Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups
Ultragenyx Pharmaceutical Inc. (RARE)
Last ultragenyx pharmaceutical inc. earnings: 2/13 04:01 pm
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Source: GlobeNewswire
NOVATO, Calif., Oct. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the first patient has been dosed in the Aurora study (NCT07157254) evaluating the efficacy and safety of GTX-102 (apazunersen), an investigational antisense oligonucleotide (ASO) for Angelman syndrome (AS). Data from the Aurora study will expand the population of Angelman patients treated to include both younger and older patients and those with other non-deletion AS genotypes not included in the Phase 3 Aspire study. The fully enrolled Phase 3 Aspire study (NCT06617429) of GTX-102 is focused on patients aged 4 to 17 years with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. "The open-label basket design of the Aurora study will enable us to efficiently evaluate the safety and efficacy of GTX-102 in younger and older patients with Angelman syndrome and those with other genotypes," said Eric Crombez, M.D., chief medical officer at Ultragenyx. “
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- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4) [Yahoo! Finance]Yahoo! Finance
- Ultragenyx Reports Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)GlobeNewswire
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