Vertex licenses blood disorder gene therapy from CRISPR Therapeutics

Sickle cell smear
Vertex and CRISPR Therapeutics expect to begin clinical trials of a gene-editing treatment for beta-thalassemia and sickle cell disease in 2018.

Vertex Pharmaceuticals bet big on gene editing in 2015, when it poured $105 million into a research collaboration with CRISPR Therapeutics. Now, Vertex is licensing the first CRISPR-based treatment to come out of the deal: a candidate targeting beta-thalassemia and sickle cell disease.

The pair will co-develop and co-commercialize the investigational treatment, CTX001, sharing equally the R&D costs and profits worldwide, they said in a statement.

Sickle cell disease and beta-thalassemia are caused by mutations in the beta-globin gene, which codes for a part of hemoglobin, the oxygen-carrying component of red blood cells. This results in missing or defective hemoglobin. CTX001 is based on the knowledge that fetal hemoglobin—found in newborn babies and later replaced by adult hemoglobin—can be protective in adults who have blood disorders.

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With CTX001, a patient’s cells are harvested and edited with CRISPR to increase fetal hemoglobin levels in the patient’s blood cells. The edited cells are then infused back into the patient, where they are expected to produce blood cells with fetal hemoglobin, thereby compensating for faulty adult hemoglobin.

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The therapy is slated to enter phase 1/2 trials in 2018. A U.S. trial will investigate its use in sickle cell disease, while a European trial will study it in beta-thalassemia. The latter study will be the first company-sponsored trial of a CRISPR-based therapy, said CRISPR Therapeutics CEO Samarth Kulkarni, Ph.D.

“Over the past two years, we’ve made significant progress with CRISPR Therapeutics on the discovery and preclinical development of multiple CRISPR/Cas9-based treatments, and we’re pleased to select CTX001 as the first of these treatments to move into clinical development as part of our collaboration,” said David Altshuler, M.D., Ph.D., Vertex’s global research chief, in the statement. “The addition of CTX001 to our clinical development pipeline provides us with a near-term opportunity to generate the first proof-of-concept clinical data for a CRISPR/Cas9-based medicine in two genetic diseases that are highly aligned with our research strategy.”

Vertex had been focusing on its cystic fibrosis drug franchise when it struck the deal with CRISPR Therapeutics two years ago. It forked over $75 million in cash and took a $30 million stake in the company. The four-year pact gives Vertex the right to option up to six programs.