- 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.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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