Apellis Pharmaceuticals’ pegcetacoplan beat out Alexion Pharmaceuticals’ Soliris in the Phase III PEGASUS study in adults with paroxysmal nocturnal hemoglobinuria (PNH).

Celgene and developmental partner Acceleron Pharma are eying a potential U.S. FDA approval of a blood-disease treatment.

Private investment firm Blackstone Life Sciences along with Novartis launched Anthos Therapeutics with a $250 million investment.

Japanese scientists will test the use of human-induced pluripotent stem cells (iPS) to treat spinal cord injuries, a health ministry panel that approved the research project said.

The U.S. Food and Drug Administration approved Stemline Therapeutics Inc.’s Elzonris for the treatment of a rare blood disease in adults and children aged 2 years and older.

Investor’s Business Daily looked at three small biotech companies that appear to be leading the CRISPR race.