Rare Daily Staff

On the heels of reporting preliminary positive results for its gene-editing therapy in two rare blood disorders, CRISPR Therapeutics initiated a $275 million capital raise by offering 4.25 million shares at $64.50 per share.

CRISPR and development partner Vertex Pharmaceuticals reported positive results from the first two patients with severe blood disorders treated with CRISPR’s experimental gene-editing therapy CTX001 in two ongoing phase 1/2 clinical trials that are designed to assess the safety and efficacy of a single dose of CTX001 in adults with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD).

TDT and SCD are genetic diseases with TDT reducing the production of hemoglobin and leading to the need for chronic blood transfusions, and SCD causing misshapen blood cells that become sticky and lead to painful vaso-occlusive crises (VOCs) and potential serious acute and chronic life-threatening complications and death.

One patient with TDT received the treatment in the first quarter of 2019 and remains transfusion independent after nine months, compared to requiring 16.5 transfusions per year over the two years before enrolling in the clinical study.

Another patient with severe SCD received the treatment in mid-2019 and was free of VOCs at four months, compared to experiencing seven VOCs per year over the past two years before enrolling in the clinical study.

Theses preliminary results are the first such data to be reported for patients with TDT and SCD treated with

CTX001 is an experimental ex vivo CRISPR gene-edited therapy in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. It is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex that began in 2015 and focuses on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 is the first treatment to emerge from the joint research program.

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