Known targets — ChEMBL curated mechanism
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)
| gene | UniProt | supporting neighbours | confidence | |
|---|---|---|---|---|
| ▸ | PAH known ✓ | P00439 | 1/20 | 0.60 |
| ▸ | NOS1 | P29475 | 6/20 | 0.64 |
| ▸ | CASP7 | P55210 | 2/20 | 0.64 |
| ▸ | HSD17B10 | Q99714 | 2/20 | 0.64 |
| ▸ | USP2 | O75604 | 1/20 | 0.64 |
| ▸ | KDM4E | B2RXH2 | 2/20 | 0.62 |
| ▸ | MAPT | P10636 | 2/20 | 0.62 |
| ▸ | BLM | P54132 | 2/20 | 0.62 |
| ▸ | KMT2A | Q03164 | 2/20 | 0.62 |
| ▸ | ALDH1A1 | P00352 | 1/20 | 0.62 |
| ▸ | GLA | P06280 | 1/20 | 0.62 |
| ▸ | HPGD | P15428 | 1/20 | 0.62 |
| ▸ | PMP22 | Q01453 | 1/20 | 0.62 |
| ▸ | NPSR1 | Q6W5P4 | 1/20 | 0.62 |
| ▸ | TDP1 | Q9NUW8 | 1/20 | 0.62 |
| ▸ | NOS3 | P29474 | 2/20 | 0.51 |
| ▸ | NOS2 | P35228 | 2/20 | 0.51 |
| ▸ | MEN1 | O00255 | 1/20 | 0.30 |
| ▸ | RGS12 | O14924 | 1/20 | 0.30 |
| ▸ | LMNA | P02545 | 1/20 | 0.30 |
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.
| Compound | similarity | top predicted | shared targets | |
|---|---|---|---|---|
| Sapropterin SCHEMBL20769839 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL37480 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL25915067 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL28826011 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL25915163 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL258544 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL1534380 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL31673726 | 0.99 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL21046203 | 0.97 | KDM4E (0.66) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL258963 | 0.97 | KDM4E (0.66) | NOS1CASP7HSD17B10USP2KDM4E |
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 15 patents. claimed = in the patent's claims; disclosed = body only.
| Patent | Title | Assignee | Published | Priority | Filing | Country | Status |
|---|---|---|---|---|---|---|---|
| CN-122075696-A | Application of MSR1 inhibitor in preparation of anti-tumor immunopotentiator | — | 2026-05-26 | — | — | CN | disclosed |
| US-20260102359-A1 | COMBINATION TREATMENT OF DERMAL AND TRANSDERMAL FIBROTIC DISEASES, DISORDERS AND ASSOCIATED PAIN AND INFLAMMATION | ERESINA LLC (US) | 2026-04-16 | — | — | US | disclosed |
| US-20250186537-A1 | TREATMENT OF WOMEN AFFECTED WITH POLYCYSTIC OVARY SYNDROME | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (FR) | 2025-06-12 | — | — | US | disclosed |
| US-12208130-B2 | Treatment of women affected with polycystic ovary syndrome | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (FR) | 2025-01-28 | — | — | US | disclosed |
| CN-113727733-B | Triamterene or nolatrexed for treating phenylketonuria | 索姆创新生物技术公司 | 2023-01-24 | — | — | CN | disclosed |
| US-20220257695-A1 | A GNRH ANTAGONIST FOR USE IN THE TREATMENT OF A WOMEN AFFECTED WITH POLYCYSTIC OVARY SYNDROME | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (FR) | 2022-08-18 | — | — | US | disclosed |
| US-20220133726-A1 | TRIAMTERENE OR NOLATREXED FOR USE IN THE TREATMENT OF PHENYLKETONURIA | SOM INNOVATION BIOTECH, S.A. (ES) | 2022-05-05 | — | — | US | disclosed |
| EP-3923988-A1 | TRIAMTERENE OR NOLATREXED FOR USE IN THE TREATMENT OF PHENYLKETONURIA | Som Innovation Biotech, S.A. (ES) | 2021-12-22 | — | — | EP | disclosed |
| WO-2020165318-A1 | TRIAMTERENE OR NOLATREXED FOR USE IN THE TREATMENT OF PHENYLKETONURIA | SOM INNOVATION BIOTECH, S.A. (ES) | 2020-08-20 | — | — | WO | disclosed |
| EP-3600373-A1 | A GNRH ANTAGONIST FOR USE IN THE TREATMENT OF A WOMEN AFFECTED WITH POLYCYSTIC OVARY SYNDROME | Institut National de la Santé et de la Recherche Medicale (FR) | 2020-02-05 | — | — | EP | disclosed |
| WO-2018177746-A1 | A GNRH ANTAGONIST FOR USE IN THE TREATMENT OF A WOMEN AFFECTED WITH POLYCYSTIC OVARY SYNDROME | INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM) (FR) | 2018-10-04 | — | — | WO | disclosed |
| WO-2015022386-A1 | COMBINED PHARMACEUTICAL PREPARATION FOR USE IN TREATING NEUROMUSCULAR DISORDERS | UNIVERSITÄTS-KINDERSPITAL BEIDER BASEL (CH) | 2015-02-19 | — | — | WO | disclosed |
| EP-2837390-A1 | Combined Pharmaceutical Preparation for Use in Treating Neuromuscular Disorders | Universitäts-Kinderspital beider Basel (CH) | 2015-02-18 | — | — | EP | disclosed |
| US-7927613-B2 | Pharmaceutical co-crystal compositions | UNIVERSITY OF SOUTH FLORIDA (US) | 2011-04-19 | — | — | US | disclosed |
| US-7790905-B2 | Pharmaceutical propylene glycol solvate compositions | MCNEIL-PPC, INC. (US) | 2010-09-07 | — | — | US | 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 (2 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.
| Patent | Title | Text reads most about | Predicted target · text-rank |
|---|---|---|---|
| US-20220133726-A1 | TRIAMTERENE OR NOLATREXED FOR USE IN THE TREATMENT OF PHENYLKETONURIA | TPMT, MPST, SLC5A6 | PAH 4/4885NOS1 362/4885CASP7 2935/4885 |
| US-20260102359-A1 | COMBINATION TREATMENT OF DERMAL AND TRANSDERMAL FIBROTIC DISEASES, DISORDERS AND ASSOCIATED PAIN AND INFLAMMATION | COL2A1, COLGALT1, PLOD3 | PAH 452/4885NOS1 2941/4885CASP7 4762/4885 |
“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.