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Vertex (VRTX) Inks New Deal for CRISPR Gene-Editing Rights

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that it has inked a new collaboration agreement with its existing partner, Arbor Biotechnologies, to develop ex vivo engineered cell therapies using the latter’s proprietary CRISPR gene-editing technology, targeting select serious diseases.

Please note that Vertex had previously entered into a collaboration with Arbor for discovery of novel proteins to support development of new gene-editing therapies in 2018. The company’s recent collaboration with Arbor will provide it with a new toolkit in cell and genetic therapies to develop cell therapies for the treatment of multiple serious diseases.

Per the recent agreement, Arbor is eligible to receive up to $1.2 billion from Vertex in potential payments related to specified research, development, regulatory and commercial milestones across up to seven potential programs. Vertex plans to research and develop ex vivo engineered cell therapies to support its goal of fully differentiated treatment of type I diabetes, next-generation approaches in sickle cell disease (SCD) and beta thalassemia, and certain other diseases.

Shares of Vertex have lost 15.5% so far this year against the industry’s increase of 1.2%.

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While Vertex’s main focus is on the development and strengthening of its key franchise of cystic fibrosis (CF) drugs, the company also has a rapidly advancing early-stage portfolio in five additional diseases beyond CF. The company has entered into multiple collaboration agreements with different companies, including Moderna (MRNA - Free Report) , CRISPR Therapeutics (CRSP - Free Report) and Kymera Therapeutics (KYMR - Free Report) , to enhance its pipeline and/or its research capabilities.

Apart from CF, Vertex’s most advanced program is CTX001, a CRISPR/Cas9-based gene editing therapy, which is being co-developed in partnership with CRISPR Therapeutics for two devastating diseases — SCD and transfusion-dependent beta thalassemia. The gene-editing therapy candidate demonstrated a consistent and sustained response to treatment in the given patient populations.

The company is also developing two mid-stage candidates — VX-147 and VX-548 — as potential treatment for APOL1-mediated focal segmental glomerulosclerosis and acute pain following bunionectomy surgery, respectively. It is also developing its pipeline candidate, VX-880, as potential treatment for type I diabetes.

Zacks Rank

Vertex currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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