Welcome to the Optimizing AAV Safety Summit

Join the Optimizing AAV Safety Summit to join 30+ senior leaders to discuss key challenges with 15+ detailed case studies and 3 interactive panel discussions ensuring key learnings and insights to optimize safety studies and de-risk development to apply in your gene therapy development programs going forward. Address the key industry challenges at the interface of enhanced gene therapy safety across 3-days of unrivaled presentations, including:



De-risking the Nonclinical Safety Package for Vector Delivered Gene Therapies

Explore current perspectives on safety risks for gene therapies, uncover approaches and strategies to de-risk gene therapy development with case-studies from Sana Biotechnology


Animal Models & AAV Toxicity: Lessons learned from the Collaborative Cross

Assess a novel animal model for toxicity, evaluate AAV biology via genetic diversity and delve into the immune comparability between humans and mice with insights from AskBio


Preclinical Studies in Large Animal Models Provide Insights into AAV Integration and the Potential for Genotoxicity

Review the risk of AAV integration and clonal expansion by AAVs with a dedicated case study showcasing a long term animal model treated with an AAV gene therapy with insights from The University of Pennsylvania


Complement Activation and Thrombotic Microangiopathy Associated with AAV Gene Therapy

Delve into the complement system and associated complementopathies including TMA, and evaluate the surveillance, diagnosis, and management of TMA with real-life case studies from Aspa Therapeutics

"Gene therapy is still a nascent field and we as a community of patients, drug developers, providers, academics, and regulators must come together and share learnings, share data, share successes, and failures so that we can design safer and more effective drugs and get them to patients faster."

Eric D

Eric David
Chief Executive Officer