Ompenaclid

DB18819InvestigationalSmall molecule

RGX-202 is a recombinant AAV8 that contains a vector genome encoding a miniaturized dystrophin protein (microdystrophin).

Chemical structure of Ompenaclid
Mechanism
Curator reviewed
Formula / weight
C4H9N3O2 · 131.135 g/mol (avg)
Code names
PNU-10483 · RGX-202 · RGX-202-01

Resolves to

Compound
UNIIUL1984YRKACAS353-09-3ChEMBLCHEMBL20489ChEBI57593PubChem67701KEGGC03065
Structure
InChIKeyKMXXSJLYVJEBHI-UHFFFAOYSA-NSMILESNC(=N)NCCC(O)=O
Targets
P48029SLC6A8

What you can answer from here — as of September 23, 2026

1Protein targetEach mapped to UniProt, with action and pharmacological action
Listed above
4Clinical trialsPhase, status and sponsor resolved per trial
Sign in
4+ReferencesStructured and connected to the statements they support
Sign in

Which drugs share a target with Ompenaclid, and which of those have an active Phase 3 trial?

Answer this in Biopharma OSSign in to your account

Create a free account or log in to explore the full data card.

Create Account
Log InPaid Account Log In