Vertex Pharmaceuticals has made two deals, worth up to $1 billion each, to enhance its gene-editing capabilities in drug development. The company is expanding its collaboration with CRISPR Therapeutics, a gene-editing company developing gene-based medicines, and has agreed to acquire Exonics Therapeutics, a developer of gene-repair technologies for treating genetic neuromuscular diseases.

First deal: CRISPR Therapeutics

In the first deal, Vertex and CRISPR Therapeutics have expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene-editing therapies for the treatment of Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1), two genetic neuromuscular diseases.

Under the agreement, Vertex will pay $175 million 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 adeno-associated virus (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 Therapeutics will share research costs for specified guide RNA research to be conducted by CRISPR Therapeutics, 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 filing, CRISPR Therapeutics has the option to forego the DM1 milestones and royalties to co-develop and co-commercialize all DM1 products globally.

The closing of this transaction is subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. The companies anticipate the transaction will close in the third quarter of 2019.

Second deal: Exonics

In its second deal, Vertex has agreed to 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 primarily upon the successful achievement of specified development and regulatory milestones for the DMD and DM1 programs.

Exonics Therapeutics is developing gene-editing therapies to treat patients with DMD and other severe genetic neuromuscular diseases and will provide Vertex intellectual property, technology, and scientific expertise in gene-editing therapies for DMD and DM1. Exonics has used its proprietary technology, SingleCut CRISPR, to genetically repair and restore dystrophin, a key protein missing in children with DMD. Exonics’ technology is licensed from the University of Texas Southwestern Medical Center and is based on the research of Eric Olson, PhD, Exonics’ founder and chief science advisor, who will continue in his role as Exonics’ chief science advisor and provide oversight and guidance on the research and development of gene-editing therapies.

The closing of this transaction will be subject to certain conditions, including the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act and other customary conditions. The companies anticipate the acquisition will close in the third quarter of 2019.

Vertex: organic expansion

Following the moves, Vertex has named Dr. John T. Gray as Senior Vice President, Genetic Therapies. He previously served as Chief Scientific Officer and Senior Vice President at Audentes Therapeutics, where he and his team focused on rAAV gene therapy for neuromuscular and liver disorders.

Vertex also says it will establish a new genetic therapies research site in the Boston area where research programs, as well as vector development and clinical manufacturing for genetic therapies, including DMD and DM1 programs, will be conducted.

Source: Vertex