The Future of Cell-Based Therapies: Challenges and Opportunities in Making Gene Therapies More Widely Available
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The Future of Cell-Based Therapies: Challenges and Opportunities in Making Gene Therapies More Widely Available

December 13, 2017

8:00 a.m. PST / 11:00 a.m. EST / 16:00 GMT / 17:00 CET

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On August 30, 2017, Novartis made history when the company received approval from the U.S. Food & Drug Administration for tisagenlecleucel, a gene therapy for treatment of childhood leukemia. Seven weeks later, Kite Pharma followed, receiving FDA approval for axicabtagene ciloleucel, for non-Hodgkin lymphoma. It took three decades of research and clinical testing for the FDA to give its first-ever approvals for gene-therapy products.

Both tisagenlecleucel and axicabtagene ciloleucel are dramatically different from any drugs previously approved by FDA. Most notably, they are patient-specific drugs: T-cells from the leukemia patients' immune systems are harvested and genetically reprogrammed to express a chimeric antigen receptor (CAR) protein. The modified CAR T-cells are reinfused into patients to hunt their cancerous prey.

Radically different biologic drugs like tisagenlecleucel and axicabtagene ciloleucel require a radically different style of manufacture. This webinar will look at challenges facing researchers and product development teams, as this technology becomes more widely adopted as a treatment.

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Key Topics:


• Treatment areas where cell-based gene therapies will play a role

• Developing manufacturing programs with automated or semi-automated processing

• Managing manufacturing process changes and demonstrating comparability

• Selecting, qualifying, and managing contract service providers for cell and gene therapy

• Handling the challenges of variable raw materials

• The regulatory pathway for gene therapy products

• The future of off-the-shelf gene therapy treatments and barriers to overcome

Who Should Attend:


• Researchers

• Laboratory managers/directors/operators

• Heads of core facilities

• Managers at CMOs and CDMOs



Don Healey, Ph.D.,
Senior VP of Operations,
KBI Biopharma
Scott R. Burger, M.D.,
Principal of Advanced Cell and Gene Therapy


Jeffrey Lee,
Senior Editor
C&EN BrandLab

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