DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for congenital factor VII deficiency — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCongenital factor VII deficiency maps to a 1-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 congenital factor vii deficiency 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
coagulation factor VII (F7) — F7 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 2rdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 5PAG · 1.36 Å · ligand (2R)-2-hydroxy-N-[[3-[5-hydroxy-4-(1H-pyrrolo[3,2-c]pyridin-2-yl)pyrazol-1-yl]phenyl]methyl]-3-methylbutanamide (7YJ). Experimental structure, not a prediction.
What the evidence adds up to
In two unrelated families studied in 1961, 4 of 68 individuals had severe factor VII deficiency (less than 0.1% factor VII) and 29 appeared to be heterozygotes with 30–60% factor VII. The inheritance pattern was an autosomal gene of intermediate expression and complete penetrance, with the haemorrhagic diathesis clearly recessive in the homozygous state. A 2016 report described a homozygous splice-site mutation (c.572-1G>A) in the FVII gene of a proband; both parents were carriers, and the method was used for prenatal diagnosis via amniocentesis.
Two patients with moderate congenital factor VII deficiency underwent surgery in a 1983 report. Neither had any history of abnormal bleeding. Coagulation screening showed a prolonged prothrombin time with a normal activated partial thromboplastin time, and specific factor VII assays confirmed moderate deficiency in both patients and some family members. Preoperative replacement therapy was given to only one patient. In both cases there was no excessive blood loss during or after surgery. The authors concluded that surgery can be performed without routine preoperative replacement therapy in such patients.
No abstract reports any therapeutic trial, any drug, or any attempt to raise factor VII levels with an agent. The 1961 and 2016 studies are purely genetic; the 1983 report is a surgical management observation. There is no evidence from these abstracts about any drug that could treat or prevent bleeding in congenital factor VII deficiency.
What remains missing is any randomised trial of a drug for this condition, any data on non-replacement therapies, and any systematic attempt to stratify patients by bleeding phenotype or genotype to predict who might need prophylaxis. The genetic basis is clear, but the clinical management data are limited to small surgical case series.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Thrombosis and Haemostasis · 1961 · 25 citations
Two Typical Hereditary Charts of Congenital Factor VII Deficiency
AbstractSummary The inheritance of congenital factor VII deficiency was investigated in 2 unrelated families. Out of 68 individuals, 4 (3 proven and 1 highly probable) were found to have severe factor VII deficiency (<C 0.1% factor VII), and 29 appeared to be heterozygotes (30—60% factor VII). The coagulation defect is due to an autosomal gene of intermediate expression and complete penetrance. The recessive character of the haemorrhagic diathesis due to the homozygous state for the abnormal gene is clearly demonstrated.
[Mutation analysis and prenatal diagnosis for a family affected with congenital factor VII deficiency].
AbstractOBJECTIVE: To provide mutation analysis and prenatal diagnosis for a family affected with congenital factor VII(FVII) deficiency. METHODS: DNA was extracted from peripheral blood samples from the proband and his parents. All exons and flanking sequence of the FVII gene were amplified with PCR and subjected to direct sequencing. Prenatal diagnosis was performed by amniocentesis. RESULTS: A homozygous mutation (NM_000131.3) c.572-1G>A was identified in the proband. Both parents of the fetus were carriers of the mutation. CONCLUSION: A method for molecular diagnosis of congenital factor VII deficiency was established and successfully applied for an affected family.
Surgery in Congenital Factor Vii Deficiency: Report of Two Cases
AbstractSummaryTwo cases of moderate congenital factor VII deficiency arc reported. Both patients were admitted for surgery. They had never experienced any abnormal bleeding tendency. Coagulation screening showed a prolonged prothrombin time and a normal activated partial thromboplastin time. Specific assay of factor VII showed a moderate deficiency in both patients and in some of their family members.Preoperative replacement therapy was administered only in one case. In both cases there was no excessive blood loss during and after surgery.In congenital factor VII deficiency, surgery can be performed without any problem and routine preoperative replacement therapy can be avoided.
Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), 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.
