Modality moves at Vertex

How Vertex hopes to reach more patients with Arbor's CRISPR tools

In licensing a suite of newly discovered gene editing tools, Vertex Pharmaceuticals Inc. is broadening its options for solving problems that its core small molecule technology can’t address.

On Jan. 3, Vertex announced a deal with Arbor Biotechnologies Inc. that gives it access to novel CRISPR endonucleases -- the workhorse enzymes of the technology -- allowing the company to sidestep the complex IP landscape surrounding the more commonly used CRISPR-Cas9 (see “Plenty of CRISPR Pie.”)

The same day, Arbor researchers published a Science study that identified four new Type V CRISPR endonucleases -- Cas12c, Cas12g, Cas12h and Cas12i -- with unique features that apply to diagnostics, base editing and high-specificity cleavage of a broad range of target sites.

The partnership is the third in just over three years that indicate Vertex is branching out the modality base of its early pipeline.

“It’s a high order priority for us to allocate capital outside the company to do collaborations to give us opportunities with more modalities and more medicines for diseases that we would be interested in,” COO and EVP Ian Smith told investors on the company’s 3Q earnings call last year.

In October 2015, Vertex partnered with CRISPR Therapeutics AG to gain rights to up to six CRISPR-Cas9-based therapies for cystic fibrosis, sickle cell disease and other indications for $105 million up front, including $75 million in cash and a $30 million equity investment. CRISPR is eligible for $420 million in milestones, plus

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