Disease Area
Spinocerebellar ataxia
SCA1, SCA2, and SCA7
SCA1: Symptoms start with balance issues and hand incoordination. It is a progressive disease to various symptoms such as speech and swallowing difficulties, involuntary eye movement, muscle atrophy, and cognitive impairments.
SCA2: It is a progressive disorder with ataxia of limbs, speech difficulty, eye movement issues, and neuropathy.
SCA7: Early symptoms include loss of coordination, macular degeneration, then slowly progress to speech and swallowing difficulties. Some populations go blind.
🧠 Number of Patients in the U.S.
SCA1: 3,300 - 6,600
SCA2: 4,000 in US and Europe
SCA7: 1,000
⏳ Onset ages
SCA1: Early adulthood, and fatal in 20 - 30 years.
SCA2: 20 - 50 years old and fatal in 10 - 15 years.
SCA7: 20 - 40 years old
🚫 0 Disease-Modifying Therapies
No approved treatments currently exist to slow, stop, or reverse disease progression
💸 Annually $30K - $60K/ 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 SCA 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 Spinocerebella Ataxia and enable safe, long-term treatment for patients.
