Therapy Areas: Hereditary Disorders
Vertex Pharmaceuticals, Obsidian Therapeutics Establish Collaboration to Discover Novel Therapies That Regulate Gene Editing
22 April 2021 - - US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) and US-based biotechnology company Obsidian Therapeutics 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 companies said.

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 established scientific and clinical capabilities in small molecule, cell and genetic therapies to more rapidly bring these approaches to patients.

Under the terms of the agreement, Obsidian will use its cytoDRiVE technology 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 USD 75m 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 USD 1.3bn 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 are not disclosed.

Obsidian Therapeutics, Inc. is a biotechnology company pioneering engineered cell and gene therapies to deliver transformative outcomes for patients with intractable diseases.

The company expects to submit an IND in mid-2022 for its lead cytoTIL program, a novel engineered tumor infiltrating lymphocyte therapy armored with regulated membrane-bound IL15 that does not require patients to receive toxic IL2.

Obsidian's proprietary cytoDRiVE technology provides a way to control protein degradation using FDA-approved small molecules, permitting precise control of the timing and level of protein expression. Obsidian is headquartered in Cambridge, Mass.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

The company has multiple approved medicines that treat the underlying cause of cystic fibrosis, a rare, life-threatening genetic disease, and has several ongoing clinical and research programs in CF.

Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases.

In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London.

Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America.


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