Investors Hand Disc Medicine $90 Million to Control Hematological Diseases Published: Sept. 2, 2021 By Heather McKenzie BioSpace   A two-year-old biopharma company tackling rare blood diseases has received a $90 million infusion of cash. Disc Medicine, which is progressing a pipeline of clinical-stage therapies for hematologic illnesses including myelofibrosis and porphyrias, announced the Series […]

Vertex Pharmaceuticals and Arbor Biotechnologies announced a new collaboration to enhance efforts in developing ex vivo engineered cell therapies, using Arbor’s proprietary CRISPR gene-editing technology for select diseases.

The U.S. Food and Drug Administration approved Nucala (mepolizumab) for adults and children aged 12 years and older with hypereosinophilic syndrome (HES) for six months or longer without another identifiable non-blood related cause of the disease.

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.