease, endocrinology, central nervous system, and anti-viral and anti-infective. Headquartered in Cincinnati, Ohio, Medpace employs approximately 3,400 people across 39 countries as of September 30, 2020.

Jerry Keybl, Ph.D., Head of Cell and Gene Therapy
Burlington, MA
Cell and gene therapies hold the promise to change lives. Even as the path to patients accelerates, manufacturing and regulatory complexity remains. From solving your unique upstream and downstream challenges to meeting urgent manufacturing timelines and navigating uncertain regulatory guidelines, a knowledgeable partner can help move your cell and gene therapy from hype to hope. At MilliporeSigma, we give shape to cell and gene therapy development every day. With 30+ years of expertise, a global organization to integrate leading manufacturing technologies with process development, scale-up, safety testing, and regulatory experience, we can meet your therapy’s needs. We have more experience in this area than almost anyone else in the industry. We were the first gene therapy CDMO to produce a commercial product following a successful regulatory inspection. Our products and services include optimized manufacturing platforms, media, and reagents; manufacturing, biosafety, and characterization testing, as well as process development services.

Cynthia Bamdad, Ph.D., CEO
Waltham, MA
Minerva Biotechnologies discovered that the growth and pluripotency of naïve stem cells is mediated by the same growth factor receptor, MUC1*, and growth factor, NME7AB, that mediate growth and metastatic potential of cancer cells. The company elucidated the molecular mechanisms that limit stem cell self-replication but allow cancer cells to self-replicate indefinitely. Minerva learned how to override the pluripotency/differentiation switch to make naïve stem cell proliferate, without spontaneous differentiation, until the company triggers the switch. Conversely, Minerva has developed therapeutic agents that block MUC1* and others that block NME7AB, which block cancer growth. Minerva makes NME7AB-containing AlphaSTEM naïve stem cell media and a synthetic peptide that flips the switch to initiate differentiation. Minerva is currently in the clinic with a first-in-human trial for a CAR-T that targets MUC1* for the treatment of metastatic breast cancers. No therapeutic that targets MUC1* has ever been tested in humans.

Charlie Silver, Ph.D., CEO
San Francisco, CA
Mission Bio is a life sciences company that accelerates discoveries and cures in oncology by equipping researchers with the tools needed to improve how we measure and predict our resistance and response to advanced therapies. Mission Bio’s multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2012, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company’s Tapestri platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously, from the same sample at single-cell resolution. The Tapestri platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies around the world to develop treatments and eventually cures for diseases.

David Main, President and CEO
Toronto, Canada
Notch Therapeutics designs and manufactures therapeutic T-cells from pluripotent stem cells. The company has developed a proprietary platform with the potential to fully enable best-in-class commercial manufacturing of stem-cell derived T-cells. As current T-cell therapies are limited by the use of a patient’s own cells, Notch T-cells could replace existing standard-of-care therapies and pave a path to novel therapeutics to address unmet need. Built on foundational discoveries in developmental immunology and enabled by world-class bioengineering and advanced cell manufacturing teams, Notch Therapeutics is poised to be a leader in cell therapy for oncology and other diseases.

Leah Bloom, Ph.D., SVP, External Innovation and Strategic Alliances
Bannockburn, IL
Novartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are working to turn promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA) and continuing with our robust AAV-based pipeline, which includes treatments for Rett syndrome and Friedreich’s ataxia. We are powered by the world’s largest gene therapy manufacturing footprint, enabling us to bring gene therapy to patients around the world at quality and scale.

Jacob Sten Petersen, Ph.D., Corporate VP, Head of Stem Cell R&D
Copenhagen, Denmark
For almost 100 years, Novo Nordisk has been translating the unmet medical needs of people living with a serious chronic disease into innovative medicines and delivery systems. From our labs to our factory floors, we are discovering and developing innovative biological medicines and making them accessible to patients throughout the world. Our focus is on the serious chronic diseases that affect hundreds of millions of people and are among the most urgent global health challenges. By combining our innovation and commercial excellence, we draw upon insights from patients and partners to transform bold ideas into life-saving and preventive medicines. We make long-term investments in novel treatments and technologies, including curative stem cell-based therapies, to continually advance the development of medical devices and digital health solutions.

