CHDI conference session V - clinical updates from Uniqure AMT-130 program

CHDI conference session V - clinical updates from Uniqure AMT-130 program

David Cooper David Cooper from Uniqure spoke about the current status and future plan for the first gene therapy trial for HD involving an AAV5 expressing a microRNA targeting HTT human exon-1 sequences, termed AMT-130. David described the preclinical work conducted to support the clinical trial data, in work that…
CHDI Conference Session V - Clinical trial updates

CHDI Conference Session V - Clinical trial updates

Drs. Lauren Boak and Peter McColgan (Roche) Drs. Lauren Boak and Peter McColgan discussed additional results from the tominersen Generation-HD1 phase III trial. Lauren spoke about the on-treatment and post-treatment effects of tominersen, both during the Phase III study and other studies involving this drug, with the focus of dose…
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