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Jenna Payesko


Gene Therapy for CLN6 Batten Disease Shows Positive Interim Results

August 03, 2019

Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.

Huntington Disease Gene Therapy AMT-130 Wins FDA Fast Track Designation

April 08, 2019

The planned phase 1/2 trial of the recombinant AAV5 vector treatment, the first one-time administered AAV gene therapy to enter clinical testing for Huntington disease, is expected to begin dosing patients in the second half of 2019.