Gene Therapy Challenges and Best Practices: From Discovery to IND and Beyond

This workshop provides a comprehensive overview of AAV gene therapy, from foundational principles and the discovery of novel therapeutics using NanoCas and Zinc Finger Proteins, to critical nonclinical and clinical development considerations. Strategies for Bioanalytical method and companion diagnostic development, as well as genotoxicity and carcinogenicity concerns will be explored. In addition, the speakers will delve into the intricate CMC, Process development and scale up, analytical and Quality Control, and formulation challenges. The workshop will also cover essential regulatory aspects, including interactions with the FDA, ensuring a holistic understanding of bringing AAV-derived genomic medicines to patients.

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