DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for complement deficiency — screening already-approved drugs against its 29-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleComplement deficiency maps to a 29-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 complement 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
complement C1q A chain (C1QA) — C1QA 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 2drdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 2WNV · 1.25 Å · ligand 2-deoxy-beta-D-erythro-pentofuranose (2DR). Experimental structure, not a prediction.
What the evidence adds up to
A 1960 case report describes a single individual with a striking deficiency in serum complement activity lasting at least seven years, attributed to a depression of the second complement component, with no secondary disease process identified. A 1987 report on familial properdin deficiency notes that inherited complement deficiencies are being recognised more frequently and that approximately half of affected persons are healthy, though typical clinical features in the remainder should suggest the diagnosis. A 1993 review titled "Complement Deficiency and Disease" was published in Rheumatology. A 2009 case describes a Japanese male with total deficiency of the second complement component and approximately half-normal levels of the ninth component, accompanied by chronic idiopathic neutropenia; despite constantly few neutrophil granulocytes, this patient showed no evidence of susceptibility to viral or bacterial infections, and his HLA type differed from that seen in Caucasian patients. A 2017 review states that studies of inherited complement deficiency states have provided apparent biological facts about the protective importance of an intact complement system, but that some observations challenge further investigation.
No treatment, no drug, and no intervention of any kind is mentioned in any of these abstracts. The 1960 case received no therapy for the deficiency itself. The 1987 properdin family report describes only the natural history of the deficiency. The 2009 neutropenia case is simply a description of a laboratory finding. There is no evidence from these abstracts that any drug has been tested to correct complement deficiency, to prevent infection in deficient patients, or to modify the course of any associated disease.
What is missing is any controlled trial, any drug candidate, any animal model of therapeutic intervention, and any systematic attempt to stratify patients by complement component, infection history, or HLA type. No funding for a treatment trial is described. The field remains at the stage of case reports and narrative reviews.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
New England Journal of Medicine · 1987 · 138 citations
Familial Properdin Deficiency and Fatal Meningococcemia
AbstractINHERITED deficiencies of various complement proteins are being recognized with increasing frequency. Careful evaluation of these deficiency states has augmented our understanding of the biologic function of individual components of the complement system, as well as of the system as a whole. Approximately half the affected persons are healthy. Nevertheless, typical clinical features have been found in the remainder, and should suggest a diagnosis of complement deficiency and identify the particular component or activation pathway that is defective.1 , 2 Inherited deficiencies of the nonregulatory components of the alternative pathway have been recognized only rarely. This report describes a large family in . . .
Brief Note: Essential Hypocomplementemia: Report of a Case
AbstractAbstract A case of essential hypocomplementemia is reported in an individual who for at least 7 years has shown a striking deficiency in serum complement activity, due to a depression in the level of second component of complement. There is no clinical evidence to suggest that the phenomenon has occurred secondary to any recognizable disease process. The significance of complement action and complement deficiency is discussed.
AbstractJournal Article COMPLEMENT DEFICIENCY AND DISEASE Get access MARK J. WALPORT MARK J. WALPORT Rheumotology Unit, Hammersmith HospitalDu Cane Road, London W12 0NN Search for other works by this author on: Oxford Academic PubMed Google Scholar Rheumatology, Volume 32, Issue 4, April 1993, Pages 269–273, https://doi.org/10.1093/rheumatology/32.4.269 Published: 01 April 1993 Article history Accepted: 01 December 1992 Published: 01 April 1993
Inherited Complement Deficiency States and Disease
AbstractStudies of inherited complement deficiency states in disease have provided a set of apparent biological facts relating to the protective importance of an intact complement system. While some of the findings seem to confirm what may have been expected on the basis of in vitro studies, other observations constitute a challenge to further investigation. In this presentation, basic findings with regard to deficiency of circulating complement proteins are briefly summarized, and some difficulties and issues of possible interest pointed out.
International Archives of Allergy and Immunology · 2009 · 0 citations
Chronic Neutropenia Associated with C2 and C9 Deficiency
AbstractA Japanese male with a deficiency of the second and ninth components of complement associated with chronic idiopathic neutropenia is presented. In this case the second component of complement is totally deficient while the ninth component is approximately half that of normal control. Neutrophil granulocytes are constantly few, but this case shows no evidence of susceptibility to either viral or bacterial infections. His HLA type is different from that of Caucasians, suggesting that the genetic abnormality responsible for the complement deficiency of this Japanese case is different from that seen in Caucasian patients.
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.
