FAST announces the launch of two biotech companies to advance investigational gene-targeted therapies for Angelman syndrome
FAST launches MavriX Bio and CourageAS Bio, two new biotech companies advancing gene replacement and gene-editing therapies for Angelman syndrome under its AS²Bio accelerator.
Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx announces the first patients dosed in its pivotal Phase 3 ASPIRE study evaluating GTX-102 for Angelman syndrome, a major step toward potential treatment.
Ionis Pharmaceuticals releases information on their planned phase 3 clinical trial for Angelman syndrome
Ionis Pharmaceuticals announces ION582 will advance into Phase 3, called REVEAL, with details to be presented at the FAST Global Science Summit on November 9th.
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
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
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 […]