Research Pipeline
Four active therapeutic programs and two humanized mouse models — driving toward the first treatments for DNM1 epilepsy.
Our therapeutic pipeline.
ASO Development
Late StageLate-stage allele-specific antisense oligonucleotide (ASO) strategy — one of the most advanced therapeutic approaches for DNM1. Targeting specific disease-causing variants with high precision.
Drug Repurposing
Funding PendingIdentifying existing approved drugs that could be repurposed to treat DNM1 epilepsy. This approach can dramatically shorten the path to patient access. Screening through Unravel Bio's platform and partner organizations.
Read Publication →RNAi Knockdown + Gene Replacement
Academic ResearchA two-pronged RNA interference strategy: silencing the mutant DNM1 gene while simultaneously replacing it with a functional copy. Developed by Professor Frankel at Columbia University.
Read Publication →RNAi Allele-Specific Silencing
Outreach OngoingPrecision RNA interference targeting only the mutant allele, leaving the healthy copy of DNM1 intact. This approach minimizes off-target effects and is being developed for the most common recurrent variants.
Building the tools science requires.
2 Humanized Mouse Models
The first-ever humanized mouse models for DNM1 epilepsy, developed at The Jackson Laboratory in Maine. Essential for preclinical testing of all therapeutic candidates.
The Jackson LaboratoryiPSC Cell Lines
The first iPSC neuronal and retinal cell lines for DNM1 outside of China, developed in Canada. Covering the two most recurrent variants (~50% of all cases).
Canada · First Outside ChinaNatural History Study
A natural history study pending FDA grant approval through one of the top hospitals in the US. This study will be critical for future clinical trial design.
FDA Grant PendingHelp us fund the next breakthrough.
Every donation goes directly to vetted, translation-focused research programs. All funds flow through Rare Village Foundation's accounting operations — only paying out to official signed contracts and invoices.
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