GTX-102
Phase 1/2- Indications
- Angelman syndrome
Company intelligence / Profile preview
GeneTx Biotherapeutics
Sarasota, Florida, United Statesultragenyx.com ↗
- Ownership
- Public · RARE
- Development stage
- Phase 1
01
Overview
GTX-102, an investigational antisense oligonucleotide for Angelman syndrome, showed promising results in a Phase 1/2 trial. Interim data from 11 children indicated meaningful clinical improvements across multiple domains of Angelman symptoms without serious adverse events. Nine out of eleven evaluable children demonstrated marked improvements in at least three out of five measured domains after 128 days, with no drug-related safety issues reported[9].
- Therapeutic areas
- Neurology
- Modalities
- Antisense oligonucleotides (ASOs)
- Industry
- Biotech
02
Drug pipeline
2 assets1 more asset in the full pipeline.
Request access →03
Partnerships
Ultragenyx Pharmaceuticals – partnered on GTX‑102 development and later acquired the company[4][9]
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