Haemophilia: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 200+ institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

No stopped trial with a stated reason in this view.

Trials whose registry record says individual participant data (IPD) will be shared.

• 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

  NCT03861273· Pfizer· Source

• A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

  NCT07416604· Hoffmann-La Roche· Source

• A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A

  NCT07830732· Novo Nordisk A/S· Source

• A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

  NCT07285460· Sanofi· Source

• A Study to Describe Patient REal-world FitusiRan ExpeRience

  NCT07895381· Sanofi· Source

• Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

  NCT05145127· Pfizer· Source

• A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

  NCT07416526· Hoffmann-La Roche· Source

• A Non-Investigational Product, Multi-Center Cohort Study to Describe Long-Term Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec

  2025-523660-21-00· Pfizer Inc.· Source

• Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products

  NCT06738485· CSL Behring· Source

• A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B

  NCT06379789· Regeneron Pharmaceuticals· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Single-Cell Analysis 1
• CRISPR-Cas Systems 6
• Gene Expression Profiling 1
• Sequence Analysis, RNA 1
• Whole Genome Sequencing 2
• Proteomics 1
• Flow Cytometry 1
• Immunohistochemistry 1
• Magnetic Resonance Imaging 6
• Machine Learning 3
• Deep Learning 2
• Cohort Studies 9

See which targets of this disease your abstract mentions, and go to the people working on them.