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Gene Therapy for Rare Disorders 2019


March 26–28, 2019

Gene Therapy for Rare Disorders 2019 is dedicated solely to tackling late-stage regulatory, reimbursement, clinical and manufacturing challenges; this meeting is your comprehensive guide to gene therapy commercialization.

50+ expert speakers will share their insights including:

  • The FDA and EMA discussing the latest guidance documents for product developers and potential applications of gene therapies to broader populations, enabling you to gain first-hand insights on the regulatory issues you’re grappling with
  • Spark Therapeutics sharing experiences gained in the pioneering Luxturna program to guide future progress in other tissue types, allowing you to benchmark against the unprecedented standards being established
  • How BioMarin have established scalable manufacturing processes, focused on producing high quality products, enabling you to understand the key constituents of long-term commercial scale gene therapy manufacturing
  • How Harvard Pilgrim Health are dealing with the unique challenges in balancing access and affordability of gene therapies, enabling you to gain a payer perspective on pricing expensive, one-time therapies
  • How REGENXBIO and 4D Molecular Therapeutics are pioneering the next generation of novel vector technologies, demonstrating the future potential of these therapeutics

We are delighted to be offering ARM members a 20% discount off the conference registration fee.

Enter code ‘ARM20′ when you register online