REsearch roadmap
At the ADLD Center, we are committed to accelerating progress toward a cure for Autosomal Dominant Leukodystrophy (ADLD). Our updated agile roadmap outlines our strategic priorities through 2027, focusing on parallel streams of clinical trials, novel drug development, and comprehensive disease tracking.
1. COLLABORATIVE DRUG TRIAL
We are moving aggressively toward clinical interventions through strategic partnerships.
Pilot Clinical Trial: Commencing in Q4 2025, we are launching a pilot clinical trial ($n=1$). Preliminary readouts are expected between Q1 and Q2 2026.
Scale-Up & Expansion: If positive preliminary data is observed, our partners will initiate CMC (Chemistry, Manufacturing, and Controls) scale-up and regulatory filings in mid-2026. This lays the groundwork for an expanded clinical trial targeted to begin in late 2026 or 2027.
2. NOVEL DRUG DEVELOPMENT
Our R&D pipeline is focused on advanced genetic therapies.
Drug Discovery (Current): We are currently synthesizing RNAi and control candidates, with vigorous in vitro and in vivo screening ongoing through early 2026.
Development Phase: Following a targeted fundraising round for Phase II in Q1 2026, we will advance to synthesizing siRNA + AAV vectors.
Path to the Clinic: We aim to file an Investigational New Drug (IND) application in Q1 2027. Our subsequent Clinical Trial design is intended to utilize our Natural History Study data as a placebo substitute, allowing us to administer the active drug to all participating patients.
3. NATURAL HISTORY STUDY
Understanding the progression of ADLD is critical for regulatory success and trial design.
Retrospective Study (Active): Initiated in Q1 2025, this study evaluates historical patient data comprehensively to establish a baseline for disease progression. This phase will run through the end of 2026.
Prospective Study (Future): Beginning in 2027, insights from the retrospective analysis will inform a Prospective Study. This phase will observe new patients’ clinical courses in real-time to capture evolving disease insights.
4. STRATEGIC GRANTS & PARTNERSHIPS
We continue to fund and collaborate on high-impact research with leading institutions.
Henry Ford Health: Research focused on discovering a plasma miRNA biomarker for ADLD is currently underway, with a final report expected in mid-2026.
Mayo Clinic: We are supporting preparations for the ASO clinical trial, with a final report also due in mid-2026.
Future Funding: As these projects conclude, we are actively identifying potential follow-on grants to sustain momentum in these critical research areas.