Key players in AAV Gene Therapy
In the order our report profiles them, not ranked by sales. Each name opens the company's page with its public records.
| # | Company | Website | Our reports |
|---|---|---|---|
| 1 |  Novartis AG |
novartis.com (opens in a new tab) | 1,551 reports |
| 2 | Sarepta Therapeutics | — | 66 reports |
| 3 | BioMarin | — | 165 reports |
| 4 | CSL Behring | — | 231 reports |
| 5 | Ultragenyx | — | 33 reports |
| 6 | REGENXBIO | — | 33 reports |
Also active in AAV Gene Therapy
Other companies that free public records show in this market: trials they lead, US approvals and WHO prequalification. They are not part of our key players research. Each count opens its source.
| Company | Where public records show them |
|---|---|
| Adverum Biotechnologies, Inc. |
|
| Beacon Therapeutics |
|
| Tenaya Therapeutics |
|
| uniQure |
|
| Innopeutics Corporation |
|
| Solid Biosciences Inc. |
|
| Brain Neurotherapy Bio, Inc. |
|
| Augelux Therapeutics |
|
Source: ClinicalTrials.gov, Active studies (recruiting, about to start or running) each company leads, found with this market's trial search, worldwide. Retrieved 13 Nov 2026.
Top trial sponsors in AAV Gene Therapy
Companies leading the most active studies found with this market's trial search, worldwide (key players included).
- Adverum Biotechnologies, Inc. 6 active trials on ClinicalTrials.gov (opens in a new tab)
- Ultragenyx 4 active trials on ClinicalTrials.gov (opens in a new tab)
- Beacon Therapeutics 4 active trials on ClinicalTrials.gov (opens in a new tab)
- Tenaya Therapeutics 3 active trials on ClinicalTrials.gov (opens in a new tab)
- uniQure 3 active trials on ClinicalTrials.gov (opens in a new tab)
- Innopeutics Corporation 2 active trials on ClinicalTrials.gov (opens in a new tab)
- Solid Biosciences Inc. 2 active trials on ClinicalTrials.gov (opens in a new tab)
- Brain Neurotherapy Bio, Inc. 2 active trials on ClinicalTrials.gov (opens in a new tab)
Source: ClinicalTrials.gov, Industry lead sponsors of active studies found with this market's trial search, worldwide. Retrieved 13 Nov 2026.
AAV Gene Therapy reports by place
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India
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Company profiles, market shares and strategies come with the full report, with researched size, forecast, growth and segments.
Key players are our report's research. Other companies and trial counts come from free public records, each with its source. Acuitas market figures are not published on this page.