Ataluren

Ataluren

SCHEMBL60614

O=C(O)c1cccc(-c2noc(-c3ccccc3F)n2)c1

nearest known ligand 1.00 ✓ in ChEMBL — recovers established targets

Full drug profile on Sugi Atlas →

Known targets — ChEMBL curated mechanism

FAURPL10RPL10ARPL11RPL12RPL13RPL13ARPL14RPL15RPL17RPL18RPL18ARPL19RPL21RPL22RPL23RPL23ARPL24RPL26RPL27RPL27ARPL28RPL29RPL3RPL30RPL31RPL32RPL34RPL35RPL35ARPL36RPL36ARPL37RPL37ARPL38RPL39RPL4RPL41RPL5RPL6RPL7RPL7ARPL8RPL9RPL9P7RPL9P8RPL9P9RPLP0RPLP1RPS10RPS11RPS12RPS13RPS14RPS15RPS15ARPS16RPS17RPS18RPS19RPS2RPS20RPS21RPS23RPS24RPS25RPS26RPS27RPS27ARPS28RPS29RPS3RPS3ARPS4XRPS4Y1RPS5RPS6RPS7RPS8RPS9RPSA

The experimentally established mechanism targets of Ataluren. The predicted profile below is derived independently by chemical similarity — agreement is a validation signal, a miss is honest.

Predicted protein targets (top 20)

geneUniProtsupporting neighboursconfidence
MAOA P21397 1/20 1.00
BLVRB P30043 1/20 1.00
HDAC6 Q9UBN7 1/20 1.00
NPC1 O15118 13/20 0.79
RAB9A P51151 13/20 0.79
KMT2A Q03164 5/20 0.69
SMN1; SMN2 Q16637 5/20 0.69
MEN1 O00255 4/20 0.69
L3MBTL1 Q9Y468 3/20 0.69
NFKB1 P19838 3/20 0.69
NFKB2 Q00653 3/20 0.69
RELA Q04206 3/20 0.69
PKM P14618 2/20 0.69
MAPK1 P28482 2/20 0.69
NR1H4 Q96RI1 1/20 0.62
MAPT P10636 2/20 0.52
TP53 P04637 1/20 0.52
GAA P10253 1/20 0.51
GPR55 Q9Y2T6 1/20 0.50
CHUK O15111 1/20 0.50

Click a target to see other patent compounds predicted against it — the reverse direction, in place.

Similar compounds — the chemically nearest patent molecules

Nearest neighbours by Morgan-fingerprint cosine across the patent-compound collection, with each neighbour's top predicted target and the predicted targets it shares with this molecule.

Compoundsimilaritytop predictedshared targets
Ataluren SCHEMBL29360108 1.00 MAOA (1.00) MAOABLVRBHDAC6NPC1RAB9A
Ataluren SCHEMBL29365205 1.00 MAOA (1.00) MAOABLVRBHDAC6NPC1RAB9A
Ataluren SCHEMBL29361641 1.00 MAOA (1.00) MAOABLVRBHDAC6NPC1RAB9A
Ataluren SCHEMBL59167 0.99 MAOA (0.97) MAOABLVRBHDAC6NPC1RAB9A
Ataluren SCHEMBL59165 0.99 MAOA (0.97) MAOABLVRBHDAC6NPC1RAB9A
SCHEMBL60044 0.91 MAOA (0.83) MAOABLVRBHDAC6NPC1RAB9A
SCHEMBL1505230 0.90 MAOA (0.81) MAOABLVRBHDAC6NPC1RAB9A
SCHEMBL60083 0.89 HDAC6 (0.80) MAOABLVRBHDAC6NPC1RAB9A
SCHEMBL1752479 0.88 NPC1 (1.00) MAOABLVRBHDAC6NPC1RAB9A
SCHEMBL17946207 0.88 MAOA (0.79) MAOABLVRBHDAC6NPC1RAB9A

Similarity is cosine over the 2,048-bit Morgan fingerprint (≈ Tanimoto). Identical fingerprints score 1.00.

Patent provenance — the patents this molecule appears in, and who filed them

Claimed or disclosed in 1334 patents — showing the first 20. claimed = in the patent's claims; disclosed = body only.

