21 May 2018 - - The US Food and Drug Administration has accepted the investigational new drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease, developed by Waltham, Massachusetts-based blood disorders therapeutics developer Bioverativ Inc. (NASDAQ: BIVV) and Richmond, California-based genomic therapies specialist Sangamo Therapeutics, Inc. (NASDAQ: SGMO), the companies said.
Bioverativ and Sangamo are developing BIVV003 as part of an exclusive worldwide collaboration to develop and commercialize gene-edited cell therapies for sickle cell disease and beta thalassemia. This IND enables Bioverativ to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy of BIVV003 in adults with sickle cell disease.
Currently, Sangamo is enrolling patients with transfusion-dependent beta thalassemia in a Phase 1/2 trial evaluating the safety, tolerability, and efficacy of ST-400, which uses the same gene-editing approach as BIVV003.
BIVV003 is a non-viral approach utilizing zinc finger nuclease gene-editing technology. By modifying a short sequence of the BCL11A gene in a patient's red blood cell precursors, sickle hemoglobin production is suppressed, and the production of fetal hemoglobin is reactivated to levels that may protect patients against the progression of their sickle cell disease.
Bioverativ is a global biotechnology company dedicated to research, development, and commercialization of innovative therapies for hemophilia and other rare blood disorders.
Sangamo Therapeutics is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy.