Trial of Cell Therapy to Raise HbF Levels Showing Potential, But Sanofi Leaving

Sangamo Therapeutics is assuming all rights to SAR445136, an investigational gene-edited cell therapy co-developed with Sanofi and now in a Phase 1/2 trial in adults with severe sickle cell disease (SCD). Early trial data support the therapy’s safety and tolerability, and its effectiveness at raising levels of fetal hemoglobin in patients,…

Gene-Editing Tool in Ongoing Trial of BIVV003 in Treating Severe Sickle Cell Supported by Early Study

A technique called zinc finger nuclease (ZFN) gene editing technology can be used to modify immature red blood cells — called precursor cells — to  boost the production of fetal hemoglobin and help ensure red blood cells maintain a normal shape in people with sickle cell disease (SCD),…

WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

FDA Accepts Collaborative IND Application for BIVV003 to Treat Sickle Cell Disease

The U.S. Food and Drug Administration recently accepted an investigational new drug (IND) application submitted by Bioverativ and Sangamo Therapeutics for BIVV003, a gene-edited cell therapy candidate for the treatment of sickle cell disease. BIVV003 uses a nonviral approach that uses zinc finger nuclease (ZFN) gene-editing technology to…