Roadmap

How inherited CSVD research moves toward targeted treatment.

A focused route from genetic cause to credible therapeutic evidence for COL4A1-related disease and CADASIL.

Therapeutic direction

Keep the science close to the disease, the evidence, and the people affected.

CSVD Therapeutics is building a practical translational path for inherited cerebral small vessel disease. The work begins with defined genetic causes, uses disease-relevant models to test candidate interventions, and prioritizes the evidence needed before any treatment concept can be responsibly moved toward patients.

Genetics

Start with a defined cause

Focus on inherited CSVD where variants in COL4A1 or NOTCH3 give a precise biological starting point.

Models

Test disease-relevant systems

Use cellular and vascular readouts to ask whether candidate therapies change measurable disease biology.

Tools

Develop credible interventions

Evaluate approaches such as plasmid systems, viral vectors, ASOs, CRISPR strategies, and drug repurposing where appropriate.

Translation

Build readiness for patients

Connect biomarkers, safety, delivery, natural history, and patient priorities into the evidence package needed for future trials.

University of Sheffield Sheffield Institute for Translational Neuroscience National Institute for Health and Care Research Alzheimer's Research UK British Heart Foundation