Hemophilia

Sanofi SA (NASDAQ: SNY) presented data from two Phase 3 studies of fitusiran for the prophylactic treatment with hemophilia A or B, with or without inhibitors. Data were shared at the American Society of Hematology (ASH) Annual Meeting.  Fitusiran, an RNAi drug designed to...

Centessa Pharmaceuticals plc (NASDAQ: CNTA) has announced positive topline results from the Phase 2a part of AP-0101, the six-month repeat dose portion of the study evaluating SerpinPC in severe hemophilia A and B patients. SerpinPC was well-tolerated. As previously disclosed, one...

Catalyst Biosciences Inc (NASDAQ: CBIO) shares are on a volatile ride Wednesday following the release of late-stage trial results for its bleeding disorder drug candidate.  The shares, which were rallying over 27 percent at one point in premarket trading, were most recently...

Uniqure NV (NASDAQ: QURE), a gene therapy company that focuses on severe unmet medical needs, could "dominate" the space with its AMT-061 therapy, according to B. Riley FBR. The Analyst B. Riley FBR's Madhu Kumar initiated coverage of Uniqure with a Buy rating and $50 price target....

Catalyst Biosciences Inc (NASDAQ: CBIO), a nano-cap clinical-stage biopharmaceutical company that focuses on the treatment of hemophilia, has high expectations for its therapy for the treatment of hemophilia B. The Analyst Chardan's Gbola Amusa maintains a Buy rating on Catalyst Biosciences...

Bioverativ Inc (NASDAQ: BIVVV) — the NASDAQ's brand-new biotech company that’s focused on treatments for hemophilia and other blood disorders — listed this week with $325 million in hand after spinning off from Biogen Inc (NASDAQ: BIIB). The company markets treatments such as...