Clinical trials
Enrolling by invitation Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
15 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2017 | 1 |
| 2020 | 1 |
| 2021 | 4 |
| 2023 | 1 |
| 2024 | 2 |
| 2025 | 3 |
| 2026 | 3 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Enrolling by invitation | 15 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| QED Therapeutics, a BridgeBio company | 3 |
| TESS Research Foundation | 2 |
| Vertex Pharmaceuticals Incorporated | 2 |
| BioMarin Pharmaceutical | 1 |
| Editas Medicine, Inc. | 1 |
| Jerry Vockley, MD, PhD | 1 |
| Massachusetts General Hospital | 1 |
| Mayo Clinic | 1 |
| Rady Pediatric Genomics & Systems Medicine Institute | 1 |
| University of California, San Francisco | 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 | 15 |
| Musculoskeletal Diseases | 4 |
| Bone Diseases | 3 |
| Hematologic Diseases | 3 |
| Mucopolysaccharidosis IV | 3 |
| Osteochondrodysplasias | 3 |
| Rare Diseases | 3 |
| Anemia, Sickle Cell | 2 |
| Dwarfism | 2 |
| Epilepsy | 2 |
Trials
Showing 1 to 15 of 15. 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.
Go deeper on Pharma Observatory
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