Destination BLA, First Stop IND! Considerations for Designing Your Gene Therapy Preclinical and CMC Development Strategies

Recorded: June 2023

This webinar discusses the considerations to be taken into account when designing your gene therapy preclinical and CMC development strategies.  We shared our experience to help build successful strategies that carefully balance time and cost whilst always ensuring quality to meet regulatory needs.

Agenda

• Regulatory, Budgetary and Scientific Considerations for
  • designing preclinical studies for AAV-based gene therapy products
  • developing and validating a phase appropriate potency assay
    
• Designing the CMC development plan for viral vectors for successful IND submission

Moderated by:

Vatsala Naageshwaran, Ph.D., Vice President, Head of Business Development, US Lab Services and CGT, Pharmaron

Speakers

Key Opinion Leader: Ashley Winslow,Ph.D., President & Chief Scientific Officer, Odylia Therapeutics

Ashley Winslow is President and Chief Scientific Officer of Odylia Therapeutics, a nonprofit biotech focused on developing gene therapies for rare diseases. Odylia develops therapeutics in collaboration with patient groups and a strategic network of CROs, academic partners, and industry, with the aim of bringing life changing therapeutics to patients.

Ashley received her PhD in Medical Genetics from the University of Cambridge and completed her postdoctoral work at Massachusetts General Hospital and Harvard Medical School. Before joining Odylia, Ashley worked in the Precision Medicine and Human Genetics and Computational Biomedicine group at Pfizer and the Orphan Disease Center at the University of Pennsylvania. She has experience overseeing drug development from target discovery to early phase clinical programs and brings to Odylia more than 15 years of drug development experience in academic, industry, and non-profit sectors. Ashley serves on the International Angelman Syndrome Research Council (INSYNC-AS), CHAMP1 Scientific Advisory Board, and the RARE-X/ Global Genes Scientific Advisory Board.

Vibhor Gupta, Ph.D., Director – CGT Business Operations at Pharmaron

Vibhor Gupta, Ph.D. is the Director - Business Operations at Pharmaron, supporting Cell & Gene Therapy services since August 2018. Dr. Gupta completed his Ph.D. in Biochemistry and Oncology from Jawaharlal Nehru University, New Delhi-India in 2010 and moved to the United States in early 2011 to pursue his postdoctoral fellowship at the University of Pennsylvania-Philadelphia. While working at Greenberg laboratory, Dr. Gupta focused his research on Autoimmune diseases and inflammatory responses. In 2016, Dr. Gupta joined the Gene Therapy Program as a Senior Research Investigator and Regulatory Writer at University of Pennsylvania. Working closely with Prof. James M. Wilson, Dr. Gupta contributed to several Cell & Gene Therapy projects and assisted in drafting several INDs to the FDA. During his research career, Dr. Gupta has experienced numerous molecular biology techniques, developed biochemical assays, published more than ten manuscripts/review articles and presented his work worldwide in diverse fields of life sciences including Metabolism, Oncology, Autoimmune diseases, and Cell & Gene Therapy.

Linda Randall, Ph.D., Associate Vice President, Biologics Development, Manufacturing and Control at Pharmaron Gene Therapy

Linda and her team support their clients with the development and manufacture of gene therapy products. Linda established and grew the gene therapy capabilities at the Pharmaron UK site and since 2016 has been actively engaged in the CMC development of these products, with a career highlight leading the development and clinical supply of a CRISPR gene editing product for retinal disease. Linda is passionate about forming strong collaborative relationships to support the development of products and innovate in this field.  In addition to her 15 years in senior roles in the development of biologics, Linda has held senior roles in drug product formulation and pharma manufacturing.  She has a degree and PhD in Chemistry and is a Fellow of the Royal Society of Chemistry.