FAQs (Frequently Asked Questions)
What is the 8th Gene Therapy Development Summit?
The 8th Gene Therapy Development Summit is your chance to unlock the latest breakthroughs shaping the future of gene therapies across rare and common diseases. Gain insights from industry leaders, explore cutting-edge advancements in gene delivery and scalable manufacturing, and tackle critical regulatory challenges. With a focus on accelerating therapies to market, this summit equips you with the knowledge and connections to drive success in gene therapy development.
Who should attend?
This summit is ideal for decision-makers across the gene therapy value chain, including those in C-level, clinical development, regulatory affairs, manufacturing, and research. Whether you're a biotech leader, pharma executive, leading academic, or service provider, this event provides the insights, strategies, and connections to help accelerate gene therapy products from concept to clinic. It’s a must-attend for anyone shaping the future of gene therapy.
- Advancements in gene delivery technologies, including AAV and non-viral systems and it’s potential in precision medicine.
- The latest advances in gene editing technologies like CRISPR
- Strategies for scalable gene therapy manufacturing to meet demand.
- Capsid engineering and its impact on vector performance.
- Overcoming immune response challenges in gene therapy.
- Regulatory pathways and expedited approval processes for gene therapies.
- The role of biomarkers in clinical trials and therapeutic differentiation.
- Enhancing patient recruitment strategies in clinical trials.
- Overcoming challenges in gene therapy delivery to target tissues like the liver and brain.
What are some of the key topics that will be covered?
What are some of the diseases that will be covered?
90% of the content will continue to focus on rare and ultra rare diseases as this reflects the current focus of the field but the Common Disease Bootcamp will deep dive into the current expansion into more common diseases to increase patient reach.
Take advantage of a huge breadth across a wide range of common and rare diseases including leukemia, neurological disorders (e.g., epilepsy, Alzheimer's, Parkinson's), Crohn's disease, and sickle cell disease.
It will also explore gene therapy's potential for treating rare conditions like cystic fibrosis, Duchenne muscular dystrophy, retinitis pigmentosa, hemophilia, alpha-1 antitrypsin deficiency, glycogen storage disease 1a, canavan disease, wet AMD, Leber's congenital amaurosis, Charcot-Marie-Tooth disease, mucopolysaccharidosis type IIIA, and spinal muscular atrophy. Experts will discuss how to advance therapies for these diseases and address the unique challenges each presents.
The meeting brings together industry leaders across biopharma, leading academics, regulatory boards, investors, and service providers. Here’s a snapshot:
- Samir Koirala, Head of Genetic, Neurodevelopmental & Epilepsy Disorders at Biogen
- Sebastian Aguirre Kozlouski, Co-Founder and Vice President of Platform Development at Carbon Biosciences
- Sitra Tauscher-Wisniewski, Vice President of Clinical Development, Analytics & Gene Therapies at Novartis
- Peter Marks, Director of the Center for Biologics Evaluation and Research at the FDA
- Priya Chockalingam, Vice President, Head of Clinical Bioanalytics and Translational Sciences at Beam Therapeutics
- Niccolo Bacchi, Chief Executive Officer at Borea Therapeutics
- Morten Sogaard, President of Gene Therapy Research and Technical Operations at Astellas Innovation Management
- Terry Cothran, Senior Pharmacy Director at Oklahoma Health Care Authority
- Tynan Patrick, Manager of Gene Therapy Purification Operations at Pfizer
- Van Hoang, Head of Analytical and Quality Control at Spark Therapeutics
These leaders will share their expertise on advancing gene therapies for both rare and common diseases.
Who are the key speakers?
How do I register?
You can register for the summit by clicking the button and visiting our registration page or contacting us at [email protected]. Early registration discounts are available.