Clinical trials
Enrolling by invitation Genetic Diseases, Inborn trials
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
17 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2017 | 1 |
| 2019 | 2 |
| 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 | 17 |
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 |
| Karolinska Institutet | 1 |
| Massachusetts General Hospital | 1 |
| Mayo Clinic | 1 |
| Rady Pediatric Genomics & Systems Medicine Institute | 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 | 17 |
| Musculoskeletal Diseases | 4 |
| Rare Diseases | 4 |
| Bone Diseases | 3 |
| Hematologic Diseases | 3 |
| Mucopolysaccharidosis IV | 3 |
| Osteochondrodysplasias | 3 |
| Anemia, Sickle Cell | 2 |
| Dwarfism | 2 |
| Epilepsy | 2 |
Trials
Showing 26 to 17 of 17. Each trial links to its page on Pharma Observatory.
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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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