PatentTitleAssigneePublishedPriorityFilingCountryStatus
US-20250228242-A1 PROTEIN AGGREGATION INHIBITING COMPOUNDS FOR PLANT DISEASE CONTROL MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. (DE) 2025-07-17 US claimed
WO-2025110781-A1 BIOMARKER COMPOSITION COMPRISING AMPD3 FOR PREDICTING SENSITIVITY TO THERAPEUTIC AGENT FOR NEUROFIBROMATOSIS, AND PHARMACEUTICAL COMPOSITION COMPRISING AMPD3 INHIBITOR FOR PREVENTING OR TREATING NEUROFIBROMATOSIS 재단법인 아산사회복지재단 2025-05-30 WO claimed
US-20250137059-A1 ACTIVATED RAS AS THERAPEUTIC AND DIAGNOSTIC TARGET FOR NEUROFIBROMATOSIS AND USE THEREOF THE ASAN FOUNDATION (KR) 2025-05-01 US claimed
EP-4473971-A2 METHOD FOR TREATING NONSENSE MUTATION MEDIATED DUCHENNE MUSCULAR DYSTROPHY IN PEDIATRIC PATIENTS PTC Therapeutics, Inc. (US) 2024-12-11 EP claimed
EP-4455670-A1 ACTIVATED RAS AS THERAPEUTIC AND DIAGNOSTIC TARGET FOR NEUROFIBROMATOSIS AND USE THEREOF The Asan Foundation (KR) 2024-10-30 EP claimed
US-20240352461-A1 RESTORATION OF THE CFTR FUNCTION BY SPLICING MODULATION YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD. (IL) 2024-10-24 US claimed
CN-114920667-B Electrochemical preparation method of atta Lu Lunzhi 湖南大学 2024-06-21 CN claimed
EP-3591052-B1 RESTORATION OF THE CFTR FUNCTION BY SPLICING MODULATION YISSUM RES DEV CO OF HEBREW UNIV JERUSALEM LTD (IL) 2023-08-30 EP claimed
EP-4101846-B1 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS AND THEIR USE FOR NONSENSE SUPPRESSION AND THE TREATMENT OF DISEASE PTC THERAPEUTICS INC (US) 2023-08-02 EP claimed
US-20230218587-A1 COMPOSITIONS FOR AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR THE TREATMENT OF DISEASE PTC THERAPEUTICS INC (US) 2023-07-13 US claimed
WO-2008039431-A2 CRYSTALLINE FORMS OF 3-[5-(2-FHJOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID PTC THERAPEUTICS, INC. (US) 2008-04-03 WO claimed
EP-1874306-A1 COMPOSITIONS OF AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENSE MUTATION SUPPRESSION THERAPY PTC Therapeutics, Inc. (US) 2008-01-09 EP claimed
US-20070161687-A1 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid; translation of mRNA is allowed to continue past a nonsense mutation point, resulting in full length protein; genetic disorders; testing whether a compound increases activity of a protein encoded by an mRNA that contains a premature stop codon PTC THERAPEUTICS, INC. 2007-07-12 US claimed
US-7202262-B2 E.g., 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid; translation of mRNA is allowed to continue past a nonsense mutation point, resulting in the production of a full length protein; genetic disorders PTC THERAPEUTICS, INC. (US) 2007-04-10 US claimed
WO-2006110483-A1 COMPOSITIONS OF AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR NONSENSE MUTATION SUPPRESSION THERAPY PTC THERAPEUTICS, INC. (US) 2006-10-19 WO claimed
CN-1802360-A 1,2, 4-oxadiazole benzoic acid compounds PTC THERAPEUTICS INC (US) 2006-07-12 CN claimed
US-20060148864-A1 Treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay such as genetic disorders PTC THERAPEUTICS, INC. 2006-07-06 US claimed
US-6992096-B2 translation of mRNA is allowed to continue past a nonsense mutation point, resulting in the production of a full length protein; genetic disorders; 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid PTC THERAPEUTICS, INC. (US) 2006-01-31 US claimed
US-20050164973-A1 E.g., 3-[5-(4-Fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid; treating diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay PCT THERAPEUTICS, INC. 2005-07-28 US claimed
US-20040204461-A1 translation of mRNA is allowed to continue past a nonsense mutation point, resulting in the production of a full length protein; genetic disorders; 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid PTC THERAPEUTICS, INC. 2004-10-14 US claimed

Patent text — is the patent's own abstract consistent with the prediction?

For each of this compound's patents that has machine-readable text (7 of them — usually the abstract, not the full specification), we ask MedCPT which protein the text reads most about, and where the chemistry-predicted target lands among 4885 human targets. A high rank means the patent's own wording is consistent with the prediction — a weak, independent signal, not proof of activity.

PatentTitleText reads most aboutPredicted target · text-rank
US-20230218587-A1 COMPOSITIONS FOR AN ORALLY ACTIVE 1,2,4-OXADIAZOLE FOR THE TREATMENT OF DISEASE SMN1; SMN2, UPF1, VHL MAOA 2571/4885BLVRB 2211/4885HDAC6 396/4885
US-20060148864-A1 Treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay such as genetic disorders UPF1, PABPC1, PABPC4 MAOA 3474/4885BLVRB 2415/4885HDAC6 620/4885
US-20250228242-A1 PROTEIN AGGREGATION INHIBITING COMPOUNDS FOR PLANT DISEASE CONTROL APP, APBA1, PRNP MAOA 1118/4885BLVRB 1212/4885HDAC6 2055/4885
US-20070161687-A1 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid; translation of mRNA is allowed to continue past a nonsense mutation point, resulting in full length protein; genetic disorders; testing whether a compound increases activity of a protein encoded by an mRNA that contains a premature stop codon UPF1, PABPC4, PABPC1 MAOA 3625/4885BLVRB 2775/4885HDAC6 2378/4885
US-20250137059-A1 ACTIVATED RAS AS THERAPEUTIC AND DIAGNOSTIC TARGET FOR NEUROFIBROMATOSIS AND USE THEREOF NRAS, KRAS, RASGRP1 MAOA 3897/4885BLVRB 2199/4885HDAC6 4788/4885
US-20050164973-A1 E.g., 3-[5-(4-Fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid; treating diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay UPF1, PABPC1, PABPC4 MAOA 2579/4885BLVRB 2649/4885HDAC6 659/4885
US-20040204461-A1 translation of mRNA is allowed to continue past a nonsense mutation point, resulting in the production of a full length protein; genetic disorders; 3-(5-p-tolyl-[1,2,4]oxadiazol-3-yl)-benzoic acid UPF1, PABPC1, PABPC4 MAOA 3758/4885BLVRB 3084/4885HDAC6 1524/4885

“Text reads most about” is the patent abstract's nearest protein in MedCPT space (background-debiased). Only ~1.4% of patents have machine-readable text, so most compounds won't have this panel.