What CRISPR Has Planned for Transfusion-Dependent Beta Thalassemia

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When CRISPR Therapeutics A.G. (NASDAQ: CRSP) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of transfusion-dependent beta thalassemia, its shares jumped on Tuesday.

Note that CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR and Vertex Pharmaceuticals Inc. (NASDAQ: VRTX).

In February 2019, CRISPR and Vertex announced that the first patient had been treated with CTX001 in a Phase 1/2 clinical study of patients with transfusion-dependent beta thalassemia, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial.

The companies are also evaluating CTX001 for the treatment of sickle cell disease (SCD) and received Fast Track Designation for CTX001 from the FDA in January 2019 for that use. The companies announced in February 2019 that the first patient had been enrolled in a Phase 1/2 clinical study of CTX001 in severe SCD in the United States and is expected to be infused with CTX001 in mid-2019. Enrollment in both studies is ongoing.

For some quick background: CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).

Shares of CRISPR were last seen up about 5% at $38.92, in a 52-week range of $22.22 to $73.90. The stock has a consensus analyst target of $55.94.

Vertex was trading at $182.00 per share. The consensus price target is $208.58, and the 52-week trading range is $144.07 to $195.81.


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