RARE Daily

Vertex and Obsidian Enter into Collaboration to Discover Novel Therapies that Regulate Gene Editing

April 22, 2021

Rare Daily Staff

Vertex Pharmaceuticals and Obsidian Therapeutics announced that the two companies have entered into a strategic research collaboration and licensing agreement focused on the discovery of novel therapies that regulate gene editing for the treatment of serious diseases.

The collaboration leverages Obsidian’s cytoDRiVE platform technology to discover gene-editing medicines whose therapeutic activity can be precisely controlled using small molecules and Vertex’s scientific and clinical capabilities in small molecule, and cell and genetic therapies to rapidly bring these approaches to patients.

“The ability to tune gene-editing activity to a specific level is an important innovation that has the potential to address several serious diseases,” said Vertex’s David Altshuler, executive vice president of global research and chief scientific officer.

Under the terms of the agreement, Obsidian will use its cytoDRiVE technology, which provides a way to control protein degradation using FDA-approved small molecules, to develop novel regulated gene editing therapy candidates for multiple serious diseases. Obsidian grants Vertex the exclusive option to license worldwide rights to candidates discovered and developed under the collaboration. Following Vertex’s exercise of its options, Vertex will be responsible for further preclinical and clinical development and commercialization.

Vertex will pay Obsidian up to $75 million in upfront payments and research milestones that may be paid during the research term, including an equity investment in Obsidian. Obsidian is eligible to receive up to $1.3 billion in potential payments based upon the successful achievement of specified research, development, regulatory, and commercial milestones across up to five potential programs. In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration. Specific diseases that are the subject of this collaboration were not disclosed.

“At Obsidian, we’re using our cytoDRiVE technology to pioneer a new generation of engineered cell and gene therapies for patients with serious diseases,” said Paul Wotton, CEO of Obsidian Therapeutics. “Partnering with Vertex, an established leader at the forefront of genetic therapies, will further advance our technology into the gene therapy field and accelerate development of controllable gene editing therapies for patients.”

Photo: David Altshuler, executive vice president of global research and chief scientific officer


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