Engage with experts in gene therapy to learn about important considerations, the necessity of a strong plan for plasmid at the early stages of candidate development, and potential obstacles. Our panel will discuss the importance of continuity and consistency from early discovery through the different stages of drug development in preparation for commercialization. Learn about the original GMP-Source® service, developed in collaboration with leading gene therapy researchers, and how this continues to enable early phase clinical research.

Key Takeaways

  • Importance of planning for plasmid in early stages of candidate development
  • Best practices for ensuring material continuity and consistency from discovery through commercialization
  • Evolution of GMP-Source® and phase-appropriate use of plasmid DNA

Featured Speakers

Cindy Biffert

Vice President, GMP Nucleic Acids Business Unit
Aldevron

Cynthia (Cindy) Biffert started her career with Aldevron in 2002 in upstream manufacturing, helping establish the company’s QC department as QC Manager, assisting in the creation of Aldevron’s proprietary GMP-Source® quality system, and in deploying the GMP service lines as a Director of Biomanufacturing. As Senior Director of Biomanufacturing, she helped launch neoGMP®, a service level allowing for personalized medicine. In July of 2020, Cindy was named Vice President, GMP Nucleic Acids Business Unit, where she focuses on growth strategy, client communication, and project execution. Cindy graduated with B.S. degrees in Biology and Chemistry from North Dakota State University, Fargo, N.D. Prior to joining Aldevron, she was employed in the microbiology laboratory at MeritCare, now Sanford, hospital, Fargo, N.D.

Barry Byrne, M.D, Ph.D.

Associate Chair of Pediatrics and Professor, University of Florida
Director, Powell Gene Therapy Center
Earl and Christy Powell University Chair in Genetics

Dr. Barry Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units.

The research team has been developing new therapies using AAV-mediated gene therapy to restore cardiac and skeletal muscle function in DMD, Friedreich’s ataxia, Pompe, and other inherited neuromuscular diseases. His group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing. The work is supported by several NIH and foundation awards.

Denis Phichith, PhD

Lead Cell Line Development & Molecular Engineering Group
Spark Therapeutics

Denis Phichith joined Spark Therapeutics in 2015, working within the Upstream team in the Process Development group. He has contributed to the successful BLA submission for Luxturna and approval in 2017. He is currently leading the Cell Line Development & Molecular Engineering group within the Technology Development & Operations Department.

Date:  Tuesday, September 7, 2021
Time:  11am ET / 8am PT
Duration:  1 Hour

Register now for this webinar