DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hereditary angioedema type 3 — screening already-approved drugs against its 4-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHereditary angioedema type 3 maps to a 4-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 hereditary angioedema type 3 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 XII (F12) — F12 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 2sdrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6X0T · 1.388 Å · ligand (2S)-1-(N,3-dicyclohexyl-D-alanyl)-4-[(4R,5S)-4-methyl-5-phenyl-4,5-dihydro-1,3-oxazol-2-yl]-N-[(thiophen-2-yl)methyl]piperazine-2-carboxamide (UJ7). Experimental structure, not a prediction.
What the evidence adds up to
Hereditary angioedema type 3 is not mentioned in any of the three abstracts. The 2015 review and the 2021 family case analysis both describe hereditary angioedema as a defect in C1 esterase inhibitor, which is the basis of types 1 and 2, not type 3. The 2021 article states that therapy is determined by the need to relieve acute attacks, prevent oedema before medical interventions, and, when indicated, provide long-term prophylaxis. It reports high efficacy of pathogenetic therapy with human C1-esterase inhibitor in the family case described, but gives no response rates or sample sizes. The 2015 review notes that management is still evolving and that drug therapy is very costly and not available worldwide.
The 2023 case report describes a 34-year-old man diagnosed with hereditary angioedema at age 10 who suffered several attacks per month. He received prophylactic antifibrinolytics and androgens without improvement, and used plasma-derived C1-INH concentrate or icatibant for on-demand treatment. After being diagnosed with multiple sclerosis at age 33, he started teriflunomide 14 mg/day. Angioedema attacks disappeared 40 days after starting treatment. The authors suggest that teriflunomide could be considered as a prophylactic option but state that its effectiveness on this condition should be further studied. This is a single case report, not a controlled trial.
No abstract provides survival data, response rates in a defined cohort, or any randomised evidence for any drug in hereditary angioedema type 3 specifically. What is missing is any trial that enrols patients with confirmed type 3 disease, any funding for such a trial, and any validated stratification that distinguishes type 3 from the C1-inhibitor-deficient forms in treatment studies.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Lebanese Medical Journal · 2015 · 1 citations
Hereditary Angioedema : A Literature Review and National Management Guidelines
AbstractBACKGROUND: Hereditary angioedema, a rare and potentially life-threatening condition, is the result of a defect in the C1 esterase inhibitor. Primary care physicians should be familiar with this condition to avoid complications and improve quality of care. METHODS: We present two cases of hereditary angioedema followed by a discussion based on a literature review of the recent guidelines and advances in this condition. OBJECTIVES: To highlight the clinical aspects, diagnosis and treatment of this condition and propose a practical local management based on the available medication. CONCLUSION: Hereditary angioedema management is still evolving. More efforts should be made concerning the drug therapy which is very costly and not available worldwide.
Meditsinskiy sovet = Medical Council · 2021 · 0 citations · open access
Hereditary angioedema: approaches to diagnosis and treatment, analysis of a clinical family case
AbstractHereditary angioedema belongs to the group of rare, orphan, genetically determined defects that represent a significant medical and social problem due to the pronounced impact on the quality of life and potential mortality, as well as the emerging difficulties associated with timely diagnosis and the appointment of adequate treatment. The article presents data on the modern classification of hereditary angioedema, clinical manifestations of the disease, approaches to diagnosis verification and treatment principles. Therapy of hereditary angioedema is determined by the need for effective relief of acute attacks of the disease, prevention of edema before medical interventions, and, if indicated, long-term prophylaxis. The article discusses a differentiated approach to the treatment of hereditary angioedema, characterizes various options for therapeutic interventions. In a clinical case, the history of several generations of a family with manifestations of hereditary angioedema is described. A modern approach to the diagnosis of the disease based on detailed history, clinical symptoms, and laboratory research results has been demonstrated. The analysis of the effectiveness of treatment was carried out and the high efficiency of pathogenetic therapy of hereditary angioedema with human C1-esterase inhibitor was shown.
European Journal of Case Reports in Internal Medicine · 2023 · 0 citations · open access
Remission of hereditary angioedema attacks associated with starting teriflunomide in a patient with multiple sclerosis
AbstractBackground: Hereditary angioedema is a rare hereditary and potentially life-threatening disorder characterized by recurrent attacks of cutaneous and submucosal swelling. In spite of the advances made in terms of pathophysiology, underlying mechanisms are not fully clear and this, in turn, hinders the development of effective therapies. Currently, on demand treatment is considered first-class, with few cost-effective, long-term prophylactic options. Case presentation: Here we describe the case of a 34-year-old man diagnosed with hereditary angioedema at the age of 10, who used to suffer several angioedema attacks per month. He was given prophylactic treatment with antifibrinolytic agents and androgens without improvement. Moreover, he was treated with plasma-derived C1-INH concentrate or icatibant for on-demand treatment of moderate and severe angioedema attacks. At the age of 33, after suffering sudden vision loss and lower limb paresthesia, he was studied and diagnosed with multiple sclerosis. Teriflunomide was administered at a dosage of 14 mg/day. Angioedema attacks disappeared 40 days after starting treatment. Conclusion: Thus, we suggest considering the pathophysiologic mechanisms on which teriflunomide could be active and consider this drug carefully as an option for prophylaxis purposes. Yet, its effectiveness on this condition should be further studied. LEARNING POINTS: Underlying mechanisms in hereditary angioedema lack clarity and hence hinder the development of effective therapies.On-demand treatment of hereditary angioedema is considered first class, with few cost-effective, long-term prophylactic options.The mechanisms of action and effectiveness of teriflunomide on hereditary angioedema should be studied further.
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.
