Hemophilia A

Swedish Orphan Biovitrum AB (OTC: BIOVF) and Sanofi SA (NASDAQ: SNY) have announced topline results from the XTEND-1 phase 3 study of efanesoctocog alfa (BIVV001) in previously treated severe hemophilia A patients aged 12 and above. The study met the primary endpoint, showing...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) announced results from its ongoing Phase 3 GENEr8-1 study of valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.  Annualized Bleeding Rate (ABR) was significantly reduced by 4.1 treated bleeds per year in...

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...

Generation Bio Co (NASDAQ: GBIO) provided an update on factor VIII expression from a series of mouse and companion non-human primate (NHP) studies in hemophilia A and an update on its development non-viral genetic medicine platform.  In mouse studies, rapid enzymatic synthesis...

Sigilon Therapeutics Inc (NASDAQ: SGTX) reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. The SIG-001 trial had been placed on FDA clinical hold following a serious...

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...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from open-label Phase 1/2 trial of valoctocogene roxaparvovec, an investigational gene therapy candidate for severe hemophilia A. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH)...

Spark Therapeutics, a unit of Roche Holdings AG (OTC: RHHBY), has announced updated data from the ongoing Phase 1/2 trial of SPK-8011 gene therapy in hemophilia A. Data were presented at the International Society of Thrombosis and Hemostasis (ISTH) 2021. These data demonstrated...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced new data from GENEr8-1 Phase 3 study for valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.  Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021...

According to Roche Holding AG's (OTC: RHHBY) Genentech division, new data from a phase 3b study reinforces the safety profile of blockbuster hemophilia A treatment Hemlibra. The new safety data from the Phase 3b STASEY study revealed that Hemlibra was effective...

The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical Inc's (NASDAQ: BMRN) marketing application seeking approval for hemophilia A gene therapy, valoctocogene roxaparvovec. With today's validation, the application review can now commence. A CHMP opinion...

The FDA has instituted a clinical hold on Sigilon Therapeutics Inc's (NASDAQ: SGTX) Phase 1/2 study of SIG-001 in patients with severe or moderately severe hemophilia A.  The clinical hold was initiated following the Company's submission of a serious adverse event...

The European Medicines Agency (EMA) has granted its request for accelerated assessment review to BioMarin Pharmaceutical Inc’s (NASDAQ: BMRN) valoctocogene roxaparvovec, gene therapy to treat severe hemophilia A, with an opinion expected in the first half of 2022,...

BioMarin Pharmaceutical Inc (NASDAQ: BMRN) has announced an update to its previously reported results from Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. Data will be shared at the International Society...

Just two months after Centessa Pharmaceuticals launched after the merger of 10 private biotech companies, the company has filed a $100 million IPO for listing on NASDAQ. Founded by Medicxi, Centessa in February raised a $250 million Series A round of funding. The Centessa portfolio...