[R-sig-genetics] Developing Gene Therapies - A Statistician's Guide (free seminar)

[Michael Zyphur]

Hi everyone

Instats is excited to offer a free seminar on Developing Gene Therapies: A Statistician's Guide <https://instats.org/seminar/developing-gene-therapies-a-statistician>, livestreaming August 19 and led by Avery McIntosh from Pfizer and Alex Sverdlov from Novartis. Gene therapy is transforming modern medicine with recent landmark approvals for sickle cell anemia, spinal muscular atrophy, and multiple oncology indications, yet its one-time, potentially curative nature introduces statistical challenges that traditional drug-development frameworks cannot address. This seminar provides a practical roadmap for navigating the entire GTx lifecycle—from non-clinical translation and biodistribution to condensed clinical phases, immunogenicity considerations, and long-term follow-up—while showcasing innovative methodologies for dose-finding with ultra-small sample sizes, Bayesian safety monitoring, and adaptive platform trials. Participants will emerge able to articulate key regulatory pathways such as RMAT and PRIME, anticipate safety and efficacy pitfalls unique to AAV vectors, and design robust studies when a single pivotal trial may enroll fewer than 20 patients. Whether you are a PhD student, academic researcher, or industry scientist, this free session delivers the statistical tools and conceptual frameworks essential for contributing to the rapidly expanding pipeline of more than 2,000 gene-therapy products in development. 

Sign up today <https://instats.org/seminar/developing-gene-therapies-a-statistician> to secure your spot, and feel free to share this opportunity with anyone who might benefit!


Best wishes

Michael Zyphur
Professor and Director
Institute for Statistical and Data Science
https://instats.org
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