CRISPR Therapeutics
NEWS
This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.
The U.K.’s Medicines and Healthcare products Regulatory Agency on Thursday provided conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel.
As Vertex and CRISPR Therapeutics await the monumental potential approval of exa-cel, bluebird bio, Iovance and Rocket Pharmaceuticals wait patiently in the wings.
The regulator’s advisory committee on Tuesday found Vertex Pharmaceuticals and CRISPR Therapeutics’ off-target analysis for its sickle cell disease candidate exa-cel to be sufficient.
In a briefing document for Tuesday’s advisory committee meeting, the FDA raised concerns about the potential off-target effects of Vertex Pharmaceuticals and CRISPR Therapeutics’ investigational gene edited therapy.
After a series of milestone approvals in the first half of 2023, the FDA is slated to decide on four more firsts before the year’s end.
Data show the potential of Editas’ sickle cell disease and transfusion-dependent beta thalassemia gene therapy candidate, but it might not be enough to overtake Vertex and CRISPR Therapeutics.
The regulatory filing puts the company alongside Vertex and CRISPR, which also await FDA approval for their SCD gene therapy.
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