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 | 0.81 |
| ▸ | NOS1 | P29475 | 10/20 | 1.00 |
| ▸ | CASP7 | P55210 | 2/20 | 1.00 |
| ▸ | HSD17B10 | Q99714 | 2/20 | 1.00 |
| ▸ | USP2 | O75604 | 1/20 | 1.00 |
| ▸ | KDM4E | B2RXH2 | 2/20 | 0.97 |
| ▸ | MAPT | P10636 | 2/20 | 0.97 |
| ▸ | BLM | P54132 | 2/20 | 0.97 |
| ▸ | KMT2A | Q03164 | 2/20 | 0.97 |
| ▸ | ALDH1A1 | P00352 | 1/20 | 0.97 |
| ▸ | GLA | P06280 | 1/20 | 0.97 |
| ▸ | HPGD | P15428 | 1/20 | 0.97 |
| ▸ | PMP22 | Q01453 | 1/20 | 0.97 |
| ▸ | NPSR1 | Q6W5P4 | 1/20 | 0.97 |
| ▸ | TDP1 | Q9NUW8 | 1/20 | 0.97 |
| ▸ | NOS3 | P29474 | 3/20 | 0.62 |
| ▸ | NOS2 | P35228 | 3/20 | 0.62 |
| ▸ | MEN1 | O00255 | 1/20 | 0.41 |
| ▸ | RGS12 | O14924 | 1/20 | 0.41 |
| ▸ | LMNA | P02545 | 1/20 | 0.41 |
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 SCHEMBL21068033 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL29538547 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL29351671 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL7942378 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL29059706 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL14072498 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL260185 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL14401407 | 1.00 | NOS1 (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL31106620 | 0.99 | KDM4E (1.00) | NOS1CASP7HSD17B10USP2KDM4E | |
| Sapropterin SCHEMBL1070424 | 0.99 | KDM4E (1.00) | 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 34 patents — showing the first 20. claimed = in the patent's claims; disclosed = body only.
| Patent | Title | Assignee | Published | Priority | Filing | Country | Status |
|---|---|---|---|---|---|---|---|
| US-20250255873-A1 | RESTORING FUNCTION OF SPECIFIC MUTATIONS AFFECTING THE ACTIN DYNAMIC BY ADMINISTRATION OF SAPROPTERIN | QRGENETICS LTD. (IL) | 2025-08-14 | — | — | US | disclosed |
| US-11878989-B2 | Method for preparing L-erythrobiopterin compound | SHANGHAI FOREFRONT PHARMA CO., LTD. (CN) | 2024-01-23 | — | — | US | disclosed |
| WO-2023148662-A1 | SAPROPTERIN FORMULATION | APR APPLIED PHARMA RESEARCH S.A. (CH) | 2023-08-10 | — | — | WO | disclosed |
| US-20230241069-A1 | Sapropterin Formulation | APR APPLIED PHARMA RESEARCH SA (CH) | 2023-08-03 | — | — | US | disclosed |
| US-20220298178-A1 | METHOD FOR PREPARING L-ERYTHROBIOPTERIN COMPOUND | SHANGHAI FOREFRONT PHARMA CO., LTD. (CN) | 2022-09-22 | — | — | US | disclosed |
| EP-4029519-A1 | TETRAHYDROBIOPTERIN FOR TREATING CONDITIONS ASSOCIATED WITH ELEVATED PHENYLALANINE LEVELS | BioMarin Pharmaceutical Inc. (US) | 2022-07-20 | — | — | EP | disclosed |
| EP-4019523-A1 | METHOD FOR PREPARING L-ERYTHROBIOPTERIN COMPOUND | Shanghai Forefront Pharma Co., Ltd. (CN) | 2022-06-29 | — | — | EP | disclosed |
| CN-112390800-B | Preparation method of L-erythro biopterin compound | 江苏众强药业有限公司 | 2022-03-11 | — | — | CN | disclosed |
| CN-114075234-A | Intermediate for preparing L-erythro biopterin compound and preparation method thereof | 江苏众强药业有限公司 | 2022-02-22 | — | — | CN | disclosed |
| CN-112390800-A | Preparation method of L-erythro biopterin compound | 江苏众强药业有限公司 | 2021-02-23 | — | — | CN | disclosed |
| US-7582799-B2 | Method for producing hydrazone derivatives | SHIRATORI PHARMACEUTICAL CO., LTD. (JP) | 2009-09-01 | — | — | US | disclosed |
| WO-2009088530-A1 | PTERIN ANALOG FOR TREATING BH4 RESPONSIVE CONDITION | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-07-16 | — | — | WO | disclosed |
| US-20090176790-A1 | For treating conditions responsive to tetrahydrobiopterin therapies | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-07-09 | — | — | US | disclosed |
| US-20090176790-A1 | For treating conditions responsive to tetrahydrobiopterin therapies | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-07-09 | — | — | US | disclosed |
| US-20090076305-A1 | METHOD FOR PRODUCING HYDRAZONE DERIVATIVES | SHIRATORI PHARMACEUTICAL CO., LTD. (JP) | 2009-03-19 | — | — | US | disclosed |
| US-20090076305-A1 | METHOD FOR PRODUCING HYDRAZONE DERIVATIVES | SHIRATORI PHARMACEUTICAL CO., LTD. (JP) | 2009-03-19 | — | — | US | disclosed |
| US-20090030191-A1 | Process for Producing Carbon-Reduced Aldose Compounds | ASUBIO PHARMA CO., LTD. (JP) | 2009-01-29 | — | — | US | disclosed |
| WO-2008089008-A2 | TETRAHYDROBIOPTERIN PRODRUGS | BIOMARIN PHARMACEUTICAL INC. (US) | 2008-07-24 | — | — | WO | disclosed |
| EP-1849793-A1 | PROCESS FOR PRODUCING CARBON-DIMINISHED ALDOSE COMPOUND | Asubio Pharma Co., Ltd. (JP) | 2007-10-31 | — | — | EP | disclosed |
| US-20070244322-A1 | Process for Preparing Tetrahydrobiopterin and Analogs of Tetrahydrobiopterin | MERCK EPROVA AG (CH) | 2007-10-18 | — | — | 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 (8 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-11878989-B2 | Method for preparing L-erythrobiopterin compound | SPR, RNPEP, DHPS | PAH 21/4885NOS1 1621/4885CASP7 2052/4885 |
| US-20090030191-A1 | Process for Producing Carbon-Reduced Aldose Compounds | ALDOA, DERA, UGT1A6 | PAH 40/4885NOS1 3214/4885CASP7 4123/4885 |
| US-20090176790-A1 | For treating conditions responsive to tetrahydrobiopterin therapies | TPMT, LTB4R2, BDKRB2 | PAH 40/4885NOS1 942/4885CASP7 3604/4885 |
| 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-20230241069-A1 | Sapropterin Formulation | PSAP, CYP46A1, HSD11B2 | PAH 2390/4885NOS1 2597/4885CASP7 1237/4885 |
| US-20090076305-A1 | METHOD FOR PRODUCING HYDRAZONE DERIVATIVES | CYP3A5, DHPS, ALDH5A1 | PAH 40/4885NOS1 798/4885CASP7 1090/4885 |
| US-20070244322-A1 | Process for Preparing Tetrahydrobiopterin and Analogs of Tetrahydrobiopterin | SPR, DHPS, BLVRB | PAH 43/4885NOS1 1075/4885CASP7 3017/4885 |
| US-20220298178-A1 | METHOD FOR PREPARING L-ERYTHROBIOPTERIN COMPOUND | SPR, RNPEP, DHPS | PAH 21/4885NOS1 1621/4885CASP7 2052/4885 |
“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.