Thumbnail for CGTP Fall Summit 2024

CGTP Fall Summit 2024

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  • Members

  • Thumbnail for CASSS Welcome & CGTP Fall Virtual Summit 2024 Introduction
    Date
    November 19, 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. 

    Session Chair

  • Thumbnail for Session I - Mastering the Art of Control: CQAs and Considerations for Control Strategies for AAV-based Therapeutics & Session I - Panel Discussion - Questions & Answers
    Date
    November 19, 2024

    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)


  • Thumbnail for Session II - Development and Manufacturing Strategies Enabling the Commercialization of AAV Therapies & Session II: Panel Discussion - Questions & Answers
    Date
    November 19, 2024

    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.

  • Thumbnail for CGTP Fall Virtual Summit 2024 Closing Remarks
    Date
    November 19, 2024

    Summit Chair: JR Dobbins, Eli Lilly and Company

    Session Chair