US FDA advisers to consider need for more studies for Vertex/CRISPR gene therapy (updated)

Crispr Therapeutics AG

27/10/2023 14:27

(Recasts paragraph 1 with details on FDA staff's request, adds
details about genomic alterations in paragraph 3)
       Oct 27 (Reuters) - The U.S. Food and Drug
Administration's staff reviewers on Friday asked the regulator's
advisory panel to consider the need for additional studies for
Vertex Pharmaceuticals  VRTX.O  and CRISPR Therapeutics'
 CRSP.BN  sickle cell disease gene therapy.
    The FDA's staff reviewers said it was not clear if the
limited donor cells used for assessment were adequate to
evaluate the potential safety risks of the therapy.
    The gene editing technology has the potential to produce
unintended genomic alterations, the staff said in the briefing
documents.
    The comments come ahead of a panel meeting of the agency's
outside advisers, who provide non-binding recommendations to the
FDA, slated on Tuesday to discuss the therapy. 
    Vertex and CRISPR Therapeutics are seeking the health
regulator's nod for the single-dose gene-editing cell therapy,
which is based on Nobel Prize-winning CRISPR technology, for the
inherited red-blood-cell disorder that causes the cells to
become sickle-shaped due to abnormal levels of hemoglobin in the
body.
    

 (Reporting by Sriparna Roy in Bengaluru; Editing by Shinjini
Ganguli)
 ((Sriparna.Roy@thomsonreuters.com;))