Vertex, Crispr Unveil More Encouraging Data From Sickle Cell, Thalassemia Gene Therapy Trials

Vertex Pharmaceuticals Incorporated VRTX and Crispr Therapeutics AG CRSP have announced new data from Phase 1/2 clinical trials evaluating CTX001 in beta-thalassemia and sickle cell disease.
Presenting data from two trials at the European Hematology Association annual meeting, the companies announced that three-month follow-up data of 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001.
All 15 patients with transfusion-dependent beta-thalassemia did not need further blood transfusions, and all seven with severe sickle cell disease were pain-free.
One thalassemia patient experienced a cerebellar hemorrhage less than three months after being treated.
The serious side effect was related to the busulfan conditioning that gene therapy patients undergo before receiving treatment.
Today, VRTX announced to discontinue VX-864 in alpha-1 antitrypsin deficiency as the treatment was unlikely to provide any clinical benefit.
Price Action: CRSP shares are up 2.43% at $126.71, while VRTX shares are down 8.62% at $198.08 during the market session on the last check Friday.