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 | 0.62 |
| ▸ | NOS1 | P29475 | 6/20 | 0.65 |
| ▸ | CASP7 | P55210 | 2/20 | 0.65 |
| ▸ | HSD17B10 | Q99714 | 2/20 | 0.65 |
| ▸ | USP2 | O75604 | 1/20 | 0.65 |
| ▸ | KDM4E | B2RXH2 | 2/20 | 0.64 |
| ▸ | MAPT | P10636 | 2/20 | 0.64 |
| ▸ | BLM | P54132 | 2/20 | 0.64 |
| ▸ | KMT2A | Q03164 | 2/20 | 0.64 |
| ▸ | ALDH1A1 | P00352 | 1/20 | 0.64 |
| ▸ | GLA | P06280 | 1/20 | 0.64 |
| ▸ | HPGD | P15428 | 1/20 | 0.64 |
| ▸ | PMP22 | Q01453 | 1/20 | 0.64 |
| ▸ | NPSR1 | Q6W5P4 | 1/20 | 0.64 |
| ▸ | TDP1 | Q9NUW8 | 1/20 | 0.64 |
| ▸ | NOS3 | P29474 | 2/20 | 0.52 |
| ▸ | NOS2 | P35228 | 2/20 | 0.52 |
| ▸ | MEN1 | O00255 | 1/20 | 0.31 |
| ▸ | RGS12 | O14924 | 1/20 | 0.31 |
| ▸ | LMNA | P02545 | 1/20 | 0.31 |
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 SCHEMBL31673726 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL25915067 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL20769839 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL25915163 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL28826011 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL1534380 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL37480 | 1.00 | NOS1 (0.65) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL1650464 | 0.99 | NOS1 (0.64) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL21046203 | 0.99 | KDM4E (0.66) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL1070420 | 0.99 | 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 1357 patents — showing the first 20. claimed = in the patent's claims; disclosed = body only.
| Patent | Title | Assignee | Published | Priority | Filing | Country | Status |
|---|---|---|---|---|---|---|---|
| US-20260137729-A1 | RECOMBINANT BACTERIA EXPRESSING PHENYLALANINE AMMONIA LYASE, PHENYLALANINE TRANSPORTER AND L-AMINO ACID DEAMINASE FOR REDUCING HYPERPHENYLALANINEMIA | SYNLOGIC OPERATING COMPANY, INC. (US) | 2026-05-21 | — | — | US | claimed |
| US-12605386-B2 | Sapropterin formulation | APR APPLIED PHARMA RESEARCH SA (CH) | 2026-04-21 | — | — | US | claimed |
| US-20250339439-A1 | Sapropterin Formulation | APR APPLIED PHARMA RESEARCH SA (CH) | 2025-11-06 | — | — | US | claimed |
| US-20250255873-A1 | RESTORING FUNCTION OF SPECIFIC MUTATIONS AFFECTING THE ACTIN DYNAMIC BY ADMINISTRATION OF SAPROPTERIN | QRGENETICS LTD. (IL) | 2025-08-14 | — | — | US | claimed |
| EP-4034122-B1 | METHODS FOR TREATING HYPERPHENYLALANINEMIA | PTC THERAPEUTICS MP INC (US) | 2025-08-13 | — | — | EP | claimed |
| WO-2025111482-A1 | COMPLEXING AGENT SALT FORMULATIONS OF PHARMACEUTICAL COMPOUNDS AT LOW STOICHIOMETRIC RATIOS | BEXSON BIOMEDICAL, INC. (US) | 2025-05-30 | — | — | WO | claimed |
| CN-119925601-A | Application of tetrahydrobiopterin pathway as target in anti-QDPR deletion type tumor | 中山大学肿瘤防治中心（中山大学附属肿瘤医院、中山大学肿瘤研究所） | 2025-05-06 | — | — | CN | claimed |
| EP-3923988-B1 | TRIAMTERENE OR NOLATREXED FOR USE IN THE TREATMENT OF PHENYLKETONURIA | SOM INNOVATION BIOTECH S A (ES) | 2025-04-30 | — | — | EP | claimed |
| US-20240398819-A1 | UTILITY OF VITAMIN D, FOLIC ACID AND NADH TO IMPROVE LIGAMENT INJURY PREVENTION CHARACTERISTICS | RHODE ISLAND HOSPITAL | 2024-12-05 | — | — | US | claimed |
| US-20240390364-A1 | Novel Pyridine Compositions and their use in methods for preventing or treating diseases, disorders and conditions | REID CHRISTOPHER BRIAN (US) | 2024-11-28 | — | — | US | claimed |
| EP-1845952-A2 | STABLE TABLET FORMULATION OF TETRAHYDROBIOPTERIN | Biomarin Pharmaceutical Inc. (US) | 2007-10-24 | — | — | EP | claimed |
| WO-2007061529-A1 | LYOPHILIZATION PROCESS AND PRODUCTS OBTAINED THEREBY | SCIDOSE LLC. (US) | 2007-05-31 | — | — | WO | claimed |
| US-20070116729-A1 | Lyophilization process and products obtained thereby | SCIDOSE LLC | 2007-05-24 | — | — | US | claimed |
| US-20060211701-A1 | Use of von tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders | BIOMARIN PHARMACEUTICAL INC. | 2006-09-21 | — | — | US | claimed |
| WO-2006055511-A2 | STABLE TABLET FORMULATION OF TETRAHYDROBIOPTERIN | BIOMARIN PHARMACEUTICAL INC. (US) | 2006-05-26 | — | — | WO | claimed |
| US-20060024365-A1 | Novel dosage form | VAYA NAVIN | 2006-02-02 | — | — | US | claimed |
| US-20060018933-A1 | Novel drug delivery system | TORRENT PHARMACEUTICALS LIMITED (IN) | 2006-01-26 | — | — | US | claimed |
| US-20060018934-A1 | Novel drug delivery system | TORRENT PHARMACEUTICALS LIMITED (IN) | 2006-01-26 | — | — | US | claimed |
| EP-1575593-A2 | USE OF VON TETRAHYDROBIOPTERINE DERIVATIVES IN THE TREATMENT AND NUTRITION OF PATIENTS WITH AMINO ACID METABOLIC DISORDERS | Orphanetics Pharma Entwicklungs GmbH (AT) | 2005-09-21 | — | — | EP | claimed |
| WO-2004058268-A2 | USE OF VON TETRAHYDROBIOPTERINE DERIVATIVES IN THE TREATMENT AND NUTRITION OF PATIENTS WITH AMINO ACID METABOLIC DISORDERS | ORPHANETICS PHARMA ENTWICKLUNGS GMBH (AT) | 2004-07-15 | — | — | WO | 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 (5 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-20060211701-A1 | Use of von tetrahydrobiopterine derivatives in the treatment and nutrition of patients with amino acid metabolic disorders | PAH, BCAT1, AADAT | PAH 1/4885NOS1 671/4885CASP7 4768/4885 |
| US-20250339439-A1 | Sapropterin Formulation | PSAP, CYP46A1, HSD11B2 | PAH 2390/4885NOS1 2597/4885CASP7 1237/4885 |
| US-20250255873-A1 | RESTORING FUNCTION OF SPECIFIC MUTATIONS AFFECTING THE ACTIN DYNAMIC BY ADMINISTRATION OF SAPROPTERIN | ACTR2, ACTB, ACTR3 | PAH 1827/4885NOS1 2695/4885CASP7 4606/4885 |
| US-20260137729-A1 | RECOMBINANT BACTERIA EXPRESSING PHENYLALANINE AMMONIA LYASE, PHENYLALANINE TRANSPORTER AND L-AMINO ACID DEAMINASE FOR REDUCING HYPERPHENYLALANINEMIA | ALAD, SLC1A5, ADA | PAH 4/4885NOS1 567/4885CASP7 3374/4885 |
| US-12605386-B2 | Sapropterin formulation | PAH, CYP46A1, CYP21A2 | PAH 1/4885NOS1 1774/4885CASP7 641/4885 |
“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.