Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
apazunersen
- Development stage
- Phase 3
- Lead developer
- Ultragenyx Pharmaceutical
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
Apazunersen is an **antisense oligonucleotide** currently in Phase 3 clinical trials for the treatment of **Angelman syndrome**. It is designed to target the underlying genetic cause of the disorder by inhibiting the mechanism that silences the paternal UBE3A gene, thus stimulating UBE3A protein expression. Apazunersen is administered via **intrathecal injection** and is under development by **GeneTx Biotherapeutics** (a subsidiary of Ultragenyx Pharmaceutical) and Ultragenyx Pharmaceutical. The drug has received multiple designations including Breakthrough Therapy, Orphan Drug, Fast Track (US), Orphan Drug and PRIME (EU), and Rare Pediatric Disease. The primary endpoint in current studies is cognitive improvement measured by the Bayley-4 cognitive raw score.
- Other names
- apazunersenGTX-102GTX102GTX 102
02
Targets
UBE3A-ATS (UBE3A antisense transcript)
Beyond the preview
Go deeper on apazunersen.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on apazunersen.