GeneTx Bio-Ultragenyx's Angelman Syndrome Study To Start Enrollment In Canada

by Vandana Singh, Benzinga Editor

May 19, 2021 3:23 PM | 1 min read |

GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc RARE have received clearance from Health Canada to begin enrolling in the Phase 1/2 study of GTX-102 pediatric patients with Angelman syndrome in Canada.
Angelman syndrome is a rare neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene.
The first patient in Canada is expected to be enrolled in the early second half of 2021, with clinical data from some patients scheduled before the end of 2021.
The Phase 1/2, open-label, multiple-dose, dose-escalating study evaluates the safety, tolerability, and plasma and cerebrospinal fluid (CSF) concentrations of GTX-102 in pediatric patients.
Price Action: RARE shares are down 2.26% at $108.91 during the market session on the last check Wednesday.

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