Boston-based biotech, Vertex, has signed a deal worth up to $1.2bn to use Arbor Biotechnologies’ CRISPR gene-editing technology to develop novel cell therapies for the treatment of serious diseases.
The deal builds on an earlier collaboration with the company that started in 2018 and will see Vertex receive rights to use Arbor’s technology to research and develop ex vivo engineered cell therapies. Vertex hopes this will give it extra momentum in two key areas; the treatment of type 1 diabetes, where it hopes to generate fully differentiated, insulin-producing hypo-immune islet cells, and sickle cell disease and beta thalassemia.
“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 Vertex cell and genetic therapies head, Bastiano Sanna. He added that Arbor’s technology combined with Vertex’s ongoing programmes and capabilities in diabetes, haemoglobinopathies and other diseases would lead to improved cell replacement therapies for “broad populations of patients”.
Under the agreement, Arbor will receive an upfront cash payment as well as up to $1.2bn in research, development, regulatory and commercial milestone payments, and tiered royalties on future net sales. Vertex will also make an investment in Arbor in the form of a convertible note.
Vertex’s existing portfolio of treatment for cystic fibrosis (CF) saw its Q2 revenues reach $1.79bn, up by 18% on the same period in 2020, with 2021 revenues expected to reach $7.4bn.
In July, the company announced the start of a phase 3 trial in a once-daily triple combination regimen in CF that, if successful, could replace its own Trikafta combination, offering to “maximise the benefit and convenience” for CF patients.
Beyond CF, Vertex also has a “robust pipeline of investigational small molecule medicines” in other serious diseases where “it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases”, said the company.
Arbor is an early-stage life sciences company that ‘uses its proprietary discovery engine to discover unique and wholly owned CRISPR-based genetic modifiers with differentiated genetic editing capabilities’. It recently announced an agreement with Basel-based Lonza to manufacture the next generation of therapeutics.
The deal with Arbor further enhances Vertex’s focus on cell and genetic therapies. In April, it announced a collaboration with Massachusetts-based Obsidian Therapeutics to leverage its proprietary cytoDRiVE platform to aid its focus on the discovery of novel therapies that regulate gene editing in serious diseases. In the same month, the company extended its collaboration with CRISP Therapeutics for the development, manufacturing and commercialisation of CTX001 in sickle cell disease and beta thalassemia. Under this deal, CRISPR received $900m upfront and will get another $200m upon CTX001 regulatory approval.