Crispr falls 12% most since 2020 as rival wins FDA backing for beta thalassemia drug
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Swiss gene-editing firm, Crispr Therapeutics (NASDAQ:CRSP) lost over 12% on Monday, its worst intraday decline since Oct. 2020, after a panel advising the FDA unanimously endorsed a gene therapy candidate developed by bluebird bio (BLUE) for Î²-thalassemia.

While the FDA advisory committees issue non-binding recommendations, the regulator usually follows them in a final decision.
On Saturday, Crispr (CRSP) and its U.S. partner Vertex Pharma (VRTX) shared data for exa-cel, a CRISPR/Cas9-based gene editing therapy the companies are developing for transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD).
Results indicated that 42 TDT patients out of 44 who received the experimental therapy were transfusion free with follow-ups ranging from 1.2 to 37.2 months.

The companies expect to submit regulatory filings for CTX001 in TDT and SCD in late 2022.