Beam Therapeutics reports positive trial results for its CRISPR sickle cell treatment
Beam Therapeutics announced that its CRISPR-based treatment for sickle cell disease successfully met the main goal in a key clinical trial. Patients achieved freedom from severe pain crises for at least 12 months, and the company plans to file for FDA approval by the end of this year.
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Beam Therapeutics revealed that its experimental CRISPR-based therapy for sickle cell disease, called risto-cel, achieved the primary goal in a pivotal clinical trial by keeping patients free from severe pain crises for at least a full year. Although detailed data have not yet been publicly disclosed, the company is moving forward quickly with regulatory plans. Beam stated it intends to submit an FDA marketing application for the therapy as early as the end of this year, marking a significant milestone for its gene-editing pipeline.
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