Vertex and Arbor Strike a $1.2 Billion Deal for Gene-Editing Programs


With a handful of drugs for cystic fibrosis already approved, Vertex Pharmaceuticals has turned its attention to expanding its pipeline through collaborations and acquisitions the past few years. Today, the California company announced a new agreement with an old partner.

Nearly three years ago, Vertex and Arbor Biotechnologies struck a deal. Vertex forked over $30 million upfront and Arbor brought its RNA-altering enzyme, Cas13d, for CRISPR gene editing to help Vertex with its CF treatments.

Now the companies are taking their partnership to a whole new level, the billion-dollar-plus level. Arbor is scoring an undisclosed, upfront cash payment with potential for up to $1.2 billion in milestone payments across up to seven possible programs. Tiered royalty sales are also on the table for any products that result from the collaboration.

The new alliance will help Vertex develop ex vivo cell therapies for next-gen approaches to treating blood diseases—sickle cell and beta thalassemia—and insulin-producing hypo-immune islet cells for the treatment of type 1 diabetes. Approaching these diseases with cell therapies goes beyond a treatment, hoping for a cure.

“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 Bastiano Sanna, Ph.D., executive vice president and chief of Cell and Genetic Therapies at Vertex.  “We are excited to bring Arbor’s technology together with Vertex’s ongoing programs and capabilities in diabetes, hemoglobinopathies and other diseases to create improved cell replacement therapies for broad populations of patients.”

Arbor is co-founded by Feng Zhang, a scientist who worked with Nobel laureate Jennifer Doudna in the CRISPR Cas9 pioneering days. He’s been focused on upping the ante ever since, looking to improve on the gene editing technology.

Source: BioSpace

Vertex is also partnered with CRISPR Therapeutics. In 2015, the two entered into a strategic research collaboration using CRISPR/Cas9 to develop new treatments targeting the underlying genetic cause of human disease.

Their first drug, CTX001, is a small molecule currently in preclinical development for beta thalassemia and sickle cell disease. It’s intended to edit a person’s hematopoietic stem cells to produce fetal hemoglobin, a form of oxygen-carrying hemoglobin naturally present at birth. If successful, the mechanism should address symptoms associated with the diseases. The drug has already received Regenerative Medicine Advanced Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA for both TDT and SCD.

Arbor has other partners on its dance card too. A recent agreement with Lonza was mum on financial details, but Arbor’s CEO David Cheng said of the deal, “Arbor’s differentiated gene editing technologies allow Lonza to manufacture the next generation of therapeutics. Arbor is committed to seeking and working with partners in this field to build an ecosystem for the research, development and manufacturing of biological and cellular therapies.”

BioSpace source: