The Latest Breaking News from Sickle Cell Disease

Precision BioSciences Inc (NASDAQ:DTIL) has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis AG (NYSE:NVS)  Precision will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a "safe harbor" location in the genome as a potential one-time…

During the European Hematology Association (EHA) conference, Forma Therapeutics Holdings Inc (NASDAQ:FMTX) presented updated results from the 12-week open-label extension (OLE) of etavopivat's Phase 1 trial in sickle cell disease. Treatment with etavopivat 400mg QD elicited a >1g/dL hemoglobin (Hb) increase in 73% of the 15 patients and an average maximal Hb increase of…

Crispr Therapeutics could have a "functional cure" for two inherited blood diseases, analysts said Monday, but CRSP stock plummeted.

The FDA signaled in briefing documents released that it may approve Bluebird bio Inc's (NASDAQ:BLUE) beti-cel gene therapy to treat β-thalassemia for those who require regular blood transfusions. The Prescription Drug User Fee Act (PDUFA) goal dates for beti-cel and eli-cel are August 19 and September 16. Bluebird bio's blood disorder treatment demonstrates "clinically meaningful"…