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.

Overcome Regulatory, Clinical, Manufacturing & Pricing Bottlenecks to Progress Safer, Efficacious, Accessible Rare Gene Therapies From Early Clinical Development Through to Approval

2022 has been a landmark year for the global gene therapy space. With August came the green light for bluebird bio’s Zynteglo in the US, the first FDA approval for a gene therapy in over three years. Hot off its heels we saw the accelerated approval of another bluebird bio product, Skysona. Overseas, July and August brought EMA approvals for PTC Therapeutics' Upstaza and BioMarin’s Roctavian, the first gene therapies for AADC deficiency and Haemophilia A respectively.

As the gene therapy field continues to break records, there are still significant challenges to overcome relating to safety, efficacy, and accessibility. The 6th Annual Gene Therapy for Rare Disorders 2023 Summit remains devoted to showcasing the top case studies and strategic learnings from the past year. With an expert speaking faculty devoted to bringing safer and more effective gene therapies to rare disease patients, key questions will be answered on how best the field can overcome regulatory, clinical, manufacturing and pricing bottlenecks to progress gene therapies into and through the clinic.

500+ leading experts from innovative biotechs, large pharma, academia and key service providers will be reuniting in Boston for 2023 to capitalize on recent success stories and collaborate over the most pressing industry challenges. An event vital to unlocking the full potential of your rare gene therapy program, join us to keep your finger on the pulse and set up for success in 2023.

Develop, Validate & Implement Cell-Based & Biophysical Analytical Methods to Guarantee Safe, High Quality, Consistent & Efficacious Gene Therapy Products at Scale

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. As the field strives to strike a delicate balance between safety and efficacy, in the context of increased regulatory scrutiny and safety challenges, attending the 4th Annual Gene Therapy Analytical Development as an analytical scientist has never been so important.

This year’s summit returns in-person to Boston to reunite 300+ analytical experts in innovative biotech, pharma and academia to continue to develop resilient, long-lasting and robust analytical tools to enhance the safety, quality and efficacy of gene therapy products.

Whether you are focusing on specific characterization methods, enhancing your genome sequencing, advancing your understanding of full and partial particles, or advancing your early-stage bioassays, with 4 tracks, 8 pre-conference workshops and a post-conference focus day, the 4th Gene Therapy Analytical Development Summit will encompass all aspects of analytical development, giving you the chance to address and overcome challenges.

If you work in quality control, quality assurance, or process development - we’ve listened and we’ve answered. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Talks include enhancing the knowledge transfer between departments, bridging between analytical methods with regards to QC/PD, and enhancing in-process development support.

Whether you're working with AAV, non-viral vectors or lentiviral vectors, this is your opportunity to enhance your existing analytical methods and explore innovative new tools to support safe and effective gene therapy development.