DeCure's autonomous Metabolic AI scientist is researching a drug-repurposing hypothesis for mucopolysaccharidosis — screening already-approved drugs against its 12-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMucopolysaccharidosis maps to a 12-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.
Research record
01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash
Current lead
No approved-drug candidate for mucopolysaccharidosis is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
glucuronidase beta (GUSB) — GUSB is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet 4rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3HN3 · 1.7 Å · ligand (4R)-2-METHYLPENTANE-2,4-DIOL (MRD). Experimental structure, not a prediction.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Genetics in Medicine · 2025 · 3 citations · open access
Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: A phase-3, 2-part study compared with a historical placebo cohort
AbstractPURPOSE: To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II, compared with a historical placebo from a previous idursulfase trial (TKT024). METHODS: The study comprised 2 sequential parts. In part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks. In the open, single-cohort part 2 study, additional participants received idursulfase beta for 52 weeks. Data from the idursulfase beta groups from parts 1 and 2 were pooled for comparisons with the historical placebo group (n = 32). The primary end point was a change in the 6-minute walk test at week 53. RESULTS: Participants in the idursulfase beta groups (n = 24) were male Asians (mean age, 12.0 years). Idursulfase beta was superior to placebo in 6-minute walk test improvement (62.2 vs 7.3 m, P < .0001). Decrease in urine glycosaminoglycan excretion (-71.13% vs 21.39%, P < .0001) and reduction in the liver (-26.67% vs -0.80%, P < .0001) and spleen volumes (-26.46% vs 7.2%, P < .0001) were significant. The safety profile of idursulfase beta was satisfactory. CONCLUSION: Idursulfase beta is a safe and effective treatment option in mucopolysaccharidosis II, addressing crucial somatic ailments presented by patients.
Elosulfase alfa for the treatment of mucopolysacchoridosis IVA
AbstractMucopolysacchariodosis IVA is a lysosomal storage disorder characterized by deficiency of the enzyme N-acetylgalactosamine-6-sulfatase leading to accumulation of glycosaminoglycans. Mucopolysacchariodosis IVA affects many organs, especially the skeletal system. The disease is progressive, leads to serious cardiopulmonary problems and is severely debilitating. Enzyme replacement therapy with elosulfase alfa is the only approved treatment for this rare genetic condition. The results from a Phase III clinical trial demonstrated that elosulfase alfa at dose 2.0 mg/kg weekly given intravenously improved the walking distance in 6 min. The results of the 3-min stair climb test and respiratory function test did not show statistically significant improvement over the placebo. However, the composite end point analysis combining changes from baseline in walking distance in 6 min, 3-min stair climb test and respiratory function showed that at dose 2.0 mg/kg weekly, subjects performed better than the placebo, indicating that the effects of treatment are clinically meaningful. Serious side effects are uncommon and infusion-associated reactions are manageable.
Disease module: DeepOracle (Open Targets). Approved indication: ChEMBL drug_indication (max_phase=4). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works, resolved on OpenAlex.
DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.
