Sernova Corp. has announced that its hemophilia A program, combining the Sernova Cell Pouch with a patient’s own cells corrected for the production of factor VIII (FVIII), has been awarded U.S. orphan drug and rare pediatric disease designations.

Sirius Therapeutics Inc. has submitted an application in Australia to begin a first-in-human trial of SRSD-107 for the prevention and treatment of thromboembolic disorders.

Biocryst Pharmaceuticals Inc. has highlighted progress in its pipeline, with five programs set to enter the clinic in the next 24 months.

Generation Bio Co. has announced a breakthrough in its nonviral genetic medicine platform with the development of a proprietary, novel DNA called immune-quiet DNA (iqDNA).

The FDA has awarded orphan drug designation to GC Biopharma Corp.’s GC-1126A for thrombotic thrombocytopenic purpura (TTP).

Recently, researchers at Cincinnati Children’s Hospital, in collaboration with colleagues in Japan, have developed a human vascular organoid model that accurately mimics the vascular damage caused by SARS-CoV-2.

Baudax Bio Inc.’s lead clinical candidate, TI-168, has been awarded U.S. orphan drug designation by the FDA for the treatment of hemophilia A with inhibitors. TI-168 is a next-generation, factor VIII (FVIII)-specific regulatory T-cell (Treg) therapy designed to address hemophilia A in patients with FVIII inhibitors.

Precision Biosciences Inc. uses its proprietary Arcus platform to develop in vivo gene editing therapies and has outlined new data from its wholly owned and partnered pipeline.

Current anticoagulant strategies include small-molecule inhibitors and biological entities targeting factor XI (FXI) which, although effective, still have bleeding as a major risk.