Experimental Gene Therapy AMT-130 Slows Huntington’s Disease Progression, says study

An experimental gene therapy has been found to slow the progression of Huntington’s disease in a pivotal Phase 1/2 study, marking a potential breakthrough toward the first genetic treatment for the condition, according to its developer.

Study Findings

• Patients who received a high dose of the therapy (AMT-130) showed a 75% slowdown in disease progression after 36 months.
• Full study results are yet to be published in a peer-reviewed journal.

How the Treatment Works

• The therapy uses a harmless virus to deliver microRNA into the brain.
• This molecule blocks faulty genetic instructions that cause the harmful buildup of the mutant huntingtin protein — the main driver of the disease.

About Huntington’s Disease

• Inherited neurodegenerative disorder that causes gradual breakdown and death of neurons in the brain.
• Affects brain regions that control voluntary movement, as well as behavior, thinking, and personality.

Key Symptoms:

• Chorea (uncontrollable, dance-like movements in fingers, feet, face, or torso)
• Abnormal body postures
• Behavioral and emotional changes
• Cognitive decline
• Symptoms usually appear in middle age (adult HD), though rare cases occur in children (juvenile HD).
• The disease progressively worsens over time.

(Source: Science)