PTC-124 | 9421

PTC-124 has been reported to help the ribosome skip over the premature stop, restore production of functional protein, and thereby potentially treat these genetic diseases. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function.

PTC-124 has been reported to help the ribosome skip over the premature stop, restore production of functional protein, and thereby potentially treat these genetic diseases. In a mouse model of cystic fibrosis caused by nonsense mutations, PTC-124 treatment has been shown to restore cystic fibrosis transmembrane conductance regulator (CFTR) protein expression and function.

9421 | PTC-124 DataSheet

Alternate Name/Synonyms: 3-​[5-​(2-​fluorophenyl)-​1,​2,​4-​oxadiazol-​3-​yl]-​benzoic acid

Appearance: White to off-white solid

Formulation: N/A

CAS Number: 775304-57-9

Structure Available?: Yes

Peptide sequence: N/A

Salt Form: No

Molecular Formula: C₁₅H₉FN₂O₃

Molecular Weight: 284.24

Cell-Permeable?: Yes

Purity: ≥98% by HPLC

Solubilities: DMSO (~50 mg/ml)

Handling: Protect from air and light

Country of Origin: USA

Tag Line: CFTR-G542X nonsense allele inhibitor

MDL Number: MFCD09864996

PubChem CID: 11219835

SMILES: C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F

InChi: InChI=1S/C15H9FN2O3/c16-12-7-2-1-6-11(12)14-17-13(18-21-14)9-4-3-5-10(8-9)15(19)20/h1-8H,(H,19,20)

InChi Key: OOUGLTULBSNHNF-UHFFFAOYSA-N