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

Justin Irizarry, CEO
South Miami, FL
OrganaBio democratizes access to critical, hard to procure resources to advance the clinical translation and commercialization of cell-based therapies. OrganaBio currently produces MSCs, NK, and T-cells from its proprietary supply chain of perinatal tissue and will soon offer adult peripheral blood mononuclear cells. OrganaBio’s products are supported by a robust QC, QA, and regulatory documentation package. OrganaBio partners can source GMP cells and manufacture their products in its novel multi-tenant cGMP facility, with access to the Quality and Manufacturing systems needed to do so. In addition, partners can leverage a host of services (process development, QA, QC, analytical testing and regulatory affairs support), eliminating the need to coordinate multiple vendors and service providers, and maintaining control over their process and product. OrganaBio’s unique end-to-end industry solution, positioned at the intersection of several critical resources, spurs rapid development of cell-based therapies, ultimately benefiting patients.

Jason Slingsby, Ph.D., Chief Business Officer
Oxford, UK
Oxford Biomedica (OXB) is a fully integrated gene and cell therapy company developing life changing treatments for serious diseases. OXB has built a sector leading lentiviral vector delivery platform (LentiVector®), which it leverages to develop in vivo and ex vivo products both in-house and with partners. OXB has created a portfolio of gene and cell therapy product candidates in oncology, ophthalmology, CNS disorders, liver diseases, and respiratory disease. OXB has also entered into a number of partnerships, including with Novartis, Bristol Myers Squibb, Sanofi, Sio Gene Therapies, Orchard Therapeutics, Beam Therapeutics, and Boehringer Ingelheim, through which it has long-term economic interests in other potential gene and cell therapy products. OXB has also signed a significant three-year master supply and development agreement with AstraZeneca for large-scale manufacture of the adeno based COVID-19 vaccine candidate, AZD1222.

Eric Halioua, President and CEO
Liege, Belgium
Founded in April 2014 as a spin-off of the French Blood Bank, PDC*line Pharma is a Belgian-French biotech company that is developing a novel class of off-the-shelf cancer vaccine based on a proprietary Plasmacytoid Dendritic Cell line (PDC*line) pre-loaded with peptides that are derived from target tumor antigens. Based on a robust preclinical package and a first-in-human Phase Ib feasibility study in melanoma, PDC*line Pharma is focusing on lung cancer with a new candidate in a Phase I/II clinical trial (PDC*lung) and neoantigens (PDC*Neo). PDC*line Pharma comprises a team of 25 highly skilled professionals based in Liège, Belgium and Grenoble, France) and has raised nearly €32M in equity and non-dilutive funding. An open-label, dose-escalation, Phase I/II trial with the cancer vaccine candidate (PDC*lung01) in 64 non-small cell lung cancer patients is currently ongoing in Belgium, France, and Germany.

Dirk Hondmann, Ph.D., CEO
Boston, MA
At PeterBio we develop innovative gene and cell therapies with a profound impact on people’s lives. We focus on gene-editing using our RITDM™ technology. Our RITDM™ technology is a new way of approaching gene-editing and is based on the insight that during normal cell division the DNA replication fork exposes short stretches of single stranded DNA to which complementary correction templates can bind. The cell’s own DNA repair processes can use these correction templates to generate daughter cells with the desired DNA change. A major benefit of our technology is that gene-editing can occur without the need to create DNA breaks, significantly reducing the risk of unintended changes in the genome. We make gene-editing safe enough that it can be used for a wide range of therapeutic applications.

Géraldine Guérin-Peyrou, Director of Marketing and Scientific Support
Strasbourg, France
Polyplus-transfection applies its 20 year expertise to the development of novel transfection solutions. Polyplus-transfection is the leading supplier of a key critical component for viral vector manufacturing for gene and cell therapy. We are proud to provide qualified and GMP-grade transfection reagents suitable from R&D to clinical trial and commercial scale, accompanied by strong scientific and regulatory support. In addition, we provide a range of effective transfection reagents to deliver most nucleic acids, including DNA and siRNA in vitro and in vivo.

