GeneTx and Ultragenyx (RARE) Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

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Go back to GeneTx and Ultragenyx (RARE) Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

GeneTx and Ultragenyx Announce Positive Interim Phase 1/2 Data on Investigational GTX-102 Demonstrating Improvement in Patients with Angelman Syndrome

October 26, 2020 4:01 PM EDT

Efficacy in multiple functional measures observed in all five patients treated

Highest doses associated with a significant but reversible safety issue

Study amendment planned to focus on lower end of dose range

Conference call to discuss results planned for 5pm Eastern Time

SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), companies partnered... More