Vertex layers on bluebird bio pressure with $1.2B Arbor gene-editing pact

CRISPR-Cas9
Arbor will give a boost to Vertex's cell and gene therapy "toolkit" for treating multiple serious diseases. (Ernesto del Aguila III, National Human Genome Research Institute, NIH)

Months after first-in-human gene-editing results made a splash around the world, Vertex is making a $1.2 billion edit to its CRISPR work through a new research pact with existing partner Arbor Biotechnologies.

After dishing out $900 million upfront to CRISPR Therapeutics for sickle cell disease and beta thalassemia in April, Vertex is doubling down in its attempt to beat bluebird bio to market with a gene-editing therapy for those blood diseases. Vertex will use Arbor's gene-editing tech know-how to layer on yet another plan of attack in the two indications plus Type 1 diabetes.

The two collaborations could total more than $2.3 billion without factoring in an undisclosed upfront payment and convertible note investment in Arbor. Vertex's CRISPR bet is no surprise given rising interest in gene editing that has reached a fever pitch on recent breakthroughs. 

Intellia and partner Regeneron showed for the first time that their in vivo CRISPR gene-editing candidate worked better than standard of care therapy in patients with transthyretin amyloidosis in June.

RELATED: Vertex takes the lead in CRISPR Therapeutics partnership with $900M upfront

Now, even Moderna wants in as the mRNA leader reels in hordes of money from its COVID-19 vaccine. Executives recently revealed they are hunting for gene-editing deals. Already this year, $14.1 billion has been funneled into the regenerative medicine space in the first half of 2021, positioning cell and gene therapy and gene-editing technology for a record year of investment.

Arbor will give a boost to Vertex's cell and gene therapy "toolkit" for treating multiple serious diseases, said Bastiano Sanna, Ph.D., Vertex's executive vice president and chief of cell and genetic therapies, in a statement.

The billion-dollar confidence in Arbor perhaps comes from the biotech's founder, CRISPR pioneer Feng Zhang, Ph.D., of the Broad Institute. Zhang is well recognized in the gene-editing space for his work forming CRISPR biotechs like Beam Therapeutics, creating RNA editing systems that could potentially treat Alzheimer's disease and, this month, unveiling a new mRNA delivery system for potential use in RNA-based gene therapies across more than one dose. 

Vertex wants to use Arbor's proprietary CRISPR gene-editing technology to research and develop ex vivo engineered cell therapies. The technology will go toward Vertex's insulin-producing hypoimmune islet cells to treat Type 1 diabetes as well as next-generation sickle cell disease and beta thalassemia treatments. The partnership includes up to seven potential programs.

Vertex is already working with CRISPR Therapeutics on CTX001, a sickle cell disease and beta thalassemia therapy that will go up against bluebird bio's candidate for the blood diseases. Bluebird already has the European green light for Zynteglo but has run into pricing snags in Germany and safety concerns in U.S. clinical trials. 

RELATED: Arbor Biotechnologies comes out of stealth with new CRISPR enzyme

These are difficult blood diseases to treat, which is perhaps why Vertex has enlisted the help of both CRISPR and Arbor. Sickle cell is an inherited disorder most commonly found in Black patients that causes red blood cells to become misshapen, die and clog blood flow in the body. Current medications or transfusions can manage complications—if caught early—but a curative treatment is needed. Beta thalassemia is also an inherited disorder that leads the body to produce less hemoglobin than normal, which can cause anemia. 

Arbor and Vertex have worked together before on protein discovery for gene-editing therapies through a 2018 deal. That program, for which the companies did not disclose financial details, was aimed at cystic fibrosis and four other undisclosed diseases.