Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AVANCE SQY51
- Development stage
- Unknown
- Lead developer
- Sqy Therapeutics
- 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
- Intravenous
01
Overview
AVANCE SQY51 (also known as SQY51) is a palmitoyl-conjugated tricyclo-DNA (tc-DNA) antisense oligonucleotide (ASO) designed for the treatment of Duchenne muscular dystrophy (DMD). Developed by Sqy Therapeutics in collaboration with Inserm, the Université de Versailles Saint-Quentin-en-Yvelines, and Université Paris-Saclay, the drug targets the pre-mRNA of the DMD gene to induce exon 51 skipping. This mechanism restores the reading frame of the mutated DMD gene, allowing for the production of a truncated but functional dystrophin protein. AVANCE SQY51 is currently being evaluated in the Phase 1/2a AVANCE-1 clinical trial for DMD patients amenable to exon 51 skipping.
02
Targets
DMD (Dystrophin)Neuraminidase
Beyond the preview
Go deeper on AVANCE SQY51.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect 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 AVANCE SQY51.