Sangamo shelves sickle cell therapy, joining rivals forced to rethink development plans

Sangamo Therapeutics is shelving later-stage development of its sickle cell drug, joining two other biotechs that, in the last 24 hours, have been forced to make similar decisions.

The company announced in its fourth-quarter business update that it would halt potential phase 3 investment of BIVV003 to instead funnel resources toward its Fabry disease program and autologous renal transplant med. The company said that it was looking for a partner for the sickle cell med that could help finance future phase 3 development. 

The news marks the third company in the past day to reconsider sickle cell treatment development. Novartis dropped a long-time ex vivo partnership with CRISPR company Intellia, with Intellia’s CEO calling the race “wide open.” Graphite Bio also axed its sickle cell program after a serious adverse event, resulting in half the company's staff being laid off and two executives departing. 

The business decisions exemplify how swiftly the sickle cell race has heated up and subsequently how quickly it can cool down for those making up the rear. 

Last December, at the American Society of Hematology annual meeting, Sangamo reported six-month data from its phase 1/2 trial on the first patient dosed using a so-called “improved method.” The results found that the patient achieved fetal hemoglobulin levels of 45% and total hemoglobin of 12.4 g/dL at Week 26. Sangamo has been tinkering with the manufacturing method for the med, called BIVV003, to “strengthen clinical outcomes and reduce manufacturing costs” for a potential phase 3 study. 

The decision to pause future sickle cell work comes just over a year after Sanofi returned the program to Sangamo. At the time, Sangamo CEO Sandy Macrae said in a press release that the company remained “committed to progressing this program.” But Macrae also teased at the time that the company would “vigorously investigate” new options to continue clinical development. 

All three companies pulling back work were chasing Vertex and CRISPR Therapeutics, which are quickly finalizing regulatory submissions around the globe. Applications to European and U.K. regulators are complete for the companies' lead asset exa-cel, designed to treat both sickle cell disease and beta thalassemia. The two companies initiated a rolling application with the FDA in November 2022 and plan to complete the filing before the end of the first quarter this year. 

Vertex Chief Operating Officer Stuart Arbuckle told investors earlier this month that the target population for exa-cel will be about 32,000 patients with severe disease in Europe and the U.S. The company is looking to establish roughly 75 treatment sites between the two regions. Left unknown is how much Vertex and CRISPR plan to price the treatment, which, if recent gene therapy approvals are any guide, could cost millions of dollars