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Vertex, looking to broaden its gene editing abilities, asks a young biotech for help

Vertex is best known for its work in cystic fibrosis, having developed four approved medicines that, by the company’s estimates, can treat about 90% of the patient population.

Those medicines are what’s known as small-molecule drugs, which, as the name suggests, means they’re tiny enough to slip into cells without much trouble. But to address the remaining 10% of cystic fibrosis patients, Vertex has proposed that more complex treatments may be needed.

As such, the company has inked partnerships with CRISPR Therapeutics, Affinia Therapeutics and Arbor, aiming to create genetic medicines that address the underlying causes of cystic fibrosis. That work hasn’t been limited to just cystic fibrosis either; Vertex and CRISPR, for example, are among the leaders in the race to develop potential cures for sickle cell and another blood disorder called beta thalassemia.

Vertex first linked with Arbor in late 2018. At the time, Arbor was still fresh on the scene, having just come out of stealth mode that March with about $16 million in funding from Arch Venture Partners, Faridan, Alexandria Real Estate Equities and some private investors.

Arbor drew interest because it found an enzyme that, according to research, could hold advantages over other enzymes used in CRISPR gene editing. To date, the company says that through its protein discovery platform, it’s identified more than 60 families of a nucleases — enzymes that break down genetic material — and over 70 CRISPR enzymes called transposases.

Vertex is now doubling down on Arbor’s technology, hoping that it will come in handy for programs targeting Type 1 diabetes, sickle cell, beta thalassemia and other diseases. In addition to the upfront payment and possible milestones, Vertex has also agreed to invest in Arbor through a convertible note.

“This new collaboration further expands our toolkit in cell and genetic therapies and, specifically, our work to discover and develop cell therapies for the treatment of multiple serious diseases,” said Bastiano Sanna, Vertex’s chief of cell and genetic therapies, in a statement Tuesday.

The new Arbor partnership adds to a string of Vertex deals focused on more cutting-edge drugmaking technologies. In 2019, Vertex bought a company called Exonics Therapeutics and expanded a research pact with CRISPR Therapeutics to jumpstart development on gene-editing therapies for the rare disorder Duchenne muscular dystrophy.

Later that year, Vertex spent $950 million to acquire Semma Therapeutics, a company trying to cure Type 1 diabetes through the use of stem cell-based therapies. Initial human testing of Semma’s therapy began in March.

More recently, Vertex dropped $900 million in April to take a larger commercial stake in the gene-editing treatment for sickle cell and beta thalassemia it’s developing with CRISPR Therapeutics.