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FDA Unveils Plausible Mechanism Framework to Accelerate Tailored Therapies for Ultra-Rare Diseases

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Source: fda.gov
FDA Unveils Plausible Mechanism Framework to Accelerate Tailored Therapies for Ultra-Rare Diseases
Photo: fda.gov
TL;DR

The FDA issued a draft guidance outlining a Plausible Mechanism Framework to accelerate approvals of genome-editing and RNA-based, individualized therapies that target the root causes of ultra-rare diseases, allowing smaller, robust studies and the use of master protocols. Sponsors must demonstrate the therapy addresses the specific disease mechanism, rely on well-characterized natural history data, and show target engagement or editing; public comments are due within 60 days.

Topics:businesshealth-policy#fda#genome-editing#health-policy#personalized-therapies#regulatory-guidance#ultra-rare-diseases
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  1. FDA Launches Framework for Accelerating Development of Individualized Therapies for Ultra-Rare Diseases  fda.gov
  2. The FDA creates a quicker path for gene therapies  NPR
  3. FDA illuminates new approval pathway for bespoke gene therapies  Fierce Biotech
  4. FDA proposes new treatment approval pathway for ultra-rare diseases  The Hill
  5. FDA proposes framework to speed rare disease gene therapy approvals  The Detroit News

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