Rare & Orphan Lab · DeCure for X

DeCure for Wet macular degeneration

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for wet macular degeneration — screening already-approved drugs against its 34-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module34 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:10873$DeCureRare

The disease map

Disease moduleWet macular degeneration maps to a 34-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 wet macular degeneration 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

double PHD fingers 3 (DPF3)DPF3 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 unxdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 5I3L · 1.85 Å · ligand UNKNOWN ATOM OR ION (UNX). Experimental structure, not a prediction.

What the evidence adds up to

A 2004 review of treatments for neovascular age-related macular degeneration noted that preliminary results with new pharmacotherapies targeting angiogenesis had been encouraging, and that longer follow-up and subgroup analyses of photodynamic therapy studies had suggested new potential indications. The review stated that a patient with wet macular degeneration at that time might have multiple investigational options that had been unavailable several years earlier. Ongoing clinical trials were aimed at determining the long-term safety and efficacy of these new pharmacologic and combination therapy modalities.

A 2017 review stated that treatment of wet-form neovascular age-related macular degeneration was based on recurrent intravitreal injections of anti-vascular endothelial growth factors, but that this treatment does not cure patients or cause complete resolution of the disease. The review noted a need for other treatment modalities such as stem cell or gene therapies, and that there were ongoing or finished trials that gave hope for treatment.

A 2015 review of genetics in age-related macular degeneration noted that striking advances in treating the neovascular stage of late AMD had occurred, but that no therapy was available for almost half of all AMD patients, specifically those affected by the atrophic form of the disease. The review highlighted the need to explore further the underlying mechanisms for both development and progression of the disease as a starting point for designing innovative therapies.

A 2023 review of dry AMD drug treatment stated that although the first FDA approved drugs had been carried out in 2023, there was still a long way to go in developing effective therapies for dry AMD. A 2023 review of traditional Chinese medicine for wet AMD found that the main source of the etiology, pathogenesis, syndrome differentiation and treatment was the author's subjective clinical experience, with no standardised or unified understanding, making research results different and treatment effect not ideal. What is still missing is a standardised understanding of pathogenesis for both forms of the disease, effective therapies for the atrophic form that affects half of patients, and treatments for wet AMD that achieve complete resolution rather than just management with recurrent injections.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Retina · 2012 · 65 citations

CURRENT KNOWLEDGE AND TRENDS IN AGE-RELATED MACULAR DEGENERATION

AbstractPURPOSE: To address the most dynamic and current issues concerning today's treatment options and promising research efforts regarding treatment for age-related macular degeneration. This review is aimed to serve as a practical reference for more in-depth reviews on the subject. METHODS: An online review of the database PubMed and Ovid were performed, searching for the key words age-related macular degeneration, AMD, VEGF, treatment, PDT, steroids, bevacizumab, ranibizumab, VEGF-trap, radiation, combined therapy, as well as their compound phrases. The search was limited to articles published since 1985. All returned articles were carefully screened, and their references were manually reviewed for additional relevant data. The web page www.clinicaltrials.gov was also accessed in search of relevant research trials. RESULTS: A total of 363 articles were reviewed, including 64 additional articles extracted from the references. At the end, only 160 references were included in this review. CONCLUSION: Treatment for age-related macular degeneration is a very dynamic research field. While current treatments are mainly aimed at blocking vascular endothelial growth factor, future treatments seek to prevent vision loss because of scarring. Promising efforts have been made to address the dry form of the disease, which has lacked effective treatment.

https://doi.org/10.1097/iae.0b013e318271f265
Current Opinion in Ophthalmology · 2004 · 27 citations

A review of treatments for macular degeneration: a synopsis of currently approved treatments and ongoing clinical trials

AbstractPURPOSE OF REVIEW: The purpose of this report is to review recent literature and ongoing clinical trials of the treatment of neovascular age-related macular degeneration. RECENT FINDINGS: Advances in the understanding of the pathogenesis of age-related macular degeneration and choroidal neovascularization have resulted in the emergence of pharmacotherapies targeting various aspects of angiogenesis. Preliminary results with the new agents have been encouraging. Moreover, longer follow-up and subgroup analyses of the Treatment of Age-Related Macular Degeneration with Photodynamic Therapy and Verteporfin in Photodynamic Therapy studies have suggested new potential indications for photodynamic therapy. SUMMARY: Currently a patient with wet macular degeneration may have multiple investigational options that were unavailable several years ago. Ongoing clinical trials are aimed at determining the long-term safety and efficacy of these new pharmacologic and combination therapy modalities.

https://doi.org/10.1097/01.icu.0000122122.24016.f1
Annual Review of Vision Science · 2015 · 21 citations

What Does Genetics Tell Us About Age-Related Macular Degeneration?

