CGTP Fall Summit 2024

Summit Chair: JR Dobbins, Eli Lilly and Company

Accelerating the Development of Adeno-Associated Viral Vector Gene Therapies: Innovations in Chemistry, Manufacturing, and Control 

Gene therapy using adeno-associated viral (AAV) vectors has emerged as a transformative approach for treating a variety of genetic conditions. Many of these novel therapies are being developed to treat rare diseases with unmet medical needs, necessitating the rapid advancement through development and commercialization. The inherent complexity of AAV vectors has historically presented challenges in advancing the chemistry, manufacturing, and control (CMC) aspects commensurate with the pace of clinical development.

This virtual summit will bring individuals from academia, industry, and health authorities together to discuss innovative CMC approaches. Attendees will gain insights into CMC strategies, challenges, and opportunities to accelerate the development and commercialization of AAV therapies. The summit will be comprised of two sessions that focus on development of the process and product control strategy along with commercialization topics such as technology transfer and process performance qualification strategies. Each session will consist of multiple presentations and a panel discussion providing attendees the opportunity to learn from and interact with experts in the field.  

By attending this summit, you will connect virtually with a relevant global network and gain tangible knowledge to help you more rapidly and robustly advance these transformative AAV vector therapies to patients who need them. 

CQAs are essential attributes that must be monitored and controlled to ensure product quality. Understanding and defining CQAs has been challenging in the relatively new field of gene therapy, but is crucial to ensure patient safety and to meet regulatory standards. We’ll explore phase-appropriate development of process controls and discuss strategies that can help accelerate commercialization of AAV gene therapies.

Session Chairs: Christiane Niederlaender, Parexel International, Leslie Nash, Health Canada, and Andrea Challand F. Hoffman La Roche Ltd

Session Speakers:


Release Quality Attributes of rAAV-Based Gene Therapy Medicinal Products

Laura Rodríguez, Spanish Agency of Medicines and Medical Products (AEMPS)


AAV Critical Quality Attributes: Comprehensive Analytical Control Strategies from Release to Characterization

Thomas Powers, Pfizer, Inc.


Control Strategies using CQAs for Method Changes and Comparability for Gene Therapies

Phillip Ramsey, Sangamo Therapeutics, Inc.


CQAs and Strategies to Accelerate the Development of Gene Therapies

Catherine Campbell, 4D Molecular Therapeutics

Additional Panelists:

Tania Rosen-Cheriyan, CBER, FDA

Francis Galaway, Medicines and Healthcare products Regulatory Agency (MHRA)

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.

Summit Chair: JR Dobbins, Eli Lilly and Company