About Event

Overcome Your Clinical & Commercial Challenges to Accelerate the Development of Your Gene Therapy for Blood Disorders to Market

Optimizing Clinical Trial Design to Demonstrate Durability to Regulators & Overcoming Pricing & Reimbursement Challenges to Ensure Patient Accessibility

With the recent news that the FDA has rejected BioMarin’s submission for approval, it is clear that demonstrating durability will pose a challenge to gene therapy for blood disorders developers. To support rapidly evolving gene editing and gene therapy pipelines, the 2nd Gene Therapy for Blood Disorders will provide a 3-day comprehensive conference specifically designed for industry experts focused on clinical development and commercial strategy. Explore non-viral delivery technologies, discover alternative patient conditioning regimes, hear patient perspectives, discuss pricing challenges and hear feedback on novel clinical trial tools with valuable insights from market leaders including Pfizer, UniQure, bluebird bio & CRISPR Therapeutics.

Join over 100 fellow industry experts from regulatory, clinical, commercial strategy and patient-focused backgrounds, from established companies in the blood disorders space as well as the new companies on the block, to benchmark yourself against your competitors and overcome your clinical and commercial challenges to accelerate the development of your gene therapy to market.