Forum Topic News
  • Conversation: UK firm Nightstar bags FDA fast-track status for gene therapy

    • June 15, 2018 12:56 PM BST
    • UK firm Nightstar bags FDA fast-track status for gene therapy

      A gene therapy for an inherited eye disease developed by Nightstar Therapeutics has picked up a coveted designation from the FDA that could accelerate its development.
      The RMAT (regenerative medicine advanced therapy) status has been given to NSR-REP1, the London-based biotech’s therapeutic for choroideremia, a rare, degenerative retinal disorder that mainly affects males and causes blindness.
      RMAT status is analogous to breakthrough status for drugs but applies to cell therapies and - since November 2017 - has been open to gene therapies. It combines the benefits of the breakthrough and fast-track programmes, which include early interaction with the regulator to help therapies that could make a big difference to patients move swiftly through development and review. There is currently no treatment for choroideremia, which typically leads to vision loss in early adulthood.
      Read more: http://www.pmlive.com/pharma_news/uk_firm_nightstar_bags_fda_fast-track_status_for_gene_therapy_1240048

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