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 SCHEMBL21046203 | 1.00 | KDM4E (0.66) | KDM4EMAPTBLMKMT2AALDH1A1 | |
| Sapropterin SCHEMBL258963 | 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 11 patents. claimed = in the patent's claims; disclosed = body only.
| Patent | Title | Assignee | Published | Priority | Filing | Country | Status |
|---|---|---|---|---|---|---|---|
| EP-2224927-B1 | PTERIN ANALOG FOR TREATING BH4 RESPONSIVE CONDITION | BIOMARIN PHARM INC (US) | 2014-08-20 | — | — | EP | disclosed |
| US-8324210-B2 | Pterin analogs | BIOMARIN PHARMACEUTICAL, INC. (US) | 2012-12-04 | — | — | US | disclosed |
| US-20120022072-A1 | PTERIN ANALOGS | BIOMARIN PHARMACEUTICAL INC. (US) | 2012-01-26 | — | — | US | disclosed |
| EP-2224927-A1 | PTERIN ANALOG FOR TREATING BH4 RESPONSIVE CONDITION | BioMarin Pharmaceutical Inc. (US) | 2010-09-08 | — | — | EP | disclosed |
| US-20100093742-A1 | PTERIN ANALOGS | BIOMARIN PHARMACEUTICAL, INC. (US) | 2010-04-15 | — | — | US | disclosed |
| US-20100016328-A1 | PTERIN ANALOGS | BIOMARIN PHARMACEUTICAL, INC. (US) | 2010-01-21 | — | — | US | disclosed |
| EP-2114944-A2 | TETRAHYDROBIOPTERIN PRODRUGS | BioMarin Pharmaceutical Inc. (US) | 2009-11-11 | — | — | EP | disclosed |
| US-7601717-B2 | Pterin analogs | BIOMARIN PHARMACEUTICAL INC. (US) | 2009-10-13 | — | — | 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 |
| WO-2008089008-A2 | TETRAHYDROBIOPTERIN PRODRUGS | BIOMARIN PHARMACEUTICAL INC. (US) | 2008-07-24 | — | — | WO | 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 (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-20100093742-A1 | PTERIN ANALOGS | DHFR, MTHFD2, HMBS | PAH 12/4885KDM4E 4678/4885MAPT 2683/4885 |
| US-20100016328-A1 | PTERIN ANALOGS | DHFR, MTHFD2, HMBS | PAH 12/4885KDM4E 4678/4885MAPT 2683/4885 |
| US-20120022072-A1 | PTERIN ANALOGS | DHFR, MTHFD2, HMBS | PAH 12/4885KDM4E 4678/4885MAPT 2683/4885 |
| US-20090176790-A1 | For treating conditions responsive to tetrahydrobiopterin therapies | TPMT, LTB4R2, BDKRB2 | PAH 40/4885KDM4E 4837/4885MAPT 1388/4885 |
“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.