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Vertex (VRTX) Ups Investment in CRISPR Gene Therapy Partnership

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Vertex Pharmaceuticals Incorporated (VRTX - Free Report) announced that it has increased investment in its existing collaboration agreement with CRISPR Therapeutics AG (CRSP - Free Report) for the manufacture and commercialization of their CRISPR/Cas9 gene-editing therapy, CTX001. The candidate is being developed as a potential treatment for sickle cell disease (“SCD”) and transfusion-dependent beta-thalassemia (“TDT”). Under the amended deal, Vertex will lead the global development and future commercialization of CTX001, which it believes has significant commercial opportunity.

Per the latest amendment, Vertex will bear 60% of the cost for the global development, manufacturing and commercialization of CTX001 and is entitled to receive 60% of profits from future net sales of the same. This represents a 10% increase compared with the previous agreement. Meanwhile, CRISPR Therapeutics will take care of the remaining 40% of costs for CTX001 and will subsequently receive 40% of profits from net sales of the product, if approved.

For the increased investment, CRISPR Therapeutics will receive an upfront payment of $900 million from Vertex and will also be entitled to a potential $200 million upon the first regulatory approval of CTX001.

CRISPR Therapeutics’ stock was up 5.8% following the announcement of the news on Tuesday.

The above transaction is subject to customary closing conditions including clearance under the Hart-Scott Rodino Antitrust Improvements Act.

Shares of Vertex have declined 6.8% so far this year, wider than the industry’s decrease of 3.6%.

price chart for VRTX


We remind investors that CRISPR Therapeutics and Vertex had entered into a strategic research collaboration in 2015 to co-develop and co-commercialize CTX001. Per this agreement, they equally shared all R&D costs and profits worldwide. However, with the latest amended agreement, CRISPR Therapeutics bears 40% cost and profits, while Vertex is responsible for 60% cost and profits related to the development of CTX001.

Enrollment and dosing are currently ongoing for both SCD and TDT studies on CTX001. In December 2020, the companies announced promising additional data on CTX001 which demonstrated a consistent and sustained response in treating patients with SCD and TDT.

Meanwhile, CTX001 has been granted Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug designations by the FDA for both TDT and SCD. The European Commission has granted Orphan Drug Designation to the gene therapy candidate for both indications.

Please note that, other than Vertex/CRISPR Therapeutics, companies such as Editas Medicine (EDIT - Free Report) , Intellia Therapeutics (NTLA - Free Report) and Beam Therapeutics, among others, are also using the CRISPR/Cas9 gene-editing technology to develop their respective candidates for addressing various ailments.

Zacks Rank

Vertex currently carries 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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