Hi E,
Gene therapies are redefining the treatment of rare diseases. However, as the field transitions from academic promise to commercial reality, unique challenges must be overcome for these therapeutics to deliver on their potential.
The 4th Annual Gene Therapy for Rare Disorders (March 30 - April 2, 2020, Boston) is dedicated solely to tackling these late-stage manufacturing, clinical, regulatory and reimbursement challenges.
This is your comprehensive guide to realizing the commercial potential of gene therapies.
Access the official agenda to learn what the 80+ expert speakers will discuss, including how:
- The FDA and Chinese NMPA are establishing new opportunities to interact directly with regulators to meet their expectations, enabling you to gain first-hand insights into the global regulatory landscape
- Spark Therapeutics have implemented a comprehensive analytical comparability strategy, allowing you to bridge early and late stage development to satisfy the regulators
- BioMarin and bluebird bio are transitioning manufacturing to the commercial scale, providing you with insights into the key constituents of long-term, robust, commercial scale gene therapy manufacturing
- Harvard Pilgrim Health and Blue Cross Blue Shield of Massachusetts are balancing access and affordability for gene therapy products, enabling you to gain a payer perspective on the future of pricing expensive, one-time therapies
- NORD and AveXis are ensuring that the patient voice is embedded into every stage of clinical and commercial development, so youcan integrate patient input into clinical and commercial decision making to address unmet needs more effectively
- 4 In-Depth Tracks from CMC & Analytical to Commercialization
- 8 Interactive Workshops addressing key challenges from endpoint identification to value-based payment models
- Brand New Discussion Day exploring the realities of immunogenicity and re-dosing approaches
I hope to meet you in Boston next year.
Kind regards,
David
David Snowdon
Program Director
Hanson Wade