In Association with Phacilitate’s Cell and Gene Therapy World Forum
The promising early clinical data from gene therapies for rare diseases has seen a resurgence in investment and optimism in the field. This On Demand Webinar provides an opportunity to hear from three leading experts at the forefront of developing these potentially curative therapies:
- Dr Sven Kili, VP and Head of Cell & Gene Therapy Development, GSK, UK
- Mr Geoff MacKay, CEO, AVROBIO, USA
- Ms Sue Washer, CEO, AGTC, USA
They share their insights and experiences in navigating the commercialization path, including:
- What factors are unique to rare disease that need to be considered in terms of demonstrating clinical efficacy and selecting clinical endpoints?
- The different types of accelerated approval tracks available for rare and ultra rare diseases and what do you need to demonstrate in order to gain approval?
- Reimbursement price points and how rare disease companies can establish a viable business strategy to support different payment models?
- How feasible is it to drive down manufacturing costs at small scale for niche patient therapies?
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