10 June 2019 - - US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is enhancing its gene editing capabilities to develop novel therapies for Duchenne Muscular Dystrophy and Myotonic Dystrophy Type 1 by expanding its collaboration with Swiss gene editing company CRISPR Therapeutics and acquiring Exonics Therapeutics, the company said.
Vertex and CRISPR Therapeutics (NASDAQ: CRSP) have expanded their collaboration and entered into an exclusive licensing agreement to discover and develop gene editing therapies for the treatment of DMD and DM1.
Vertex and Exonics Therapeutics have entered into a definitive agreement under which Vertex will acquire privately held Exonics, a company focused on creating transformative gene editing therapies to repair mutations that cause DMD and other severe neuromuscular diseases.
Under the terms of this strategic collaboration and license agreement, Vertex will pay USD175 m 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.
For the DMD programme, Vertex is responsible for all research, development, manufacturing, and commercialization activities and all related costs.
For the DM1 programme, 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 USD 1bn 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-commercialise all DM1 products globally.
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. The companies anticipate the transaction will close in 3Q19.
Exonics Therapeutics is developing gene editing therapies to treat patients with DMD and other severe genetic neuromuscular diseases and brings to Vertex intellectual property, technology, and scientific expertise in gene editing therapies for these serious diseases.
In multiple small and large animal DMD preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with DMD.
Exonics' technology is licensed from UT Southwestern Medical Center and is based on the research of Dr. Olson. Dr. Olson will continue in his role as Exonics' chief science advisor and provide oversight and guidance on the research and development of transformative gene editing therapies.
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 USD 1bn, including USD 245m upfront and potential future payments based primarily upon the successful achievement of specified development and regulatory milestones for the DMD and DM1 programmes.
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 3Q19.
Vertex has appointed Dr. John T. Gray as senior vice president, Genetic Therapies effective June 17, 2019.
With more than twenty-five years in academic and industry settings, John is a proven leader in the genetic therapies space with experience in both the scientific and manufacturing sides of product development.
His capabilities and expertise are an ideal fit with Vertex's strategy to bring the multi-faceted components of genetic therapies together and to advance the science of genetic therapies, such that the Vertex genetic therapies platform offers the best of all this emerging approach has to offer.
Vertex will establish a new genetic therapies research site in the Boston area where research programmes, as well as vector development and clinical manufacturing for genetic therapies, including DMD and DM1 programmes, will be conducted.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District.
CRISPR Therapeutics is a gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR Therapeutics AG is headquartered in Zug, Switzerland.
Exonics Therapeutics is developing gene editing therapies to treat patients with Duchenne muscular dystrophy and other severe genetic neuromuscular diseases.
In multiple Duchenne preclinical models, Exonics has used SingleCut CRISPR to genetically repair and restore dystrophin, the key protein missing in children with Duchenne. Exonics is initially focused on repairing mutations that cause Duchenne in order to develop a therapy to treat many children with the devastating disease, for which there is no cure.
Exonics' technology is licensed from UT Southwestern Medical Center and is based on the research of Eric Olson, Ph.D., Exonics' founder and chief science advisor. Exonics is located in Watertown, Mass.