AB-1009
Asset
Delivers a functional GAA gene to restore acid alpha-glucosidase activity and reduce lysosomal glycogen accumulation.
- Stage
- Phase 1
- Modality
- Gene therapy
- Target
- GAA
- 3
- Indications
- 1
- Trial
- 1
- Developer
Indications
3LOPD
Phase 1, Recruiting, NCT07282847, clinicaltrials.gov, 16 Oct 2026
Pompe disease
Phase 1, Recruiting, NCT07282847, clinicaltrials.gov, 16 Oct 2026
Pompe disease late-onset
Phase 1, Recruiting, NCT07282847, clinicaltrials.gov, 16 Oct 2026
In the full profile
- Targets and what it contains1
- Owner and partners2
- Trials1
- Evidence for its targets1
- Competitors2
- Signals3
Every asset’s record: target, stage, trials and the evidence behind it.