Mark Clein, Co-Founder and CEO, Precision Medicine Group
Bethesda, MD
Since 2013, Precision’s interdisciplinary teams have been advancing clinical, manufacturing and commercialization solutions to help innovators transform patient lives. We know that delivering a cell or gene therapy to market requires a nuanced, comprehensive and integrated approach, scientific and manufacturing expertise, and the right partner. That’s precisely why we’ve launched Precision ADVANCE, a collection of interconnected services and complementary teams uniquely focus on the complexities of clinical, regulatory, manufacturing, and commercial needs to successfully bring cell or gene therapies to market. Learn more about how we accelerate clinical development with Precision for Medicine, optimize next-gen manufacturing with Project Farma and drive commercial success with Precision Value & Health.

Cindy Atwell, VP, Business Development
Durham, NC
Precision BioSciences is a clinical-stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform. ARCUS is a highly specific and versatile genome editing platform designed with therapeutic safety, delivery, and control in mind. Using ARCUS, Precision is developing allogeneic CAR-T for cancer and in vivo gene correction therapies for rare diseases.

Tony Khoury, EVP, Technical Services
Chicago, IL
Project Farma (PF) is a consulting firm providing biomanufacturing strategy and execution. Our services include turnkey capital projects, tech transfers, and facility builds; owner’s representation; program/project management; validation; quality, regulatory and compliance; engineering and automation; serialization track and trace; reliability, maintenance and asset management and more. We partner with startups, established life science corporations, advanced therapy organizations, universities, hospitals, government agencies, financial institutions, not-for-profits, and CROs/CMOs.

Karolis Rosickas, CEO
Sydney, Australia
Regeneus is a clinical-stage regenerative medicine company using stem cell technologies to develop a portfolio of novel cell-based therapies to address unmet medical needs in human health markets with a focus on neuropathic pain, including osteoarthritis and various skin conditions, with its platform technologies Progenza and Sygenus.

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

Olav Hellebo, CEO
Pencoed, UK
ReNeuron is a global leader in cell-based therapeutics, harnessing its unique stem cell technologies to develop off the shelf stem cell treatments, without the need for immunosuppressive drugs. The company’s lead cell therapy candidate is in clinical development for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that treat diseases of the brain. The company also has the ability through its conditionally immortalised induced pluripotent stem cell (iPSC) platform to make any tissue cells of choice; in-house programmes are focused on treatments for blood cancers and diabetes. ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L.

Atta Behfar, M.D., Ph.D., CEO
Rochester, MN
Rion is a clinical-stage biotech company that has established a platelet-based exosome therapeutic platform, call Purified Exosome Product (PEP), as the first-ever fully off-the-shelf regenerative medicine with widespread tissue healing application. Rion has developed PEP into a clinic-ready therapeutic that can be cGMP manufactured with high consistency, and demonstrated PEP’s safety, efficacy, and reproducibility in numerous preclinical animal studies across many types of tissue, including heart, muscle, skin, tendon, and nerve. Rion is working with research collaborators to establish efficacy in additional applications such as other cardiovascular diseases, women’s health, COPD, tendon repair, and nerve regeneration.

Shigeo Taniuchi, President and CEO
Osaka, Japan
Santen Pharmaceutical is one of the world’s leading specialist ophthalmic pharmaceutical companies, founded in Japan over a century ago and now serving patients all over the world. We’re the ophthalmology market leader in Japan. We’ve been active in the European market since 1994 and are enjoying strong growth in EMEA (Europe, Middle East, Africa) and worldwide. We’re passionate about improving eye health and vision for patients and making beneficial treatments accessible to patients and healthcare systems. With our sole focus being ophthalmology, we can assess, respond, and act quickly in this specialist field where we have deep knowledge and experience.

Juergen Heitmann, Ph.D., Chief Business Officer
Montpellier, France
Sensorion is a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat, and prevent within the field of hearing loss disorders.

Philip Toleikis, Ph.D., President and CEO
Ontario, Canada
Sernova is a clinical-stage company developing regenerative medicine therapeutic technologies using a medical device and immune protected therapeutic cells (i.e. human donor cells, corrected human cells, and stem cell-derived cells) to improve the treatment and quality of life of people with chronic metabolic diseases such as insulin-dependent diabetes, blood disorders including hemophilia, and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. Sernova’s Cell Pouch System including therapeutic cells has been shown to provide long-term safety and efficacy in preclinical models and has been proven to provide a biologically compatible environment for insulin-producing cells in humans. In ongoing assessments in a Phase I/II clinical trial, Sernova has shown persistent blood levels of c-peptide both during glucose tolerance tests and under fasting conditions, and efficacy measures that matter to patients, including reduction in HbA1c, hypoglycemic unaware events, and reduction in insulin use.

