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ASK
Research Triangle Park, North Carolina, USA
- Ownership
- Private
- Development stage
- Other
01
Overview
Initial safety data from the Phase 1/Phase 2 LION-CS101 trial for AB-1003 in LGMD2I/R9 showed no dose-limiting toxicities or serious adverse events over 52 weeks post-treatment, with mild-to-moderate side effects and transient, asymptomatic transaminase elevations resolving after corticosteroid adjustment[1].
- Therapeutic areas
- Rare Diseases
- Modalities
- Gene therapyAAV-based therapyBiologic
- Industry
- Pharma
02
Partnerships
Bayer AG
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