Vertex and Arbor Biotechnologies Collaborating to Develop Cystic Fibrosis Gene Therapies

Vertex Pharmaceuticals and Arbor Biotechnologies have established a partnership to discover new proteins to advance the development of gene therapies for cystic fibrosis.

The companies also plan to develop new genome-editing approaches for four other diseases but haven’t revealed which ones yet.

Under the terms of the agreement, Arbor will primarily carry out the laboratory research and Vertex will finance the work. The deal comes with an upfront cash payment, research and commercial milestones support, as well as royalties on future net sales — all Vertex’s responsibility.

Vertex will also invest in Arbor in the form of a convertible note, and gain an observer seat on the Arbor board of directors.

Using its protein biodiscovery platform, Arbor has access to an array of technologies including machine learning, genome sequencing, gene synthesis, and high-throughput screening. This know-how will add to Vertex’s efforts to design new gene therapies for the treatment of cystic fibrosis.

“Our strategy of developing transformative medicines for serious diseases is based on addressing causal human biology with innovative therapeutics,” David Altshuler, MD, PhD, executive vice president, global research, and chief scientific officer of Vertex, said in a press release. “Arbor’s proprietary high-throughput screening platform will enhance our ongoing efforts to develop innovative gene-editing therapies.”

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Researchers from both companies will work together on the discovery of new highly active, reliable DNA endonucleases or nickases. These proteins are DNA-cutting enzymes that break the DNA molecule at a specific site. What distinguishes the two enzymes from one another is that an endonuclease cuts both strands of the DNA molecule, while a nickase breaks only a single strand.

These proteins are appealing to genetic research because of their ability to manipulate the genome, as well as to introduce new DNA sequences in an organism, which could ultimately lead to the production of the desired gene.

“Vertex has a proven track record in discovering and developing innovative medicines and is an ideal partner for Arbor,” said David Walt, PhD, Arbor co-founder. “We believe that by using our powerful biodiscovery platform we will be able to identify complementary and next generation tools to enhance Vertex’s pipeline of transformative medicines and new gene-editing therapies.”