Rogerio Vivaldi, M.D., President and CEO
Cambridge, MA
At Sigilon, we are pioneering a new class of therapeutics and seeking to develop functional cures for patients with a wide range of chronic diseases. Our Shielded Living Therapeutics™ platform combines advanced cell engineering with innovations in biocompatible materials, potentially enabling our product candidates to produce a wide range of therapeutic molecules that may be missing or deficient in the body, including proteins, antibodies, and enzymes. Our platform technology was inspired by more than a decade of work at MIT by our academic cofounders, Bob Langer and Dan Anderson, and supported by Flagship Pioneering’s strategic vision. Together, this work paved the way for us to build a diverse pipeline spanning rare blood, lysosomal storage, and endocrine diseases.

Parag Meswani, Chief Commercial Officer
New York, NY
Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients. Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies. We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinson’s disease, which affects millions of patients globally. Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry, and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Stephane Boissel, CEO
Paris, France
SparingVision is a biotechnology company focused on the discovery and development of innovative therapies for the treatment of blinding inherited retinal diseases. SparingVision is developing SPVN06, a gene-independent treatment for retinitis pigmentosa, the most common inherited retinal degeneration. There is currently no treatment to treat all genetic forms of this rare retinal disease that leads to blindness and affects nearly two million people worldwide.

Robert Shaw, Executive Director
Gaithersburg, MD
The Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-Based Drug Discovery (SCB) looks to complement the current SDO processes for standards development by engaging regenerative medicine stakeholders to ensure that new or revised standards provide the greatest benefits to the broad regenerative medicine community. To keep up with the fast-paced growth of regenerative medicine and advance standards specific to the field, SCB; 1) Engages the broader community in the identification, prioritization, and advancement of potential standards to incorporate a range of perspectives and expertise; 2) Coordinates and communicates about standards activities across the regenerative medicine community to accelerate standards advancement; 3) Educates the community about available standards and their benefits, standards development processes, and standards implementation.

Robert Deans, Ph.D., Chief Scientific Officer
Redwood City, CA
Synthego is a genome engineering company that enables the acceleration of life science research and development in the pursuit of improved human health. The company leverages machine learning, automation, and gene editing to build platforms for science at scale. With its foundations in engineering disciplines, the company’s platform technologies vertically integrate proprietary hardware, software, bioinformatics, chemistries, and molecular biology to advance basic research, target validation, and clinical trials. By providing both commercial and academic researchers and therapeutic developers with unprecedented access to cutting-edge genome engineering, Synthego is at the forefront of innovation, accelerating the development of truly engineered biology.

Neal Goodwin, Ph.D., Chief Scientific Officer
Hollister, CA
Teknova is a partner to the biopharma sector that provides end-to-end solutions in the development of reagents and processes for a multitude of life sciences applications, including cell and gene therapy. We have a dedicated R&D laboratory that offers expertise from prototype product design to GMP manufacturing of media, buffers, and reagents for plasmid DNA production, fermentation, cell clone development, and bioreactor virus production scale-up. Our research services facility is ideally located at our worldwide headquarters near the San Francisco Bay area in Hollister, CA, USA.

Christopher Murphy, VP and General Manager of Viral Vector Services
Waltham, MA
Thermo Fisher Scientific provides industry-leading pharma services solutions for drug development, clinical trial logistics and commercial manufacturing to customers through our Patheon brand. With more than 55 locations around the world, we provide integrated, end-to-end capabilities across all phases of development, including API, biologics, viral vectors, cGMP plasmids, formulation, clinical trials solutions, logistics services and commercial manufacturing and packaging. We give pharma and biotech companies of all sizes instant access to a global network of facilities and technical experts across the Americas, Europe, Asia and Australia. Our global leadership is built on a reputation for scientific and technical excellence. We offer integrated drug development and clinical services tailored to fit your drug development journey through our Quick to CareTM program. As a leading pharma services provider, we deliver unrivaled quality, reliability and compliance. Together with our customers, we’re rapidly turning pharmaceutical possibilities into realities.

