GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. announced that GeneTx's Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020. GeneTx's Phase 1/2 study, named KIK-AS (Knockdown of UBE3A-antisense in Kids with AS, is expected to enroll 20 0patients at multiple trial sites, beginning in the first half of 2020. The goal of this multiple dose, dose escalating, open-label study is to examine the safety, tolerability, and pharmacokinetics of GTX-102 in pediatric patients with Angelman syndrome.