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 |
| ▸ | KDM4E | B2RXH2 | 2/20 | 0.66 |
| ▸ | MAPT | P10636 | 2/20 | 0.66 |
| ▸ | BLM | P54132 | 2/20 | 0.66 |
| ▸ | KMT2A | Q03164 | 2/20 | 0.66 |
| ▸ | ALDH1A1 | P00352 | 1/20 | 0.66 |
| ▸ | GLA | P06280 | 1/20 | 0.66 |
| ▸ | HPGD | P15428 | 1/20 | 0.66 |
| ▸ | PMP22 | Q01453 | 1/20 | 0.66 |
| ▸ | NPSR1 | Q6W5P4 | 1/20 | 0.66 |
| ▸ | TDP1 | Q9NUW8 | 1/20 | 0.66 |
| ▸ | NOS1 | P29475 | 6/20 | 0.64 |
| ▸ | CASP7 | P55210 | 2/20 | 0.64 |
| ▸ | HSD17B10 | Q99714 | 2/20 | 0.64 |
| ▸ | USP2 | O75604 | 1/20 | 0.64 |
| ▸ | 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 SCHEMBL423477 | 1.00 | KDM4E (0.66) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL21046203 | 1.00 | KDM4E (0.66) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL1070420 | 1.00 | KDM4E (0.66) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL31673726 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL20769839 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL37480 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL1534380 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL28826011 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL258544 | 0.99 | NOS1 (0.65) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL25915067 | 0.99 | NOS1 (0.65) | 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 597 patents — showing the first 20. claimed = in the patent's claims; disclosed = body only.
| Patent | Title | Assignee | Published | Priority | Filing | Country | Status |
|---|---|---|---|---|---|---|---|
| US-20240282425-A1 | AUTHENTICATION METHODS AND SYSTEMS FOR DISPENSED PRESCRIPTIONS | LOW GORDON KEITH (US) | 2024-08-22 | — | — | US | claimed |
| US-11331319-B2 | Combination treatment for neuropsychiatric disorders | UNIVERSITY OF MASSACHUSETTS (US) | 2022-05-17 | — | — | US | claimed |
| WO-2021061065-A1 | SOLID ORAL PHARMACEUTICAL FORMULATIONS COMPRISING SAPROPTERIN DIHYDROCHLORIDE AND AT LEAST ONE PHARMACEUTICAL EXCIPIENT | SANOVEL ILAC SANAYI VE TICARET ANONIM SIRKETI (TR) | 2021-04-01 | — | — | WO | claimed |
| WO-2021061067-A1 | PHARMACEUTICAL FORMULATIONS COMPRISING SAPROPTERIN DIHYDROCHLORIDE AND AT LEAST ONE ANTIOXIDANT | SANOVEL ILAC SANAYI VE TICARET ANONIM SIRKETI (TR) | 2021-04-01 | — | — | WO | claimed |
| WO-2021061066-A1 | EFFERVESCENT FORMULATIONS OF SAPROPTERIN DIHYDROCHLORIDE | SANOVEL ILAC SANAYI VE TICARET ANONIM SIRKETI (TR) | 2021-04-01 | — | — | WO | claimed |
| EP-3697400-A1 | METHODS OF USING EHMT2 INHIBITORS IN TREATING OR PREVENTING BLOOD DISORDERS | Epizyme, Inc. (US) | 2020-08-26 | — | — | EP | claimed |
| CN-111315371-A | Methods of treating or preventing hematologic disorders using EHMT2 inhibitors | 埃皮兹姆公司 | 2020-06-19 | — | — | CN | claimed |
| EP-1687308-B1 | Process for preparating (6R)-L-erythrotetrahydrobiopterin hydrochloride crystalline form B from other crystalline forms | Merck & Cie (CH) | 2019-06-12 | — | — | EP | claimed |
| US-20190160070-A1 | DRY BLEND FORMULATION OF TETRAHYDROBIOPTERIN | BIOMARIN PHARM INC (US) | 2019-05-30 | — | — | US | claimed |
| US-20190127801-A1 | SYSTEM AND METHOD FOR DIAGNOSIS AND TREATMENT OF NEUROPSYCHIATRIC DISORDERS | THE RESEARCH FOUNDATION FOR MENTAL HYGIENE, INC. (US) | 2019-05-02 | — | — | US | claimed |
| US-20090198055-A1 | METHOD OF SYNTHESIZING TETRAHYDROBIOPTERIN | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-08-06 | — | — | US | claimed |
| US-7566462-B2 | Stable tablet formulation | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-07-28 | — | — | US | claimed |
| WO-2008128049-A2 | METHODS OF ADMINISTERING TETRAHYDROBIOPTERIN, ASSOCIATED COMPOSITIONS, AND METHODS OF MEASURING | BIOMARIN PHARMACEUTICAL INC. (US) | 2008-10-23 | — | — | WO | claimed |
| US-20080207626-A1 | Storage stability; mixture of tetrahydrobiopterin and excipient | BIOMARIN PHARMACEUTICAL INC. (US) | 2008-08-28 | — | — | US | claimed |
| US-20080175924-A1 | System and Method For Diagnosis of Neuropsychiatric Disorders | THE RESEARCH FOUNDATION FOR MENTAL HYGIENE, INC. (US) | 2008-07-24 | — | — | US | claimed |
| WO-2008070296-A2 | SYSTEM AND METHOD FOR DIAGNOSIS AND TREATMENT OF NEUROPSYCHIATRIC DISORDERS | THE RESEARCH FOUNDATION FOR MENTAL HYGIENE, INC. (US) | 2008-06-12 | — | — | WO | claimed |
| CN-1894251-A | Crystalline forms of (6R)-l-erythro-tetrahydrobiopterin dihydrochloride | MERCK EPROVA AG (CH) | 2007-01-10 | — | — | CN | claimed |
| EP-1687308-A2 | CRYSTALLINE FORMS OF (6R) -L-ERYTHRO-TETRAHYDROBIOPTERIN DIHYDROCHLORIDE | Merck Eprova AG (CH) | 2006-08-09 | — | — | EP | claimed |
| US-20060035900-A1 | Crystalline forms of ( 6R)-L-erythro-tetrahydrobiopterin dihydrochloride | MERCK & CIE KG (CH) | 2006-02-16 | — | — | US | claimed |
| WO-2005065018-A2 | CRYSTALLINE FORMS OF (6R) -L-ERYTHRO-TETRAHYDROBIOPTERIN DIHYDROCHLORIDE | MERCK EPROVA AG (US) | 2005-07-21 | — | — | 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 (4 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-20190160070-A1 | DRY BLEND FORMULATION OF TETRAHYDROBIOPTERIN | QDPR, HTR1B, HTR3B | PAH 87/4885KDM4E 4599/4885MAPT 280/4885 |
| US-20060035900-A1 | Crystalline forms of ( 6R)-L-erythro-tetrahydrobiopterin dihydrochloride | BLVRB, QDPR, SPR | PAH 55/4885KDM4E 4288/4885MAPT 1089/4885 |
| US-20080207626-A1 | Storage stability; mixture of tetrahydrobiopterin and excipient | QDPR, BHMT2, BHMT | PAH 30/4885KDM4E 3957/4885MAPT 127/4885 |
| US-20090198055-A1 | METHOD OF SYNTHESIZING TETRAHYDROBIOPTERIN | SPR, THOP1, PTMA | PAH 37/4885KDM4E 4788/4885MAPT 4033/4885 |
“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.