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CMC Requirements for Cell and Gene Therapy

January 19, 2018  •  Webinar  •  Leslie Wolfe
Posted In Other News & Insights


After decades of setbacks, Cell and Gene Therapy (CGT) – one of the most talked-about innovations in the industry – has made major strides in recent months. Since August 2017, the FDA has approved three gene therapy products. Most recently, the Agency announced the approval of Luxturna, which corrects a defective gene in a rare form of congenital blindness. These most recently approved therapies – two of which treat cancer – are the first of their kind to receive approval from the FDA. With hundreds of Cell and Gene Therapy products now in clinical trials, the industry is expecting more approvals in this product class this year. For over 30 years, Cell and Gene Therapy was known as the “future” of the biopharma industry, but the field has rapidly advanced to the “here and now”.

With enthusiasm continuing to grow around these novel therapies, it is imperative to understand and assess the Chemistry, Manufacturing and Controls (CMC) factors required for manufacturing and development of commercially viable CGT products.

This webinar provides expert insight on:

  • The basics of Cell and Gene Therapy including definitions and differences between the two therapies, Ex Vivo and In Vivo methodologies, and FAQs about the CMC development of CGT products
  • Details and descriptions of vector and cell production methods, as well as quality and characterization strategies
  • The latest FDA thinking and Industry standards for mapping a successful development plan for your CGT products
  • The most common pitfalls and challenges in Cell and Gene Therapy, and important things to consider throughout the CMC and development processes

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About the Host

Dr. Leslie Wolfe has over 20 years of experience in the Cell and Gene Therapy field. Wolfe’s expertise allows her to provide guidance and input in analytical assays, process development, regulatory documentations with various Health Authorities, as well as strategies for sourcing, distribution and packaging. After completing her doctoral studies in Biochemistry at Boston University, Dr. Wolfe spent several years in academics before joining Genzyme Biosurgery (now part of Sanofi) in 1996. For the next 18 years, Dr. Wolfe worked on cell therapies and regenerative medicine (CTRM), rising to the position of Vice President, Technology Development CTRM before retiring from the company in 2014. She was part of the leadership team that established the organizational structure which delivered on three autologous cell therapies, coordinated manufacturing activities to support regulatory filings, oversaw the production of clinical trial materials, validation of manufacturing processes and distribution of cell therapy products globally. In 2007, Leslie’s team developed a unique, proprietary identity quality control assay for the culturing of chondrocytes which received the special recognition of a Cum Laude Award at the International Cartilage Repair Society meeting. In 2012, this project also received an award from Sanofi Industrial Affairs organization for Outstanding Innovation.

Wolfe received a B.S. in chemistry in 1984 and her PhD in biochemistry in 1990 from Boston University.

About The Biologics Consulting Expert Edge Webinar Series

The Expert Edge Webinar Series was created by Biologics Consulting in an effort to share the latest Agency guidance and best practices with leaders throughout the biopharmaceutical and medical device industries. Each interactive, web-based workshop in the series is hosted by an accomplished subject-matter expert and designed to provide participants with a pathway to success through some of the most complex regulatory and product development challenges in the Industry today.