Research Roadmap

Antisense oligonucleotides are synthetic molecules designed to silence the overexpressed LMNB1 gene that causes ADLD. Our first-in-human trial at Mayo Clinic is the first therapeutic intervention ever attempted for this disease, and our work through 2026 focuses on moving from a single patient to an expanded cohort.

A global, remote study of ADLD patients tracking symptoms, imaging, and blood samples over time. The dataset serves as a synthetic control arm for future trials, and the blood samples power our biomarker program so we can measure, in real time, whether a treatment is working.

A parallel genetic approach uses short hairpin RNA delivered by adeno-associated virus to reduce LMNB1 expression in brain cells. This program is designed as a second shot on goal, so that if one approach stalls, another is ready to move forward.

The fastest path to an approved treatment often starts with drugs that are already FDA-approved for other diseases. The ADLD Center pursues two complementary repurposing strategies: partner-led functional screening of compounds in ADLD cell models, and targeted repurposing based on shared disease biology.

Beyond our in-house programs, we are in active conversations with biotech companies whose platforms could be rapidly adapted to ADLD. Each candidate partnership is evaluated for mechanism, delivery, and how quickly it could reach patients.

Every drug candidate needs to prove it reaches the brain and reduces Lamin B1 in a living animal before the FDA will approve human trials. We are standing up a dedicated ADLD mouse model with The Jackson Laboratory that serves as a shared testing ground for ASOs, shRNA, and repurposed drugs.