Sapropterin

Sapropterin

SCHEMBL29406211

CC(O)C(O)C1CNc2nc(N)[nH]c(=O)c2N1.Cl.Cl

nearest known ligand 1.00 ✓ in ChEMBL — recovers established targets

Full drug profile on Sugi Atlas →

Known targets — ChEMBL curated mechanism

PAH

The experimentally established mechanism targets of Sapropterin. 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
PAH known ✓ P00439 1/20 0.79
KDM4E B2RXH2 2/20 1.00
MAPT P10636 2/20 1.00
BLM P54132 2/20 1.00
KMT2A Q03164 2/20 1.00
ALDH1A1 P00352 1/20 1.00
GLA P06280 1/20 1.00
HPGD P15428 1/20 1.00
PMP22 Q01453 1/20 1.00
NPSR1 Q6W5P4 1/20 1.00
TDP1 Q9NUW8 1/20 1.00
NOS1 P29475 10/20 0.97
CASP7 P55210 2/20 0.97
HSD17B10 Q99714 2/20 0.97
USP2 O75604 1/20 0.97
NOS3 P29474 3/20 0.61
NOS2 P35228 3/20 0.61
MEN1 O00255 1/20 0.40
RGS12 O14924 1/20 0.40
LMNA P02545 1/20 0.40

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
Sapropterin SCHEMBL31106620 1.00 KDM4E (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL1070424 1.00 KDM4E (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL29059706 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL260185 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL29538547 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL14072498 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL7942378 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL14401407 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL21068033 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1
Sapropterin SCHEMBL29351671 0.99 NOS1 (1.00) KDM4EMAPTBLMKMT2AALDH1A1

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 14 patents. claimed = in the patent's claims; disclosed = body only.

PatentTitleAssigneePublishedPriorityFilingCountryStatus
WO-2025230907-A1 A CXCR4 ANTAGONIST, A STATS ACTIVATOR, AND/OR AN AGENT THAT INCREASES NITRIC OXIDE CONTENT FOR USE IN METHODS OF IMPROVING NERVE REGENERATION THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM (US) 2025-11-06 WO claimed
EP-4426274-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY Baxter International Inc. (US) 2024-09-11 EP claimed
US-20240285831-A1 METHODS OF USE OF MODULATORS TO IMPROVE NERVE REGENERATION KEEFER, Edward (US) 2024-08-29 US claimed
US-11969522-B2 Use of immune modulators to improve nerve regeneration THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM (US) 2024-04-30 US claimed
WO-2023081681-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY BAXTER INTERNATIONAL INC. (US) 2023-05-11 WO claimed
US-20230140515-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY BAXTER INTERNATIONAL INC. 2023-05-04 US claimed
WO-2025230907-A1 A CXCR4 ANTAGONIST, A STATS ACTIVATOR, AND/OR AN AGENT THAT INCREASES NITRIC OXIDE CONTENT FOR USE IN METHODS OF IMPROVING NERVE REGENERATION THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM (US) 2025-11-06 WO disclosed
EP-4426274-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY Baxter International Inc. (US) 2024-09-11 EP disclosed
US-20240285831-A1 METHODS OF USE OF MODULATORS TO IMPROVE NERVE REGENERATION KEEFER, Edward (US) 2024-08-29 US disclosed
US-20240269343-A1 USE OF IMMUNE MODULATORS TO IMPROVE NERVE REGENERATION NERVES INC (US) 2024-08-15 US disclosed
WO-2023081681-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY BAXTER INTERNATIONAL INC. (US) 2023-05-11 WO disclosed
US-20230140515-A1 NITRIC OXIDE DONORS FOR USE IN SURGICAL RECOVERY BAXTER INTERNATIONAL INC. 2023-05-04 US disclosed
EP-3996703-A1 USE OF IMMUNE MODULATORS TO IMPROVE NERVE REGENERATION THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM (US) 2022-05-18 EP disclosed
CN-114126608-A Use of immunomodulators to improve nerve regeneration 得克萨斯州大学系统董事会 2022-03-01 CN disclosed

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

For each of this compound's patents that has machine-readable text (1 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-20240285831-A1 METHODS OF USE OF MODULATORS TO IMPROVE NERVE REGENERATION CXCR3, CXCL12, CXCR4 PAH 4879/4885KDM4E 3304/4885MAPT 4089/4885

“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.