CRISPR Therapeutics
NEWS
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.
Currently, there are no gene editing–based treatments on the market, but the technology continues its march toward potential FDA approval, with several products in mid- and late-stage trials.
In a potential leap forward for sickle cell disease, Vertex moved closer to launching its one-time treatment for sickle cell disease and transfusion-dependent beta-thalassemia.
Partners CRISPR and ViaCyte (Vertex), and Genprex are pioneers in the quest to develop a gene therapy for Type 1 diabetes.
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