Ultragenyx Pharma (RARE) Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance
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Ultragenyx Pharma (RARE), GeneTx Report Investigational New Drug Application Active for GTX-102 in Patients with Angelman Syndrome
January 15, 2020 4:02 PM ESTUltragenyx Pharmaceutical Inc. (NASDAQ: RARE) and GeneTx Biotherapeutics LLC, today announced that GeneTxs 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.
"The FDAs clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community, said Paula Evans, Chief Executive Officer at GeneTx. Assessing an... More
