Nov 18, 2024 · For a high-level overview of this guidance document, please view this recorded webinar featuring Dr. Andrew Byrnes, Gene Transfer & Immunogenicity Branch Chief, Office of Gene Therapy CMC, Office...

Jul 1, 2021 · This is because clinical development of AAV gene therapy has outpaced CMC, manufacturing, and formulation development. This review discusses the various manufacturing steps and challenges encountered during AAV production and storage and provides a roadmap to improve the efficiency in manufacturing workflow and improve product shelf-life.

Oct 1, 2023 · Here we review how their successful use for antibody manufacture development is translating to AAV, but also how this will depend critically on improved analytical methods and adaptation of the tools as more understanding is gained on the critical attributes of AAV required for successful therapy.

Apr 17, 2008 · Recombinant adeno-associated virus (AAV)-based vectors expressing therapeutic gene products have shown great promise for human gene therapy. A major challenge for translation of promising...

CMC ASPECTS OF GENE THERAPY MEDICINAL PRODUCTS SME workshop: Focus on chemistry, manufacturing and controls (CMC) regulatory compliance for biopharmaceuticals and advanced therapies Presented by Matthias Renner on April 16, 2015 Division Medical Biotechnology, Paul- Ehrlich-Institut, Germany

Jun 1, 2024 · Points to consider for CMC, non-clinical and clinical aspects of AAV-based gene therapies are discussed. Specific challenges in gene therapy development and several policy recommendations are presented.

May 5, 2017 · We, FDA, are providing you, sponsors of human gene therapy Investigational New Drug Applications (INDs), recommendations regarding chemistry, manufacturing, and control (CMC) information...

Ways to balance early CMC investment and optimize resources, while still enabling an aggressive fast-to-licensure timeline to bring urgent therapies to patients, are also covered.

This framework shows how QbD can be applied to adeno-associated virus (AAV) gene therapy products and collects initial thoughts on where data gaps or interpretation uncertainties of the regulatory framework impede the chances of regulatory approval.

AAV vector manufacturing. Hitchcock et al., 2017. CGT Product Manufacturing is Complex www.fda.gov • There is often a need for significant in -process characterization.