Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) released details supporting the Orphan Designation of BIVV003, an investigational ex vivo gene-edited cell therapy product candidate currently being evaluated for the treatment of sickle cell disease in the Phase 1/2 PRECIZN-1 study partnered with Sanofi. The Committee’s decision to grant Orphan Designation was based in part on early data from three patients that had 52 weeks, 13 weeks, and 29 days of follow-up, respectively.
In recently published minutes, the Committee considered the preliminary clinical observations of BIVV003 as well as the potential of long-term effects that may obviate the need for frequent treatment suggested a clinically relevant advantage. The Committee’s published minutes report information on select patient characteristics for the first three patients treated, including genotype and history of red blood cell transfusions and vaso-occlusive crises. Sangamo and Sanofi expect to enroll a total of eight patients in the PRECIZN-1 study.
As previously indicated, Sangamo and Sanofi expect to submit updated data from the PRECIZN-1 study for presentation at a medical meeting later this year. At that time, the Companies will also provide an update on the partnered ongoing Thales study evaluating ST-400 in beta thalassemia.