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Vertex/CRISPR's Sickle Disease Candidate Put on Hold by FDA

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) and partner CRISPR Therapeutics AG (CRSP - Free Report) announced that the FDA has placed a clinical hold on the companies’ investigational new drug (IND) application for CTX001. The companies had filed the IND application last month to start a phase I/II study on CTX00 in sickle cell disease in the United States.

As part of the review of the IND, the FDA will put forward certain questions to the companies. Vertex and CRISPR Therapeutics plan to work rapidly with the FDA to resolve the issue so that the clinical hold is lifted and the study initiated.

In December, as part of a strategic research collaboration formed in 2015, Vertex and CRISPR Therapeutics selected CTX001 to move into clinical development as a gene edited treatment for sickle cell disease and β-thalassemia developed using CRISPR Therapeutics’ proprietary CRISPR/Cas9 technology. Back then, the companies had opted toco-develop and co-commercialize CTX001 and equally share all R&D costs and profits worldwide. Vertex has rights to license up to six new gene editing treatments (including CTX001) developed using the CRISPR/Cas9 technology that emerge from the joint research deal.

An application has also been submitted to begin a phase I/II study in β-thalassemia in 2018 in Europe. The plan to begin this study — in the second half of this year — is intact and unaffected by the hold on the sickle cell disease study.

Shares of CRISPR Therapeutics were down more than 14% in after-hours trading on Wednesday in response to the negative pipeline update while Vertex’s stock did not show any movement. In fact, so far this year CRISPR Therapeutics’ shares have gained 213.4% while that of Vertex are up 4.5% compared to the industry’s decline of 9.9%.

Genomic editing technology using CRISPR technology to repair defective genetic material that causes diseases is probably one of the most promising and exciting healthcare innovations in decades. Though there are several methods to use CRISPR to target genetic defects that cause specific diseases, the most promising is the use of an enzyme called Cas9 to deliver CRISPR to affected cells.

Other than CRISPR Therapeutics, Intellia Therapeutics (NTLA - Free Report) and Editas Medicine, Inc (EDIT - Free Report) also plan to carry out clinical studies using CRISPR Cas9 to address diseases.

Shares of both Intellia Therapeutics and Editas Medicine were down more than 3% in after-hours on Wednesday.

While CRISPR Therapeutics currently has a Zacks Rank #1 (Strong Buy), Vertex has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank stocks here.

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