Pipeline
Designed to achieve a significant therapeutic advantage, our current pipeline explores AAV-mediated approaches, novel immunomodulation strategies, and dual routes of administration.
| Discovery | Preclinical | Phase I | Phase II | Commercial |
|---|---|---|---|---|
| Discovery | Preclinical | Phase I | Phase II | Commercial |
|
Friedreich’s ataxia
(Preclinical)
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Undisclosed
(Preclinical)
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Undisclosed
(Discovery)
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Undisclosed
(Discovery)
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As we move forward, expect to see a diversified pipeline, including emerging technologies that will be evaluated for their potential to target underlying disease and help bring groundbreaking genetic therapies to more patients