Sam Wadsworth, Ph.D., Chief Scientific Officer
Novato, CA
Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

Jonathan Garen, Chief Business Officer
Amsterdam, The Netherlands
uniQure is delivering on the promise of gene therapy — single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease, and other severe genetic diseases. We are advancing a focused pipeline of innovative gene therapies. We are currently conducting a pivotal Phase III trial in our lead indication, hemophilia B, and have initiated a Phase I/II trial 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.

Tania Pereira Chilima, Deputy Chief Technology Officer
Woluwe-Saint-Pierre, Belgium
Univercells Technologies is a global provider of innovative biomanufacturing technologies to achieve cost-effective viral production from R&D to commercial scales. The company offers a comprehensive technology portfolio leveraging the strengths of process intensification and chaining as a direct answer to the growing demand of viral vectors and viral vaccines. Univercells Technologies is committed to helping customers increase performance with minimized footprint and costs today, while anticipating the needs of tomorrow. Building upon years of expertise and capitalizing on technology vetted by world leaders, Univercells Technologies was incorporated in Belgium in 2020 with the support of the Univercells group.

Petter Bjorquist, Ph.D., CEO
Gothenburg, Sweden
VERIGRAFT AB is a Swedish biotechnology company with a unique, breakthrough technology in the field of advanced therapies and regenerative medicine. We make transplantation possible without the severe risks of immunosuppression, and develop advanced therapies and tissue engineered products that will be able to help millions of patients with serious diseases. Our first product P-TEV is a human vein segment graft for use in transplantation to replace a defective or missing part of a patient’s vein. A P-TEV segment with functioning valves is implanted to replace a non-functioning venous valve in the femoral vein of a patient suffering from severe chronic venous insufficiency (CVI). CVI is a multi-billion USD market and these patients do not receive any curative treatment today.

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

Jean-Simon Diallo, Ph.D., CEO and Scientific Founder
Ottawa, Canada
Virica Biotech is solving many of the development challenges facing viral medicine developers that are caused by the natural defenses that host cells mount against viruses. These challenges range from affecting how the products are manufactured to how they are used to treat patients. Our core Viral Sensitizer (VSE) technology is a versatile additive small molecule platform that fundamentally alters how cells defend themselves against viral infection. Customized VSE formulations optimize research processes and enhance production. VSEs help bring your treatments to patients on an accelerated timeline.

Natalia Elizalde, Ph.D., Business Development Director
San Sebastian, Spain
VIVEbiotech is a GMP Contract Development and Manufacturing Organization (CDMO) fully specialized in the bioprocess development (from very early stages) to GMP manufacture of lentiviral vectors. The company addresses key aspects such as regulatory compliance, scalability, and cost effectiveness from the beginning to enhance project success in subsequent phases. VIVEbiotech is working in compliance with both FDA and EMA regulations and our customers are located in Europe, U.S., and Asia. Our lentivectors are being used for different applications such as rare/ultra-rare diseases and immune-cell therapy for cancer (CAR-Ts, TCRs, CAR-NKs, TILs). VIVEbiotech is now immersed in an expansion plan that will allow manufacturing lentiviral vectors in a wide range of scales. VIVEbiotech has dedicated USP and DSP development and R&D departments which are continuously working on the improvement of the production process. Therefore, any found enhancements are directly transferred to our customers´ projects.

Brian Mullan, Ph.D., Head, Innovation, Analytical and Process Development
Corbeil-Essonnes, France
Yposkesi is a leading Contract Development and Manufacturing Organization (CDMO) for gene therapy vector manufacturing. Created in November 2016 in Corbeil-Essonnes, France by AFM-Telethon and the SPI fund managed by Bpifrance, Yposkesi provides integrated services covering bioprocess development (USP and DSP) from small/pilot to large-scale production, analytical development, GMP manufacturing of lentiviral and AAV vectors, and regulatory support. Our current facility consists of a 50,000f2 (approx. 5,000m2) building, operating multiple production suites for bulk drug substance manufacturing and fill and finish. By 2022, Yposkesi will increase its global footprint to 100,000ft2 (approx. 10,000m2) with the addition of a second large-scale facility designed for EMA and FDA compliance. Capitalizing on the more than 25 years of expertise of Genethon, Yposkesi invests significantly in innovation in bioprocessing to deliver on high quality projects, cost effectively. Yposkesi has also entered into several strategic partnerships with numerous biotech and pharmaceutical companies, at various stages of product development.

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