FDA grants Breakthrough Therapy Designation to Angelman Syndrome treatment

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Ionis Pharmaceuticals for ION582, a novel, antisense oligonucleotide (ASO) therapy designed to treat Angelman Syndrome. Angelman syndrome is a rare neurodevelopmental disorder characterized by changes in brain structure, severe intellectual disability, impairments in speech, motor function, epilepsy, sleep, and unique behaviors that ­affects 1 in 10,000-24,000 people and for which no cure or treatment is currently available. This is a significant step that allows the trial to expedite the development and review of the treatment, bringing hope for faster access to treatment for patients and families.

Years of foundational research led to this milestone, including pivotal work by Dr. Mingshan Xue at the Duncan NRI. His proof-of-concept studies revealed that both children and adults may benefit from ASO therapy - broadening the therapeutic window and shaping the clinical trial’s design.

This designation not only marks a major advancement for Angelman Syndrome patients but also highlights Texas Children’s Hospital as a key site in the clinical trial journey.

Read more about this designation here: https://ir.ionis.com/news-releases/news-release-details/ionis-receives-us-fda-breakthrough-therapy-designation-ion582

Read about Dr. Xue’s work here: https://www.texaschildrens.org/content/research/therapeutic-window-for-angelman-syndrome-patients-may-be-broader-than-previously 

About the Texas Children’s Duncan NRI

Founded in 2010 at Texas Children’s Hospital, the Duncan NRI researches and discovers treatments for devastating brain disorders that impact more than a billion people worldwide, including 300 million children. As the largest children’s hospital in America, Texas Children’s Hospital is uniquely positioned to study brain development in children to understand how the brain changes later in life. The Texas Children’s Duncan NRI spans 344,000 square feet and covers 13 floors. It is home to more than 400 multidisciplinary researchers who collaborate with physicians for rapid lab-to-bedside discovery, facilitating innovation that would be impossible in traditional scientific silos. This approach has led to 1,700 published discoveries, the identification of 90 novel disease-causing genes, and major breakthroughs in autism, epilepsy, Alzheimer’s disease, depression, obsessive compulsive disorder, and other neurological and psychiatric disorders. Texas Children’s Hospital covers all of the Duncan NRI’s annual operating costs, so every dollar donated directly supports research. 

About Texas Children’s Hospital

Texas Children’s, a nonprofit health care organization, is committed to creating a healthier future for children and women throughout the global community by leading in patient care, education and research. Consistently ranked as the best children’s hospital in Texas and among the top in the nation, Texas Children’s has garnered widespread recognition for its expertise and breakthroughs in pediatric and women’s health. The system includes the Texas Children’s Duncan NRI; the Feigin Tower for pediatric research; Texas Children’s Pavilion for Women, a comprehensive obstetrics/gynecology facility focusing on high-risk births; Texas Children’s Hospital West Campus, a community hospital in suburban West Houston; Texas Children’s Hospital The Woodlands, the first hospital devoted to children’s care for communities north of Houston and Texas Children’s Hospital North Austin, the new state-of-the-art facility providing world-class pediatric and maternal care to Austin families. The organization also created Texas Children’s Health Plan, the nation’s first HMO focused on children; Texas Children’s Pediatrics, the largest pediatric primary care network in the country; Texas Children’s Urgent Care clinics that specialize in after-hours care tailored specifically for children; and a global health program that is channeling care to children and women all over the world. Texas Children’s Hospital is affiliated with Baylor College of Medicine.