Vertex Pharmaceuticals to buy US firm Exonics Therapeutics

Vertex Pharmaceuticals has signed an agreement to acquire US-based Exonics Therapeutics in a deal valued up to $1bn (£787m).

Under the deal, Exonics equity holders will receive $245m (£192m) upfront payment from Vertex, as well as potential future payments based on the achievement of specified development and regulatory milestones for the Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1) programmes.

Based in Watertown of Massachusetts, Exonics Therapeutics is involved in the development of gene editing therapies for the treatment of patients with DMD and other severe genetic neuromuscular diseases.

Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin in multiple small and large animal DMD preclinical models.

Exonics’ technology, which is licensed from UT Southwestern Medical Center, is based on the research of Dr. Olson.

Subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions, the deal is expected to be completed in the third quarter of this year.

Vertex chairman, president and CEO Dr Jeffrey Leiden said: “Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform for DMD and DM1.”

Vertex has also expanded its collaboration with CRISPR Therapeutics to discover and develop gene editing therapies for the treatment of DMD and DM1.

As per terms of the deal, Vertex will pay $175m (£137m) upfront for the exclusive worldwide rights to CRISPR Therapeutics’ existing and future intellectual property, including foundational CRISPR/Cas9 technology, novel endonucleases, single and double cut guide RNAs, and AAV vectors for DMD and DM1 gene editing products.

Vertex will take responsibility for all other research, development, manufacturing, and commercialisation costs.

CRISPR Therapeutics will secure payments of up to $1bn (£787m) , including the upfront and potential future payments, based on the achievement of specified research, development, regulatory, and commercial milestones for the DMD and DM1 programmes.

Vertex will also pay tiered royalties on future net sales on any products, which may result from this collaboration.

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