Ultragenyx Community Update October 2024
The Phase 3 Efficacy and Safety Study of GTX-102 (ASO) in Pediatrics, sponsored by Ultragenyx, is now listed on ClinicalTrials.gov. Interested in learning more? Watch the Ultragenyx Community Webinar
Neuren Pharmaceuticals announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS)
Neuren Pharmaceuticals (ASX: NEU) announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). Read the full press release here. Highlights: Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline after 13 weeks that was statistically significant (Wilcoxon signed […]
Jim Wilson move from UPENN to Biotech and what this means for Angelman syndrome.
“This is a great decision by Dr. Wilson and that of The University of Pennsylvania to get the development of these gene therapies out of academia and into a well-resourced biotech environment that can execute more efficiently. This is exciting news for rare disease and for other companies that can now leverage the expertise of […]
To Further Advance Gene Therapy Research, Industry Pioneer Jim Wilson is Forming Two Independent Companies Dedicated to Genetic Medicine
Read the press release
A new publication represents a possible small-molecule approach to potentially treat the synaptic dysfunction observed in Angelman syndrome.
Peptidomimetic inhibitors targeting TrkB/PSD-95 signaling improves cognition and seizure outcomes in an Angelman Syndrome mouse model. Link to Article: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11185757/ We are excited to announce a new publication from Dr. John Marshall at Brown University made possible by FAST funding. The project started in late September 2022, aiming to modulate synaptic function with two novel […]
Ionis announces positive detailed results from the HALOS Study of ION582 in people with Angelman syndrome
Ionis Pharmaceuticals issued a press release today regarding their Phase 1/2a HALOS clinical trial of ION582 for individuals living with Angelman syndrome. Join the livestream on July 24th starting at 3:10pm ET where Ionis and other industry leaders will present information to parents and caregivers at the Angelman Syndrome Foundation (ASF) Family Conference. You can […]
Ultragenyx Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program
Today, Ultragenyx Pharmaceuticals shared a press release announcing a “Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome Program.” Read the full press release here.
Ionis announces positive topline results from Phase 1/2a trial of ION582 for Angelman syndrome
Exciting news for the Angelman syndrome community! Ionis’ Angelman syndrome program, ION582, has just shared positive preliminary results from the Phase 1/2a HALOS clinical trial. Ionis will be providing an update at the upcoming Angelman Syndrome Foundation meeting in July, which will be streamed for those who cannot attend. Read the community letter from Ionis […]
Ultragenyx Announces Positive Interim Phase 1/2 Data in Patients with Angelman Syndrome After Treatment with GTX-102
We’re thrilled to share an update from Ultragenyx regarding reported positive interim data from the ongoing Phase 1/2 study in patients living with deletion genotype Angelman syndrome after treatment with investigational GTX-102. From company press release: “The totality of these interim data demonstrates that treatment with GTX-102 resulted in rapid, multi-domain improvements that continued during […]
New FAST-TRAC grant collaboration with Dr. Alvin Huang
A FAST-funded study unveils STEP-RNP, a non-viral gene editing platform showing improved motor, cognitive function in AS mice—an early but promising step toward treating Angelman syndrome.