BIOBRIEF
Huntington's has no cure. Twelve patients who received a one time gene therapy into the brain still outpace untreated peers at year four, by 44%, down from 80% at year three.
Twelve people received a one-time gene therapy for Huntington's disease four years ago in an early-stage uniQure trial. They are still ahead of an untreated comparison group on a standard clinical scale, but the gap is narrower than at year three.
uniQure's September 29 release reports 44% slowing on cUHDRS (a composite scale measuring cognition, movement and daily function) in 12 high-dose patients versus propensity-matched external controls drawn from the ENROLL-HD natural-history dataset. The result did not reach statistical significance (p=0.144). On TFC, a daily-function rating, the same 12 showed 61% slowing (nominal p=0.008). At 36 months, the 15-patient update had read 80% on cUHDRS and 67% on TFC.
The narrower year-four number carries a sponsor's caveat: 53% of matched control patients were missing from the 48-month comparison, disproportionately faster-progressing ones. A post-hoc re-run against an earlier control set yields 53.5% on cUHDRS (nominal p=0.041) and 68.3% on TFC (nominal p=0.001). That is uniQure's interpretation, not independent verification.
AMT-130 is delivered once by MRI-guided neurosurgery to silence the huntingtin gene. The release reports treatment-related serious central-nervous-system inflammation that resolved. The company says its BLA was filed before these results. The four-year data are the first long-term signal in a fatal inherited disease with no approved disease-modifying treatment, and the new engineering question is what 44% at year four becomes at year five.