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September 2023 Community Letter from Ultragenyx

Ultragenyx Pharmaceuticals (sponsor of the GTX-102, an Antisense oligonucleotide for the potential treatment of Angelman syndrome) has issued a letter to both ASF and FAST providing an update on their program. While they did not provide any timelines, they acknowledged actively working toward evaluating the safety and efficacy of GTX-102 in patients younger than 4 years of age, as well as those with non-deletion-type Angelman syndrome (i.e., mutations, imprinting center defects, uniparental disomy, mosaicism). They also commented on individuals previously treated with other ASOs, and that they are still gathering the information necessary to determine how and when they could be treated with another investigational product.

Click here to read the letter.