UniQure to release four-year Huntington’s gene therapy results by September
UniQure, a biotech firm, will report four-year data from its Huntington’s disease gene therapy trial before the end of September.
The therapy, AMT-130, has already been submitted to regulators in the United States and United Kingdom. Earlier three-year data showed a high dose given to 12 patients slowed disease progression by about 75% compared with an external control group drawn from a natural history study.
The upcoming readout is closely watched by investors and clinicians because it will provide longer‑term efficacy and safety information that could influence regulatory decisions and market expectations.
This writeup was produced by pharmadog from original reporting by STAT.
Original headline: “STAT+: What to expect from UniQure’s upcoming Huntington’s gene therapy readout”
read at STAT ↗
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