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 SCHEMBL258544 | 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
Appears in 8195 patents — a generic fragment claimed broadly, so it's down-weighted as IP noise. Top by claim status then date:
| 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 |
| CN-122056386-A | Moisture absorption resistant slow release microcapsule powder and preparation method and application thereof | 大连医诺生物股份有限公司 | 2026-05-19 | — | — | CN | claimed |
| CN-122030612-A | Shear-resistant slow-release microcapsule powder and preparation method and application thereof | 大连医诺生物股份有限公司 | 2026-05-15 | — | — | CN | claimed |
| US-20260130876-A1 | ADMINISTRATION OF BETA-HYDROXYBUTYRATE AND RELATED COMPOUNDS IN HUMANS | AXCESS GLOBAL SCIENCES LLC (US) | 2026-05-14 | — | — | US | claimed |
| US-12605386-B2 | Sapropterin formulation | APR APPLIED PHARMA RESEARCH SA (CH) | 2026-04-21 | — | — | US | claimed |
| US-12599579-B2 | Compositions and compounds containing ketone bodies and/or ketone body precursors and one or more amino acids | AXCESS GLOBAL SCIENCES, LLC (US) | 2026-04-14 | — | — | US | claimed |
| EP-4679997-A1 | PEROVSKITE COMPOSITE COMPRISING PSEUDOHALIDE ADDITIVE, ELECTRONIC DEVICE COMPRISING SAME, AND MANUFACTURING METHOD THEREOF | POSTECH Research and Business Development Foundation (KR) | 2026-01-14 | — | — | EP | claimed |
| US-12509698-B2 | Synthesis and structure of high potency RNA therapeutics | ARCTURUS THERAPEUTICS, INC. (US) | 2025-12-30 | — | — | US | claimed |
| US-12496283-B2 | Administration of R-beta-hydroxybutyrate and related compounds in humans | AXCESS GLOBAL SCIENCES, LLC (US) | 2025-12-16 | — | — | US | claimed |
| US-20250339439-A1 | Sapropterin Formulation | APR APPLIED PHARMA RESEARCH SA (CH) | 2025-11-06 | — | — | US | claimed |
| US-5502050-A | DIHYDROXYPHENYL-SUBSTITITED PYRIDINES OR QUINONES, HYPERTENSIVE AGENTS | CORNELL RESEARCH FOUNDATION, INC. (US) | 1996-03-26 | — | — | US | claimed |
| EP-0674627-A1 | PYRIMIDINE, PYRIDINE, PTERIDINONE AND INDAZOLE DERIVATIVES AS ENZYME INHIBITORS | THE WELLCOME FOUNDATION LIMITED (GB) | 1995-10-04 | — | — | EP | claimed |
| WO-1995014468-A1 | BLOCKING UTILIZATION OF TETRAHYDROBIOPTERIN TO BLOCK INDUCTION OF NITRIC OXIDE SYNTHESIS | CORNELL RESEARCH FOUNDATION INC. (US) | 1995-06-01 | — | — | WO | claimed |
| WO-1994014780-A1 | PYRIMIDINE, PYRIDINE, PTERIDINONE AND INDAZOLE DERIVATIVES AS ENZYME INHIBITORS | THE WELLCOME FOUNDATION LIMITED (GB) | 1994-07-07 | — | — | WO | claimed |
| EP-0178600-B1 | USE OF A COMBINATION OF TETRAHYDROBIOPTERINS AND LYMPHOKINES FOR THE MANUFACTURE OF A MEDICAMENT POSSESSING AN ENHANCED IN VIVO AND IN VITRO LYMPHOKINE ACTIVITY | Biotest Aktiengesellschaft (DE) | 1989-11-29 | — | — | EP | claimed |
| US-4752573-A | Use of tetrahydrobiopterin to enhance the proliferative activity of interleukin-2 on T cells | BIOTEST-SERUM-INSTITUT GMBH (DE) | 1988-06-21 | — | — | US | claimed |
| EP-0138995-A4 | REGULATION OF ENZYMES WHICH UTILIZE TETRAHYDROBIOPTERIN OR TETRAHYDROFOLATE COFACTORS WITH 6, 6-DISUBSTITUTED-TETRAHYDROPTERIDINES. | UNIV SOUTH ALABAMA (US) | 1988-05-10 | — | — | EP | claimed |
| EP-0178600-A1 | Use of a combination of tetrahydrobiopterins and lymphokines for the manufacture of a medicament possessing an enhanced in vivo and in vitro lymphokine activity | Biotest Aktiengesellschaft (DE) | 1986-04-23 | — | — | EP | claimed |
| EP-0138995-A1 | REGULATION OF ENZYMES WHICH UTILIZE TETRAHYDROBIOPTERIN OR TETRAHYDROFOLATE COFACTORS WITH 6, 6-DISUBSTITUTED-TETRAHYDROPTERIDINES. | UNIV SOUTH ALABAMA (US) | 1985-05-02 | — | — | EP | claimed |
| WO-1984004040-A1 | REGULATION OF ENZYMES WHICH UTILIZE TETRAHYDROBIOPTERIN OR TETRAHYDROFOLATE COFACTORS WITH 6, 6-DISUBSTITUTED-TETRAHYDROPTERIDINES | UNIV SOUTH ALABAMA (US) | 1984-10-25 | — | — | WO | claimed |