Clinical trials
Phase 1/2 Genetic Diseases, Inborn trials in the United States
Search ClinicalTrials.gov registrations by keyword, then narrow by condition, phase, status, country and start year.
26 trials match Source: ClinicalTrials.gov registrations
Trials started per year
By phase, 2000 to 2026.
| Category | Trials |
|---|---|
| 2008 | 1 |
| 2010 | 1 |
| 2012 | 1 |
| 2015 | 1 |
| 2016 | 1 |
| 2017 | 1 |
| 2019 | 2 |
| 2020 | 2 |
| 2021 | 5 |
| 2022 | 2 |
| 2023 | 1 |
| 2024 | 3 |
| 2025 | 1 |
Status
Trials by registry status. Select a bar to filter.
| Category | Trials |
|---|---|
| Completed | 10 |
| Active, not recruiting | 7 |
| Recruiting | 3 |
| Not yet recruiting | 2 |
| Terminated | 2 |
| Unknown status | 1 |
| Enrolling by invitation | 1 |
Top lead sponsors
A sponsor with a page on Pharma Observatory links to it.
| Category | Trials |
|---|---|
| ModernaTX, Inc. | 5 |
| Editas Medicine, Inc. | 2 |
| Spark Therapeutics, Inc. | 2 |
| University of California, San Diego | 2 |
| 4D Molecular Therapeutics | 1 |
| Agnieszka Czechowicz | 1 |
| Alexion Pharmaceuticals, Inc. | 1 |
| BioMarin Pharmaceutical | 1 |
| Children's Hospital Los Angeles | 1 |
| DNage B.V. | 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 | 26 |
| Metabolic Diseases | 7 |
| Metabolism, Inborn Errors | 7 |
| Hematologic Diseases | 6 |
| Anemia, Sickle Cell | 5 |
| Lysosomal Storage Diseases | 4 |
| Blood Coagulation Disorders | 3 |
| Blood Coagulation Disorders, Inherited | 3 |
| Coagulation Protein Disorders | 3 |
| Hemophilia A | 3 |
Trials
Showing 1 to 25 of 26. 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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