Vertex, CRISPR Therapeutics enter gene editing research collaboration

Vertex Pharmaceuticals and CRISPR Therapeutics announced Monday a partnership focused on the use of the latter's CRISPR-Cas9 gene editing platform to discover and develop potential therapies aimed at the underlying genetic causes of disease. David Altshuler, chief scientific officer at Vertex, described CRISPR-Cas9 as "an important scientific and technological breakthrough that holds significant promise for the future discovery of potentially transformative treatments for many genetic diseases."

Under the agreed terms, Vertex made an upfront commitment of $105 million to CRISPR, including $75 million in cash and a $30-million equity investment, while CRISPR is also eligible to receive development, regulatory and sales milestones of as much as $420 million for each of as many as six licensed products in addition to royalty payments on future sales. Under the collaboration, Vertex will also receive a seat on CRISPR's board, which will be filled by Altshuler.

The drugmakers noted that the collaboration will initially centre on the use of CRISPR-Cas9 to possibly correct mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are known to result in the defective protein that causes CF, as well as edit other genes related to the disease. The companies will additionally seek to identify and develop gene-based treatments for haemoglobinopathies such as sickle cell disease, with additional efforts focused on a specific number of other genetic targets.

Vertex and CRISPR will cooperate regarding the research, development and commercialisation of treatments for haemoglobinopathies that emerge from the collaboration. For haemoglobinopathies in particular, the drugmakers will equally split all R&D costs and sales, with CRISPR leading commercialisation efforts within the US. For other disease areas including CF, Vertex will head all development and global commercialisation activities. CRISPR will primarily be responsible for discovery activities, while Vertex will fund related expenses. Vertex additionally will be solely responsible for the development expenses of licensed treatments.

A number of other drugmakers have entered into deals to use CRISPR genome-editing technology to develop new therapeutics. In January this year, AstraZeneca announced four collaborations with a number of parties to use CRISPR across its drug discovery platform in areas including cardiovascular, respiratory and immune system medicine as well as oncology. Earlier that month, Novartis entered into licensing agreements with Intellia Therapeutics and Caribou Biosciences aiming to develop treatments using the gene-editing technology.

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