SNY - Sangamo highlights EMA opinion on BIVV003 Orphan Designation

Sangamo Therapeutics (SGMO) announces 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 (SNY).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.The committees recent disclosure suggests that BIVV003 showed a clinically relevant advantage during preliminary clinical observations, and hinted at the therapy's potential of long-term effects that could eliminate the need for frequent treatment, the company said.Sangamo and Sanofi expect to submit updated data from the PRECIZN-1 study for presentation at a medical meeting later this year.Sangamo shares up marginally during after market trading.

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Sangamo highlights EMA opinion on BIVV003 Orphan Designation