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

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 Angelman syndrome,” said Paula Evans, Chief Executive Officer, GeneTx. “Receiving Fast Track designation for GTX-102 is an important recognition of our antisense oligonucleotide program and the promise it might offer to this patient population.”

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