A transforming therapeutic landscape worth monitoring daily
Hemophilia is one of the most commercially active rare disease therapeutic areas, undergoing a fundamental transformation. The approval of gene therapies for hemophilia A (fitusiran, valoctocogene roxaparvovec) and hemophilia B (etranacogene dezaparvovec) has validated the approach commercially, while next-generation programs seek to improve durability and expand eligible patient populations. Simultaneously, non-factor replacement therapies — led by emicizumab — continue expanding their role in the treatment paradigm.
Key signals in the hemophilia pipeline in 2026:
- Next-generation AAV gene therapy programs for hemophilia A (Factor VIII deficiency) with improved durability and seroprevalence coverage
- Hemophilia B (Factor IX deficiency) gene therapy programs and long-term follow-up studies for approved products
- Non-factor replacement therapies: bispecific antibodies, fitusiran (antithrombin targeting), and rebalancing agents
- Inhibitor management programs — patients who develop inhibitors to factor replacement represent a particularly challenging and commercially important subgroup
- Gene editing approaches (CRISPR, base editing) for hemophilia as curative strategies
- Pediatric programs: eligibility expansion for gene therapy and non-factor approaches to younger patients
- Real-world evidence and long-term safety studies following gene therapy approvals
Get daily hemophilia trial alerts
Set your profile once. Receive a filtered daily digest of new and updated hemophilia trials. free plan.
Get Free AlertsWhat DataLookout monitors for hemophilia
Configure your profile with condition and mechanism keywords matched to your specific intelligence priorities:
- Condition keywords: "hemophilia A", "hemophilia B", "hemophilia", "Factor VIII deficiency", "Factor IX deficiency", "von Willebrand disease"
- Mechanism keywords: "gene therapy", "AAV", "Factor VIII", "Factor IX", "emicizumab", "fitusiran", "non-factor", "bispecific antibody"
- Subtype specific: "hemophilia with inhibitors", "severe hemophilia", "moderate hemophilia"
- Phase filter: Phase 2/3 for commercial and BD intelligence, or all phases for comprehensive pipeline coverage
- Sponsor filter: Industry-sponsored for competitive tracking, NIH/academic for emerging science
What DataLookout adds to ClinicalTrials.gov
ClinicalTrials.gov RSS feeds tell you that a study record was updated. DataLookout compares each record against the previous day across 12 tracked fields and tells you which one changed, with the old and new values, for every Hemophilia trial on your watchlist. Each change is classified as critical, notable, or minor, so a trial quietly adding a dozen new sites does not read the same as a contact-details edit.
Who uses hemophilia trial monitoring
Rare disease pharma BD and strategy teams
Companies active in hemophilia — spanning gene therapy, protein engineering, and novel mechanisms — track new trial registrations to identify partnership opportunities and anticipate competitive dynamics. The gene therapy landscape in hemophilia is evolving rapidly, with durability, immune response management, and patient selection all active areas of clinical investigation.
Hematology biotech analysts and investors
Investors covering rare hematology use hemophilia trial registrations as leading indicators of pipeline development. Gene therapy program phase transitions and non-factor therapy expansion into new patient populations represent significant investment signals that daily trial monitoring enables.
Hemophilia treatment centers and medical affairs teams
Hemophilia treatment centers (HTCs) and medical affairs professionals at hemophilia companies track new trial registrations to understand the competitive landscape, identify opportunities for site participation in upcoming trials, and stay current on emerging evidence in gene therapy long-term outcomes.
National Hemophilia Foundation and patient advocacy
Patient advocacy organizations monitor the clinical trial pipeline to connect patients — particularly those with inhibitors or those who are not candidates for current gene therapy programs due to pre-existing AAV antibodies — with appropriate clinical study opportunities.
Frequently asked questions
How current is the hemophilia trial data?
Our pipeline fetches from ClinicalTrials.gov every morning. Studies posted or updated in the preceding 24 hours appear in that day's digest. ClinicalTrials.gov is updated as sponsors register new trials or submit protocol amendments, typically within 24–48 hours.
Can I track hemophilia A and hemophilia B programs separately?
Yes. On the Pro plan ($149/month), you can create separate search profiles — for example, one focused on hemophilia A gene therapy and another monitoring hemophilia B programs and non-factor therapies — each delivering a focused daily digest.
Does DataLookout cover gene therapy long-term follow-up studies?
Yes. Long-term follow-up (LTFU) studies required after gene therapy approval are registered on ClinicalTrials.gov and will appear in your digest when matching your profile keywords.
Does DataLookout cover von Willebrand disease trials?
Yes — adding "von Willebrand disease" or "VWD" to your profile keywords will surface relevant clinical trials, which are often conducted alongside or in comparison to hemophilia programs.
178 active trials, 90 recruiting. Phases: Early Phase 1: 2, Phase 1: 22, Phase 2: 20, Phase 3: 40, Phase 4: 14.
| Top sponsor | Trials |
|---|---|
| Novo Nordisk | 15 |
| Bayer | 7 |
| Pfizer | 7 |
| Sanofi / Regeneron | 7 |
| CSL Behring | 6 |
| Trial | Status | Latest tracked change | Seen |
|---|---|---|---|
| Lentiviral FIX Gene Therapy NCT03961243 | Not Yet Recruiting | Completion pushed: 2028-06-01 → 2028-12-31 | 2026-08-27 |
| Ultrasound Assessment of Joint Health in Patients With Mild Hemophilia (Factor… NCT07719647 | Not Yet Recruiting | Completion pushed: 2027-09 → 2028-02 | 2026-08-18 |
| Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients NCT06922045 | Active, Not Recruiting | Enrollment closed, study ongoing | 2026-08-13 |
| Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs… NCT04278404 | Recruiting | Completion moved earlier: 2027-07 → 2026-12 | 2026-08-22 |
| ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders NCT04398628 | Recruiting | Trial sites expanded: 71 → 76 locations | 2026-08-18 |
Each trial page shows every change DataLookout has recorded for that trial.
DataLookout checks ClinicalTrials.gov every day and records what changed on each trial: status, enrollment, phase, primary endpoint, completion dates, sites, sponsor, and the stated reason a trial stopped. Add a sponsor or a disease to your watchlist, or save any search, and the changes arrive in a daily or weekly email digest.
Free, $0 forever: 1 sponsor + 1 disease watchlist, 1 saved search with email alerts, track up to 5 individual trials, daily change detection, Trials at Risk (top trial). No credit card.
Starter, $49/month: 3 sponsor + 3 disease watchlists, 3 saved searches with email alerts, track up to 25 individual trials, full Trials at Risk list and landscape reports.
Pro, $149/month: unlimited sponsor and disease watchlists, unlimited saved searches, track unlimited individual trials, CSV export, priority email support.