DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for hemophilia — screening already-approved drugs against its 7-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleHemophilia maps to a 7-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 hemophilia 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
Since the 1960s, prophylactic factor replacement has become the standard of care in high-income countries, shifting the goal from preventing death to preserving joint function and quality of life. A 2014 review of haemophilic children states that prophylaxis has removed the hallmark of crippling disease with lifelong disabilities, and that advances in treatment have produced continuous improvement in quality of life and life expectancy. The same review notes that inhibitors remain the most severe complication of therapy, and that compliance is the key to successful management. No numbers for survival, bleeding rates, or quality-of-life scores are given in that abstract.
Newer approaches include extended half-life factors, non-factor products, and gene therapy. A 2017 review says these have the potential to decrease infusion frequency, promote prophylaxis, and offer alternatives for inhibitor patients, but that none specifically address the challenge of dispersing treatment to the developing world. A 2021 review states that late-phase clinical trials of gene therapy have shown promising results, but that there remain unanswered questions about both efficacy and safety, and that widespread application still requires overcoming future challenges. No specific response rates, factor activity levels, or adverse event counts are reported in either abstract.
A 2019 review notes that the success of prophylactic therapy has established a new standard of care, but that with gene therapy and treatments that mimic sustained factor replacement, the outcome measures used in past trials need to be reassessed. It emphasises the importance of patient-reported outcomes. A 2003 abstract on mutation detection in haemophilia A describes the factor VIII gene structure but provides no clinical data.
What is still missing: large-scale, long-term safety data for gene therapy; validated outcome measures that capture patient priorities; any evidence that these therapies can be delivered in low-resource settings; and trial designs that stratify patients by inhibitor status, baseline factor level, or joint health.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Blood · 2017 · 123 citations · open access
Novel approaches to hemophilia therapy: successes and challenges
AbstractNew therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.
Hemophilia Gene Therapy: Approaching the First Licensed Product
AbstractThe clinical potential of hemophilia gene therapy has now been pursued for the past 30 years, and there is a realistic expectation that this goal will be achieved within the next couple of years with the licensing of a gene therapy product. While recent late phase clinical trials of hemophilia gene therapy have shown promising results, there remain a number of issues that require further attention with regard to both efficacy and safety of this therapeutic approach. In this review, we present information relating to the current status of the field and focus attention on the unanswered questions for hemophilia gene therapy and the future challenges that need to be overcome to enable the widespread application of this treatment paradigm.
Research and Practice in Thrombosis and Haemostasis · 2019 · 57 citations · open access
Hemophilia trials in the twenty‐first century: Defining patient important outcomes
AbstractTreatment for hemophilia has advanced dramatically over the past 5 decades. Success of prophylactic therapy in preventing bleeding and decreasing associated complications has established a new standard of care. However, with the advent of gene therapy and treatments that effectively mimic sustained coagulation factor replacement, outcome measures that worked well for assessing factor replacement therapies in past clinical trials need to be reassessed. In addition, while therapies have advanced, so has the science of outcome assessment, including recognition of the importance of patient important and patient reported outcomes. This manuscript reviews strengths and limitations of outcome measures used in hemophilia from both a provider and patient perspective.
Pediatric Hematology and Oncology · 2014 · 30 citations
Current Management of the Hemophilic Child: A Demanding Interlocutor. Quality of Life and Adequate Cost-Efficacy Analysis
AbstractHemophilias are the most known inherited bleeding disorders. The challenges in the management of hemophilic children are different from those in adults: prophylaxis regimen removed the hallmark of crippling disease with lifelong disabilities; individualized regimens are being implemented in order to overcome venous access problems. Presently, at least in high-income countries, advances in treatment of hemophilia resulted in continuous improvement of the patients' quality of life and life expectancy. Inhibitors remain the most severe complication of hemophilia therapy. The treatment' compliance is the key to achieve a successful management. The patient, his family, the medical and psychological team are the players of a comprehensive care system. The current management of hemophilic children is the example of huge resource investments enabling long-term benefits in particular quality of life as a primary objective of the healthcare process.
Detection of Mutations in Hemophilia A Patients by Chemical Cleavage of Mismatch Method
AbstractHemophilia A is an X-linked disorder that leads to a defect in blood coagulation. This is caused by mutations in the factor VIII gene, which results in its activity being reduced or abolished in the blood-clotting cascade. The factor VIII gene is 186 kb long with 26 exons, varying from 69 bp (exon 5) to 3106 bp (exon 14) (1). The factor VIII mRNA is 9028 bases in length with a 7053 nucleotides long coding region (2).
Research and Practice in Thrombosis and Haemostasis · 2025 · 0 citations · open access
Trenonacog alfa safety, efficacy, and pharmacokinetics in previously treated pediatric hemophilia B
AbstractBackground: Trenonacog alfa is a recombinant factor IX approved for adolescents and adults with hemophilia B. Objectives: The aim of this study was to assess the pharmacokinetics (PK), efficacy as prophylaxis, control of bleeding episodes, and safety of trenonacog alfa in previously treated participants aged <12 years with severe or moderately severe hemophilia B and no current or history of inhibitors. Methods: The study had 3 phases: (1) PK evaluation after a single infusion of 75 ± 5 IU/kg, (2) treatment phase in which participants received trenonacog alfa prophylaxis 35 to 75 IU/kg for 50 exposure days, and (3) a continuation phase in which prophylaxis could be administered for ≥50 additional exposure days. Results: The PK of trenonacog alfa was comparable between adolescents and adults except for higher clearance, shorter mean residence time and elimination half-life, and lower incremental recovery. Prophylaxis resulted in a median annualized bleeding rate of 0.86 (mean = 2.34) for the combined treatment and continuation phases; 33.3% of participants had zero bleeds; and 83.7% of bleeds treated resolved with 1 or 2 infusions. One adverse event was possibly related to trenonacog alfa, a nonserious hypersensitivity reaction leading to early study termination. The efficacy and safety of trenonacog alfa for prophylaxis and bleeding treatment in previously treated pediatric participants were consistent with those reported for adults and adolescents. There appeared to be no clinically important differences between the results for participants aged <6 years and those aged 6 to <12 years. Conclusion: Trenonacog alfa is a suitable option for the management of pediatric persons with hemophilia B.
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.
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