Session II - Development and Manufacturing Strategies Enabling the Commercialization of AAV Therapies & Session II: Panel Discussion - Questions & Answers

Session Chairs: JR Dobbins, Eli Lilly and Company, Darius Pillsbury, ValSource Inc., and Jennifer Wellman, Akouos, Inc.

Historically, the rapid advancement of AAV therapies through clinical development has resulted in the development of the manufacturing process to be on the critical path, potentially impacting the CMC readiness for commercializing these innovative therapies. Thus, it is imperative early in the development lifecycle to consider the commercialization strategy. As AAV products progress into late-stage development, programs are often presented with challenges related to comparability necessitated by process, scale or site changes, process performance qualification and stability strategies to support commercialization. This session will build upon the morning session by exploring effective process development and manufacturing strategies along with regulatory considerations to support the rapid and effective development and commercialization of AAV therapies.

Session Speakers:

The Journey Towards Commercialization

Scott Cross, Dark Horse Consulting

Late-Stage Tech Transfer and Comparability Considerations for AAV-based Gene Therapy: A Case Study

Shengjin Jin, Sarepta Therapeutics, Inc.

Navigating Complexities of Late-Stage Analytical Development for Ensuring Comparability of Manufacturing Process Change in a Mutation-Agnostic Gene Therapy

Samar Mohanty, Nanoscope Therapeutics, Inc.