Rare Daily Staff

Vertex Pharmaceuticals said it is enhancing its gene editing capabilities to develop novel therapies for Duchenne muscular dystrophy (DMD) and myotonic dystrophy type 1 (DM1) by expanding its collaboration with CRISPR Therapeutics and acquiring Exonics Therapeutics.

Exonics Therapeutics is developing gene editing therapies to treat patients with DMD and other severe genetic neuromuscular diseases. The acquisition gives Vertex intellectual property, technology, and scientific expertise in gene editing therapies for these diseases. In multiple preclinical DMD models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with DMD.

Under the terms of the acquisition, Vertex will acquire all outstanding shares of Exonics, which will become a separate, wholly owned subsidiary of Vertex. Exonics equity holders are eligible to receive payments of approximately $1 billion, including $245 million upfront and potential future payments based upon the successful achievement of specified development and regulatory milestones for the DMD and DM1 programs.

The companies anticipate the acquisition will close in the third quarter of 2019.

Under the terms of the strategic collaboration and license agreement with CRISPR Therapeutics, Vertex will pay $175 million upfront for the exclusive worldwide rights to the company’s 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.

For the DMD program, Vertex is responsible for all research, development, manufacturing, and commercialization activities and all related costs. For the DM1 program, Vertex and CRISPR will share research costs for specified guide RNA research to be conducted by CRISPR, and Vertex is responsible for all other research, development, manufacturing, and commercialization costs.

CRISPR Therapeutics is eligible to receive payments of up to $1 billion inclusive of the upfront and potential future payments based upon the successful achievement of specified research, development, regulatory, and commercial milestones for the DMD and DM1 programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. At IND filing, CRISPR has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally.

 “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. These transactions are highly aligned with our strategy of investing in scientific innovation to create transformative medicines for people with serious diseases,” said Jeffrey Leiden, chairman, president and CEO of Vertex. “We are continuing to build a toolbox of small molecule and nucleic acid technologies and capabilities that will allow us to drive scientific innovation to produce transformative medicines for a broad portfolio of diseases.”

Photo: Jeffrey Leiden, chairman, president and CEO of Vertex

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