Dr. Raymond Miller, Head of Corporate Communications at GenScript, officially opened the forum with a keynote focused on the advancements and future of human cell therapeutics. His remarks highlighted significant progress in gene therapies for Type 1 diabetes, referencing FDA-approved treatments like Lantidra, and new approaches in clinical evaluation including Sana Biotechnologies new islet replacement therapy. The FDA has approved 42 gene and cell therapies to date, addressing various diseases. Investment in gene therapies reached $10.9 billion in the first half of 2024, signaling a promising future for the industry.
The featured speaker, Thomas Whitehead, President and Co-founder of the Emily Whitehead Foundation, delivered a powerful speech about his family's journey with CAR-T cell therapy. He shared the story of his daughter, Emily, who became the first pediatric patient to receive CAR-T cell therapy at the age of five after being diagnosed with acute lymphoblastic leukemia. Following 22 months of unsuccessful chemotherapy, Emily's CAR-T treatment led to remission in just 23 days, and she has been cancer-free for over 12 years. Now, the Emily Whitehead Foundation, along with other organizations, supports CAR-T therapy through various initiatives. They work to improve global cell therapy accessibility by sharing personal stories and advocating for better access to advanced therapies worldwide.
Dr. Carl June, Professor of Immunotherapy in the Department of Pathology and Laboratory Medicine at the Perelman School of Medicine of the University of Pennsylvania, detailed the evolution and advancements in CAR-T cell therapy. His discussion covered early trials in the late 1980s leading to FDA approval in 2017. Key milestones include the first patient treated in 2010, the development of efficient T-cell culture systems, and the use of lentivirus for gene transfer. Over 1,000 trials are ongoing, with significant progress in treating blood cancers. CAR-T cell therapy has treated about 50,000 patients so far. Dr. June then expanded on the future forms of CAR-T cell therapies including dual CARs and T-cell redirected cytokine killer (TRUCK) cells, which showed promising results in relapsed lymphoma and glioblastoma. He also discussed the potential expanded use of CAR-T cells in treating autoimmune diseases and additional cancers.
Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research at the Food and Drug Administration (FDA), discussed the agency's efforts to advance the development of gene therapy. His discussion focused on advancements in COVID therapy and gene therapy at the FDA. Key points included the approval of 22 CAR-T cells, with refined box warnings indicating a low risk of secondary T cell malignancies. Decentralized manufacturing of CAR-T cells is gaining traction, facilitated by automated systems. CRISPR's role in enabling multiple gene edits in allogeneic CAR-T cells was highlighted, potentially reducing costs and improving efficacy. The FDA is exploring accelerated approval pathways and international regulatory harmonization to expedite gene therapy development. Platform technologies and synthetic biology are seen as key to scaling gene therapy production and reducing costs, with a vision of addressing many rare diseases in the future.
Following the keynote speeches, GenScript's event featured multiple panel discussions that delved into the future of cell and gene therapy, biomanufacturing, and CAR-T development. In the first discussion, led by Dr. Vincent Xiang of 7G BioVentures, experts shared optimistic visions for 2025, highlighting global innovation, particularly from China and Japan, and the invaluable contribution of patient data to advancing therapy effectiveness. Despite technical and market challenges, panelists emphasized a steadfast commitment to making therapies safe and accessible and stressed the importance of communicating the transformative power of these innovations to the public and investors. The expert panelists included Alan Bash, Bin Li, Joshua Resnick, Caroline Stout, and Yan Zhu.
During the second panel discussion led by Dr. Matthew Pillar, Chief Editor of Bioprocess Online, experts dove deep into the future of biomanufacturing. They highlighted several key points: automation and robotics are set to revolutionize the manufacturing of advanced therapies, enhancing efficiency and precision; the promising potential of in vivo development of CAR-T cells, which could transform treatment paradigms; a strong emphasis on building robust infrastructure to expedite high-quality product delivery to patients while reducing costs; and the expanding reach of cellular therapies beyond cancer to tackle autoimmune diseases, necessitating innovation in manufacturing processes to meet rising demand. The panelists included Jason Bock, Jonathan H. Esensten, M.D., Ph.D., Rey (Reut) Mali, Hari Pujar, Michael Vreeland, MS, and Hing W.
The third panel discussion on CAR-T cell development were shared under the expert guidance of BioSpace's Lori Ellis. Industry leaders discussed how these therapies are reshaping the field of cell and gene therapy, addressing diseases at their source, and offering hope to many patients. They emphasized the critical role of innovation and creative problem-solving in overcoming current obstacles, and considered the potential expansion of CAR-T applications beyond cancer. The discussion also touched on bedside manufacturing—producing CAR-T cells on-site to improve patient care and outcomes—and the importance of fail-safe technologies to enhance treatment reliability. Throughout the conversation, the panelists underlined the necessity of a patient-centric approach, valuing the insights of patients, doctors, and payers as essential for the future development of cell therapies.
GenScript's successful 2025 JPM Global Forum brought together experts who shared critical insights and strategies to advance the fields of cell and gene therapies. GenScript looks forward to next year's event, anticipating strong interest and continued discussions on advancements in the industry.
About GenScript
Founded in 2002 in New Jersey, GenScript Biotech Corporation accelerates innovation in healthcare and consumer goods by providing researchers and companies with the building blocks needed to develop groundbreaking treatments and products. Guided by its mission to make people and nature healthier through biotechnology, and its role as a trusted global leader, GenScript has a team of over 5,000 employees and has served more than 200,000 customers across 100 countries. Learn more here.
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SOURCE GenScript Biotech Corporation
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