Vertex boosts cell and genetic therapies toolkit in deal worth up to $1.2bn with Arbor

By Jane Byrne contact

- Last updated on GMT

© GettyImages/vchal
© GettyImages/vchal

Related tags: Vertex, Gene therapy, Cell therapy, CRISPR

Boston-based company, Vertex, has signed a deal to use Arbor Biotechnologies’ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.

The agreement, worth up to $1.2bn, expands on their original collaboration, established in 2018.

Vertex will receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies towards its goal of generating fully differentiated, insulin-producing hypoimmune islet cells for the treatment of type 1 diabetes, for next-generation approaches in sickle cell disease and beta thalassemia, and for the treatment of other diseases.

Arbor uses its discovery engine to identify CRISPR-based genetic modifiers with differentiated genetic editing capabilities that can be tailored to address the underlying pathology of genetic diseases.

The research pact terms sees Arbor set to receive an upfront cash payment, with it also eligible to get up to US$1.2bn in potential payments based upon the realization of specified research, development, regulatory and commercial milestones across up to seven potential programs.

In addition, Vertex will pay tiered royalties on future net sales on any products that may result from this collaboration.  

Novel therapies

A market leader for cystic fibrosis treatment, Vertex has a market share of over 50% in that segment and it is simultaneously researching other rare diseases.

Various acquisitions in 2019 saw the company, which started out in small molecules, move into new areas. Deals that year intended to yield a new generation of breakthrough medicines included its purchase of Exonics Therapeutics for its gene editing technology and pipeline of programs targeting diseases including Duchenne muscular dystrophy (DMD) along with Semma Therapeutics and its cell therapy treatment for type I diabetes.

A recent addition to its portfolio, through a deal struck with CRISPR Therapeutics in April this year, is CTX001, a treatment for beta-thalassemia and sickle-cell disease based on gene-editing therapy.  That same month, Vertex announced an alliance with Obsidian Therapeutics to harness its cytoDRiVE platform to aid its focus on the discovery of novel therapies that regulate gene editing in serious diseases.

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