First clinical trial in the UK starts soon
GeneTx and Ultragenyx Receive Approval from UK Regulatory Agency to Begin Clinical Study of GTX-102 for the Treatment of Angelman Syndrome
Clinical trials for GTX-102 will start in the early second half of 2021 in the UK.
Under the amended protocol approved in the U.K. and Canada, approximately 12 patients will be enrolled into two cohorts split by age: patients ages 4 to 7 years will be enrolled into Cohort 4, and patients ages 8 to 17 years will be enrolled into Cohort 5
Full press release is here.
Read about the KIK-AS (Knockdown of UBE3A-antisense in kids with Angelman syndrome) study here.
These two talks from 2020 FAST Science Summit are about GTX-102 treatment and Phase 1/2 clinical trials results in the US: