Ultragenyx Pharma (RARE), GeneTx Report Investigational New Drug Application Active for GTX-102 in Patients with Angelman Syndrome

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Go back to Ultragenyx Pharma (RARE), GeneTx Report Investigational New Drug Application Active for GTX-102 in Patients with Angelman Syndrome

GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

January 15, 2020 4:01 PM EST

SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, 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.

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