Disease Area
Huntington's Disease
A fatal, inherited neurodegenerative disorder driven by toxic RNA repeat expansions
Huntington's disease causes progressive motor dysfunction, cognitive decline, and psychiatric symptoms, with no approved disease-modifying therapies available. The underlying pathology is directly linked to expanded RNA repeats that disrupt neuronal function and ultimately lead to irreversible neurodegeneration.
🧠 30,000 Patients in the U.S.
Living with Huntington's disease today, while many more remain genetically at risk
⏳ Ages 30–50
The most common window for symptom onset, striking during peak working and family-raising years
🚫 0 Disease-Modifying Therapies
No approved treatments currently exist to slow, stop, or reverse disease progression
💸 Annually $60K–$120K / Patient
Annual care costs driven by medical visits, long-term medications, physical and psychiatric care, assistive devices, and increasing caregiving needs as independence is lost
🧬 50% Inheritance Risk
Each child of an affected parent has a 50% chance of inheriting the Huntington’s disease mutation, extending the impact far beyond those already diagnosed
Our Mission & Vision
Our mission is to develop scalable, small-molecule therapies that directly target the RNA-level cause of Huntington's disease and enable safe, long-term treatment for patients.