BioPharma, Pharma

FDA green-lights CRISPR Therapeutics, Vertex Pharmaceuticals’ gene-editing study

The agency also accepted the two companies’ IND to begin the Phase I/II study of CTX001 in patients with sickle cell disease, sending CRISPR shares up more than 11 percent Thursday morning.

Shares of a Swiss biotechnology firm focused on gene editing were up Thursday as the Food and Drug Administration lifted a hold on a clinical trial it was running with a US drugmaker.

Zug, Switzerland-based CRISPR Therapeutics and Vertex Pharmaceuticals said Wednesday that the FDA had lifted the clinical hold on a Phase I/II study of its lead candidate, CTX001, in sickle cell disease, which it was to run under a partnership with Boston-based Vertex. The agency also accepted the Investigational New Drug application for the companies to start the trial. Shares of CRISPR, which trades on the Nasdaq under the ticker symbol CRSP, were up more than 11 percent at the start of trading Thursday morning. Vertex’s shares, already down over the past couple of days, appeared unaffected.

The FDA had slapped the hold on the study in May when the companies filed an Investigational New Drug application to initiate it. The hold was placed “pending the resolution of certain questions that will be provided by the FDA as part of its review” of the IND, though it did not affect a planned Phase I/II study of CTX001 in beta-thalassemia that remains on track for initiation in the second half of this year. In addition to the FDA’s acceptance of their IND, CRISPR and Vertex said regulatory authorities in multiple countries outside the US had also accepted their applications to run clinical trials of CTX001 in SCD and beta-thalassemia. A Phase I/II study of transfusion-dependent beta-thalassemia patients is currently enrolling in Europe.

With the therapy, SCD and beta-thalassemia patients’ cells are engineered outside the body to produce high levels of fetal hemoglobin, or HbF, in red blood cells, thereby alleviating blood transfusion burden for beta-thalassemia patients and sickle crises in SCD patients.

Last month, the US Court of Appeals for the Federal Circuit ruled that the Broad Institute’s patent on CRISPR-Cas9 gene-editing technology did not interfere with the University of California Berkeley’s patent application for its own version of the technology, a win for the Broad. CRISPR Therapeutics – along with gene-editing companies Intellia Therapeutics and Caribou Biosciences – in-licesed Berkeley’s technology.

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