Vertex Pharmaceuticals is inking another gene-editing deal, days after retooling a partnership with CRISPR Therapeutics to the tune of $900 million. This time, the company is teaming up with Obsidian Therapeutics to discover new gene-editing treatments that can be controlled with small molecules.
Vertex will fork over up to $75 million in equity, upfront payments and research milestone payments, with the promise of up to $1.3 billion down the line if the duo meets certain research, development, regulatory and commercial goals, according to a statement.
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Under the multi-year deal, Obsidian will use its technology to discover gene-editing treatments whose effects—namely, protein expression—can be dialed up or down using small molecules. Its platform allows for the control of protein degradation by exploiting drug-responsive domains, or regions of protein chains that are unstable inside of cells but can be reversibly stabilized using small molecules.
“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 David Altshuler, Vertex’s chief scientific officer and executive vice president of global research, in the statement.
The duo did not divulge the targets of the deal, only that they would pursue treatments for “serious diseases.” Vertex has the exclusive option to license any candidates that come out of the deal and would pick up further preclinical and clinical development and commercialization for those targets.
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Vertex is striking the Obsidian deal just days after taking the lead in a gene therapy partnership with CRISPR Therapeutics. In the retooled deal, Vertex is taking responsibility for 60% of the costs for CTX001, which the partners are developing for the treatment of sickle cell disease and beta-thalassemia. Vertex will hand over $900 million upfront and a potential $200 million in milestones if the treatment is approved, but will receive 60% of the profits from the future worldwide sales of CTX001 in return.
Vertex and CRISPR originally inked a research agreement in 2015 to develop treatments using CRISPR/Cas9 to address genetic diseases. CTX001 is the first asset to come out of the deal, which was originally worth $75 million in cash paid to CRISPR and $30 million in the company’s equity for Vertex.