Health and Healthcare

CRISPR Wins FDA Approval for Human Trials

Chris Lange

Published: October 11, 2018 12:05 pm

Last Updated: January 11, 2020 4:33 pm

CRISPR Therapeutics A.G. (NASDAQ: CRSP) shares made a handy gain on Thursday after the company announced a key U.S. Food and Drug Administration (FDA) decision concerning its collaboration with Vertex Pharmaceuticals Inc. (NASDAQ: VRTX).

The FDA announced that it has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD).

For some quick background: CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

In addition to the acceptance of the IND, CRISPR and Vertex previously announced that they had obtained approvals of clinical trial applications for CTX001 in multiple countries outside the United States for both β-thalassemia and SCD. The companies remain on track to initiate a Phase 1/2 clinical study in SCD by the end of 2018 and are currently enrolling patients with transfusion-dependent β-thalassemia in a Phase 1/2 trial in β-thalassemia in Europe.

Excluding Thursday’s move, CRISPR has outperformed the broad markets, with the stock up 88% in the past 52 weeks. In just 2018 alone, the stock is up 56%.

Shares of CRISPR were last seen up about 4% on the day to $38.06, with a consensus analyst price target of $67.69 and a 52-week trading range of $16.51 to $73.90.

Vertex traded down 2% at $172.74 a share. The consensus price target is $198.27, and the 52-week range is $136.50 to $194.92.