Clinical trials
Recruiting Phase 2 Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
6 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2018 | 1 |
| 2024 | 1 |
| 2025 | 1 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Recruiting | 6 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Biossil Inc. | 1 |
| CSL Behring | 1 |
| Children's Hospital of Philadelphia | 1 |
| GenSight Biologics | 1 |
| National Institute of Blood and Marrow Transplant (NIBMT), Pakistan | 1 |
| PepGen Inc | 1 |
Top conditions
MeSH conditions the trials were registered with, not their broader categories. A condition with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| Genetic Diseases, Inborn | 6 |
| Anemia | 2 |
| Anemia, Sickle Cell | 2 |
| Nervous System Diseases | 2 |
| Anemia, Hemolytic | 1 |
| Anemia, Hemolytic, Congenital | 1 |
| Congenital, Hereditary, and Neonatal Diseases and Abnormalities | 1 |
| Eye Diseases | 1 |
| Hematologic Diseases | 1 |
| Hemic and Lymphatic Diseases | 1 |
Trials
Showing 1 to 6 of 6. Each trial links to its page on Pharma Observatory.
Recently changed trials
Changes observed in the last 7 days, newest first, site-count changes left out. Source: ClinicalTrials.gov.
No changes to these trials in the last 7 days.
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