Vertex, CRISPR's Sickle Disease Drug Gets FDA's Fast Track Tag

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Vertex Pharmaceuticals IncorporatedVRTX and partner CRISPR Therapeutics AG CRSP announced that the FDA has assigned Fast Track designation to their gene editing candidate, CTX001, for the treatment of sickle cell disease (SCD), a severe hereditary form of anaemia.

The fast track status from the FDA is designed to provide certain benefits for new drugs that treat serious or life-threatening conditions and demonstrate potential to address the unmet medical needs. With this designation, the drug is expected to be granted a priority review once it files a new drug application (NDA).

Shares of Vertex and CRISPR Therapeutics were each up 5.5% and 8% on Friday in response to the news. In fact, in the past year, the CRISPR Therapeutics stock has rallied 16.1% while Vertex has increased 10.1% versus the industry's decline of 18.9%.

We remind investors that last May, the FDA had placed a clinical hold on the companies' investigational new drug (IND) application for CTX001. Both companies had filed the IND application in April to start a phase I/II study on CTX001 for SCD in the United States. The clinical hold was lifted in October. Vertex and CRISPR Therapeutics are presently enrolling in the phase I/II SCD study on CTX001 in the United States. Also, a phase I/II study of CTX001 for adult transfusion-dependent b-thalassemia is currently enrolling patients in Europe.

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, which is 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 emerged from the joint research deal.

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 seen in decades. Though there are several methods to use CRISPR for targeting genetic defects that cause specific diseases, the most promising is the use of an enzyme called Cas9 to deliver CRISPR for the affected cells.

Other than CRISPR Therapeutics, Intellia Therapeutics NTLA and Editas Medicine, Inc EDIT plan to carry out clinical studies using CRISPR Cas9 to cure diseases.

While CRISPR Therapeutics currently carries a Zacks Rank #2 (Buy), Vertex has a Zacks Rank #4 (Sell).You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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