Home Health Care Vertex Pharma inks another R&D alliance to expand its gene-editing ambitions

Vertex Pharma inks another R&D alliance to expand its gene-editing ambitions

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Cystic fibrosis drugs are the mainstay of the Vertex Pharmaceuticals portfolio now, but the company has been trying to diversity and it has made gene-editing therapies a key part of that strategy. On Thursday, Vertex announced a partnership with Obsidian Therapeutics that gives it access to that biotech’s technology for engineering cell and gene therapies.

No disease targets were disclosed, and the financial aspects of the partnership were described only in broad terms. Boston-based Vertex has committed to pay Obsidian up to $75 million, a sum that covers an upfront payment, research milestones, and an equity investment in the biotech.

Obsidian’s proprietary technology, called cytoDRiVE, uses small molecules to precisely control when a protein is expressed and how much is expressed. Under the terms of the Vertex agreement, Obsidian will use its technology to develop regulated gene editing therapies for multiple serious diseases. Vertex has an exclusive option to license candidates discovered and developed under the collaboration. If it exercises options on any therapeutic candidates, Vertex will be responsible for further preclinical and clinical development, and commercialization if they win FDA approval.

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

Vertex has expanded into genetic medicines in recent years by striking deals with other companies. It has been working with Switzerland-based CRISPR Therapeutics since 2015, an alliance that has been expanded several times, most recently on Monday. Vertex agreed to pay $900 million up front for a greater share of a gene-editing therapy the two companies are developing for two rare blood disorders, sickle cell disease and beta thalassemia. In 2019, Vertex paid $245 million up front to acquire Exonics Therapeutics, a company using CRISPR gene editing to develop therapies for rare muscular disorders.

Obsidian, based in Cambridge, Massachusetts, has not yet reached the clinic but it’s on a path to get there. The company plans to file clinical trial paperwork with the FDA in the middle of 2022 for its lead program, a type of cancer immunotherapy called a tumor infiltrating lymphocyte (TIL) that is engineered with the signaling protein interleukin 15. TILs are also the focus of Obsidian’s research partnership with the MD Anderson Cancer Center. In a separate alliance, Obsidian has been working with Bristol Myers Squibb to develop new cell therapies for cancer. The pharmaceutical giant inherited that partnership via its acquisition of Celgene.

If Vertex’s multi-year agreement with Obsidian leads to a commercialized product, the Boston company could pay its partner up to $1.3 billion spanning up to five potential programs.

Image: vchal, Getty Images

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