TOPIC: Gene Therapy 2.0: AAV beyond monogenic gene correction

OVERVIEW: Gene therapy using adeno-associated virus (AAV) as a vector has emerged as a novel therapeutic modality that has the potential to lead to substantial disease modification in many monogenic disorders, or perhaps even cures. The approach has been further boosted by the recent approval of two AAV-based gene therapies by the US FDA, and a multitude of initial public offerings and mergers and acquisitions in the space. However, the success of the AAV gene therapies has thus far been mainly confined to replacing monogenic defects in the retina, the liver, and the brain. In this talk, I will explore the current state of AAV gene therapy and its recent clinical and financial progress, as well as its potential applicability beyond simple gene correction and the importance of meta-analysis and evidence-based planning in gene therapy clinical trial design.

• HOST: Dr. James Boyle, Exec. Director of Venture Development, OCR

• MODERATOR: Roy S. Herbst, MD, PhD, Ensign Professor of Medicine, Professor of Pharmacology, Chief of Medical Oncology, Director, Thoracic Oncology Research Program Associate Director for Translational Research, Yale Comprehensive Cancer Center, Yale School of Medicine

• FEATURED SPEAKER: Dr. Dmitry ‘Dima’ Kuzmin MSc, Managing Partner of 4BIO Capital