GeneTx, Ultragenyx (RARE) Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome

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Go back to GeneTx, Ultragenyx (RARE) Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome

GeneTx and Ultragenyx Announce GTX-102 Granted Fast Track Designation by U.S. FDA for Treatment of Angelman Syndrome

May 4, 2020 8:31 AM EDT

SARASOTA, Fla. and NOVATO, Calif., May 04, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS.

GeneTx is committed to improving the quality of life of individuals living with... More