AbstractAge-related macular degeneration (AMD) is a chronic degenerative disease of the central retina and a major cause of vision impairment and blindness with millions of people affected in the elderly population. In recent years, considerable efforts have been made to understand disease pathology with the long-term goal of designing novel and effective treatment options for this devastating disease. Although striking advances in treating the neovascular stage of late AMD have occurred, no therapy is available for almost half of all AMD patients, specifically those who are affected by the atrophic form of the disease. This review highlights current knowledge on the genetic factors associated with early- and late-stage forms of the disease. It also summarizes the findings regarding the extent to which these factors may play a role in the transition from one disease stage to another, and it emphasizes the need to explore further the underlying mechanisms for both development and progression of this disease as a starting point for designing innovative therapies for it.

https://doi.org/10.1146/annurev-vision-082114-035609
Seminars in Ophthalmology · 2023 · 3 citations

Controversies and Disparities in the Management of Age-Related Macular Degeneration

Abstractcentury, there were no effective treatments for this devastating disease. However, the last 20 years have heralded the development of treatments for both the nonexudative and exudative forms. The invention of AREDS vitamin supplements and anti-VEGF therapies forever changed the treatment of dry and wet age-related macular degeneration, respectively. The rapid adoption and expansion of these vision preserving treatments has created controversy regarding their cost, burden of administration, development, and use of new technologies, genetic considerations, and observed societal disparities. Many of these controversies and disparities persist today and will require further research to resolve.

https://doi.org/10.1080/08820538.2022.2152705
Expert Review of Ophthalmology · 2023 · 2 citations

What is on the horizon for dry age-related macular degeneration drug treatment?

AbstractIntroduction Age-related Macular Degeneration (AMD) is one of the leading causes of irreversible visual acuity reduction among the elderly population. Dry AMD, the most frequent AMD form, is characterized by photoreceptor loss, Retinal Pigmented Epithelium (RPE) dysfunction and retinal degeneration. Although in the last years several clinical trials have been carried out and many therapeutic molecules have been examined to treat dry AMD, currently its treatment remains unsatisfactory.Areas covered In this review we report and analyze the most important molecules and treatment that have been studied and carried out till nowadays, and the future therapies that may be a hope in this complex and serious disease. We have provided a narrative review after having analyzed the literature and we have selected more than 120 papers to deliver this article.Expert opinion Although in 2023 has been carried out the first FDA approved drugs, there is still a long way to go in developing effective therapies for dry-AMD. In the future, a better understanding of its pathogenesis may lead to the development of targeted or genetic therapies that not only have an anatomical effect on the retina but also have a functional impact.

https://doi.org/10.1080/17469899.2023.2267179
Güncel Retina Dergisi (Current Retina Journal) · 2017 · 0 citations · open access

Stem Cell and Gene Therapy For Wet-Form (Neovascular) Age-Related Macular Degeneration

AbstractTreatment of wet-form (neovascular) Age-Related Macular Degeneration is nowadays based on recurrent intravitreal injections of anti-vascular endothelial growth factors. But this treatment does not cure the patients or does not cause complete resolution of the disease. So there is a need for other treatment modalities such as stem cell or gene therapies. Nowadays there are ongoing or finished trials that give hope for the treatment of this disease.

https://doi.org/10.37783/crj-0039
Frontiers in Medical Science Research · 2023 · 0 citations · open access

Research Advances of the Method of Replenishing Deficiency and Removing Dirty Stasis in the Treatment of Wet Age-Related Macular Degeneration

AbstractWet age-related macular degeneration is a third-leading blinding eye disease that occurs mostly in people over 50 years of age, with loss of central vision as the primary symptom, and involves the macular area of the retina. By collating and analyzing literatures related to the treatment of wet age-related macular degeneration with traditional Chinese medicine in recent years, it was found that the main source of the etiology, pathogenesis, syndrome differentiation and treatment of the disease is the author's subjective clinical experience, there is no standardized and unified understanding, which makes the research results different and the treatment effect is not ideal. The article will analyze and summarize the etiology, pathogenesis, syndrome differentiation and traditional Chinese medicine treatment of wet age-related macular degeneration, which can provide drug guidance for clinical treatment and improve the therapeutic effect.

https://doi.org/10.25236/fmsr.2023.051004
Docs.school Publications · 2007 · 0 citations

Personal and Societal Alchemy in Early Daoist Scriptures

AbstractCurrent management of age-related macular degeneration (AMD) can be divided into two categories: first, anti-vasoendothelial growth factor (anti-VEGF) injection for wet macular degeneration; second, anti-oxidant vitamins for dry macular degeneration. New therapies are being developed for both of these diseases using novel technologies and different modes of administration. The hope is that some of these therapies will achieve significant improvement to current management and prevent future loss of vision in this devastating eye condition.

https://doi.org/10.12703/p6-29

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.