Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Shots: Ionis has completed enrollment in Cohort 1 (n=136; 2 to <18yrs.) of the global P-III (REVEAL) study assessing obudanersen (ION582) in pts with AS with genetic confirmation of either a UBE3A deletion or UBE3A mutation The Cohort 2 comprises adult pts (18 to ≤50yrs.) living with AS, with enrollment expected to be completed in…

ByRidhi RastogiJuly 7, 2026

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Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

Shots: The US FDA has granted BTD to ION582 (UBE3A protein expression stimulant) for the treatment of Angelman syndrome Designation was based on P-I/II (HALOS) trial assessing ION582 in pts with Angelman syndrome, which showed improvements across all functional domains incl. communication, cognition & motor function; safety was favorable Additionally, ION582 is being investigated in…

ByRidhi RastogiSeptember 12, 2025

Ionis Completes Patient Enrollment in Cohort 1 of P-III (REVEAL) Study of Obudanersen in Angelman syndrome (AS)

Ionis’ ION582 Receives the US FDA’s Breakthrough Therapy Designation to Treat Angelman Syndrome

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