This page keeps track of new/upcoming treatment options for hemophilia and bleeding disorders. What’s in development? What’s promising? What’s available soon? Find out below…
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News
- Newly Approved Drug Being Called ‘Game Changer’ For People Who Suffer From Hemophilia Sep 4, 2019
- UniQure stays a step ahead in hemophilia B Sep 3, 2019
- Pediatric hematologists introduce a novel way to treat pediatric hemophilia A patients Aug 29, 2019
- Recent advances in thrombin-targeting therapeutic strategies that aim to prevent bleeding in patients with hemophilia and other rare bleeding disorders Jun 7, 2019
- BioMarin says data shows hemophilia gene therapy effects could wane May 28, 2019
- Biomarin to seek approval for hemophilia gene therapy, but durability questions persist May 28, 2019
- Gene Therapy: Paving New Roads in the Treatment of Hemophilia May 16, 2019
- Potential Gene Therapy Safely Restores FVIII Clotting Factor in Animals and Human Cells, ASC Therapeutics Reports May 3, 2019
- uniQure Announces New Preclinical Data in Hemophilia A [AMT-180] in Oral Presentations at the 22nd ASGCT Annual Meeting May 2, 2019
- Sigilon Therapeutics Delivers Oral Presentation on SIG-001 for Hemophilia A at 2019 ASGCT Meeting May 1, 2019
- Applied StemCell Therapeutics Cures Hemophilia A in Mice Apr 25, 2019
- “A number of biopharma companies are making progress in developing [hemophilia] treatments, therapies, and potentially even cures” Apr 17, 2019
- Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia Apr 15, 2019
- SB-525 Continues to Show Encouraging Results for Hemophilia A in Phase 1/2 Trial Apr 3, 2019
- Sangamo and Pfizer Announce Phase 1/2 Interim Data for Investigational Hemophilia A Gene Therapy (SB-525) Apr 2, 2019
- Catalyst Biosciences Receives Orphan Designation from the European Commission for Marzeptacog Alfa (Activated) for the treatment of haemophilia B Apr 1, 2019
- Coming to Grips with The Potential Benefits Of Gene Therapy – “a recently developed gene therapy promises to cure [hemophilia] patients with a single intervention, promising enormous improvements” Mar 15, 2019
- Hemlibra Receives Approval for Severe Hemophilia A Without Factor VIII Inhibitors from the European Commission Mar 14, 2019
- Recent Developments in Hemophilia: Gene Therapy — Approach offers new paradigm of treatment, though hurdles remain Mar 11, 2019
- They Thought Hemophilia Was a ‘Lifelong Thing.’ They May Be Wrong. Mar 1, 2019
- GC starts Phase 1 trials of MG1113, an anti-TFPI antibody treatment for hemophilia Feb 28, 2019
- Roche Bids $4.8 Billion for U.S.-Based [Hemophilia] Gene Therapy Specialist Spark Feb 25, 2019
- Esperoct Approved for Treatment of Hemophilia A in Adults and Children Feb 21, 2019
- Novo Nordisk Snags FDA Approval for Hemophilia A Treatment Feb 20, 2019
- New Treatment for Hemophilia A Receives FDA Approval Feb 19, 2019
- Novo Nordisk receives US FDA approval of ESPEROCT® (turoctocog alfa pegol, N8-GP) Feb 19, 2019
- Single Dose of Gene Therapy AMT-061 Proves Effective in Hemophilia B Feb 14, 2019
- UniQure update bolsters outlook for hemophilia B gene therapy Feb 11, 2019
- uniQure Announces Updated Clinical Data from Phase IIb Study of AMT-061 in Patients with Hemophilia B Feb 8, 2019
- Catalyst Biosciences Announces Preclinical Proof-of-Concept Data of Gene Therapy Candidate CB 2679d-GT for the Treatment of Hemophilia B Feb 6, 2019
Research
- Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII Nov 14, 2019
- Mesenchymal stem cells as carrier of the therapeutic agent in the gene therapy of blood disorders Nov 6, 2019
- Tissue Factor-Negative Cell-Derived Microparticles Play a Distinctive Role in Hemostasis Jun 13, 2019
- Limitations of prophylactic treatment in patients with hemophilia May 30, 2019
- The central role of thrombin in bleeding disorders May 22, 2019
- Gene Therapy: Paving New Roads in the Treatment of Hemophilia May 16, 2019
- Current and Emerging Biologics for the Treatment of Hemophilia Apr 30, 2019
- Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A Apr 16, 2019
- CRISPR/Cas9-mediated in vivo gene targeting corrects hemostasis in mice Apr 11, 2019
- Hemophilia in a Changing Treatment Landscape Mar 29, 2019
- FVIII expression by its native promoter sustains long-term correction avoiding immune response in hemophilic mice Mar 12, 2019
- RNA Viruses as Tools in Gene Therapy and Vaccine Development Mar 1, 2019
- Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9 Feb 13, 2019
- Gene-based FVIIa prophylaxis modulates the spontaneous bleeding phenotype of hemophilia A rats Jan 31, 2019
- Potential long-term treatment of hemophilia A by neonatal co-transplantation of cord blood-derived endothelial colony-forming cells and placental mesenchymal stromal cells Jan 22, 2019
- Novel therapies for hemophilia A – the role of the von Willebrand factor chaperone Jan 16, 2019
- The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world Jan 3, 2019
- Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy Dec 31, 2018
- Gene therapy for blood diseases Dec 29, 2018
- [Gene therapy of haemophilia – has the cure come within reach?] Dec 29, 2018
Clinical Trials
- Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B Jun 6, 2019
- Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B Jun 4, 2019
- Lentiviral FVIII Gene Therapy for Hemophilia A [YUVA-GT-F801] May 23, 2019
- A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors May 22, 2019
- A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors May 22, 2019
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors May 15, 2019
- Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors May 2, 2019
- Study to evaluate safety, immunogenicity and hemostatic efficacy of PEGylated Recombinant Factor VIII (BAX 855) in previously untreated patients with severe hemophilia A Apr 25, 2019
- Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B Apr 24, 2019
- Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Apr 23, 2019
- Gene Therapy Study in Severe Haemophilia A Patients With Antibodies Against AAV5 Apr 8, 2019
- Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B Apr 3, 2019
- A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study) Apr 3, 2019
- Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial Apr 1, 2019
- Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B Mar 26, 2019
- Hemophilia Inhibitor Prevention and Eradication Clinical Trials (INHIBIT) Platform Mar 26, 2019
- Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX Mar 25, 2019
- A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A Mar 22, 2019
- A Gene Therapy (SPK-9001) Study in Subjects with Hemophilia B Mar 22, 2019
- Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy Mar 21, 2019
- Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A Mar 18, 2019
- A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A Mar 15, 2019
- HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients Mar 14, 2019
- A Gene Transfer Study for Hemophilia A Mar 13, 2019
- Human-cl-rhFVIII in Previously Untreated Patients Mar 13, 2019
- Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors Mar 13, 2019
- FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR) Mar 13, 2019
- Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors Mar 12, 2019
- Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants Mar 11, 2019
- A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors Mar 8, 2019
- Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A Mar 5, 2019
- A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2) Mar 4, 2019
- Factor VIII Gene Therapy Study in Patients With Hemophilia A Feb 28, 2019
- A Study to Investigate the Safety, Tolerability, PK and PD of MG1113 in Healthy Subjects and Hemophilia Patients Feb 27, 2019
- PF-06741086 Long-term Treatment in Severe Hemophilia Feb 27, 2019
- Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE) Feb 18, 2019
- Hemophilia Inhibitor Prevention (INHIBIT) Trial (INHIBIT) Feb 6, 2019
