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The first successful gene therapy for Huntington’s disease slows down the progression of the disease by 75%
Sep
Experimental gene therapy developed by unique For the first time he showed a dramatic slowdown in Huntington’s disease in humans, offering hope for a long -term disease.
How the process worked
Therapy, called AMT-130It was administered using a one -off neurosurgical procedure lasting between 12 to 20 hours using a modified viral vector to provide a corrective DNA to the brain areas affected by the disease. Patients treated High dose I saw their disease progression 75% slower over a period of 36 months compared to tailored external controls. Secondary results showed 60% slower decline in functional abilities. Treatment was generally well tolerated, without any serious safety problems.
Sarah TabriziThe director of the UCL’s Huntington’s Disease Center said that these discoveries mean “We now have the treatment of one of the more terrible diseases in the world. This is absolutely huge. I am really delighted.” Uniqure is planning to submit now Application for a biological license (BLA) to the American Food and Drug Agency at the beginning of 2026.
Regulatory progress and future steps
Earlier in 2025 FDA granted the AMT-130 Designation of groundbreaking therapywhich can speed up the review and regulatory paths. In the regulatory update, Uniqure revealed that he adapts key analytical and production plans to the FDA guidelines before submitting BLA.
The company intends to use External control setsLike Everoll-HD, for primary comparisons in your statistical analysis plan.
Why this breakthrough is so significant
- Modifying therapy: Until now, Huntington’s treatment only concerned symptoms; AMT-130 indicates the possibility of changing the trajectory of the disease.
- One -time administration: Because it is supplied with one surgical surgery, its benefits – if permanent – can be in recent years without repetitive dosage.
- High unmet need: In the USA, O 41,000 people live with Huntington’s and the Elder 200,000 Wear risk alleles.
- Scientific validation: The size of the effect in this study is one of the most convincing neurodegenerative diseases in research.
How AMT-130 is compared to other gene therapies
The AMT-130-one approach neurosurgical supply of a vector designed for suppressing mutated hunting protein-is a bit unique among genetic therapy, both in the delivery method and the purpose of the disease.
Here are some comparison points:
| Therapy | Delivery mode / regime | Indication / age group | Key strengths | Known restrictions / risk |
|---|---|---|---|---|
| AMT-130 (Uniqure) | Single stereotactic brain surgery (direct pond infusion) | The disease of the early Huntington manifesto | Promising function stabilization, one -time treatment, possible to improve the biomarker | Surgical risk, inflammatory complications, long -term long -term effectiveness unknown |
| Zolgensma (ONASEMNOGENE ABPARVOVEC-AVXS-101) | Individual intravenous or doopon in some attempts | Muscular spine (infants and older children) | Showed a dramatic improvement in survival, motor function; Among the most famous successful gene therapies | Very high costs, immune reactions, limited age window, administrative logistics |
| SKEWERSEN (Spinraza) | Repeated doopon injections (ongoing) | SMA in age groups | Reduces the burden of the disease, extends life, widely used; A well -understood safety profile | Requires regular dosage, invasiveness, patient/guardians, costs |
Warnings and restrictions
- No medicine: While the slowdown is impressive, does not reverse the disease or fully stop the course.
- Risk and operations costs: Invasive nature and complexity of delivery limit its use, especially in less talented surgical conditions.
- Unknown long -term durability: Whether the effect lasts over three years, it will just turn out.
- Regulatory obstacles and access: Approval does not guarantee access, and the valuation, reimbursement of costs and production scale remain the main challenges.
