Angelman Syndrome Research
OVID RX Clinical Trial Approach
The STARS Phase 2 clinical trial will begin enrolling participants soon. We called on the Angelman syndrome community to help us design and identify the relevant endpoints of the trial. The community also helped select the final STARS clinical trial name and logo. The STARS logo was designed by Kaline, a proud parent of a child diagnosed with Angelman syndrome. The image “reaching for the STARS” is a depiction of her son, and every other person diagnosed with Angelman syndrome, standing with one foot firmly planted while using the “T” to stretch as far as they can to reach the brightest star in the sky. We thank everyone who participated in the process. We believe this name reflects how we feel about our Angelman syndrome community, and the clinical program reflects their needs.
Angelman Syndrome Gene Therapy
Agilis Biotherapeutics have entered into an exclusive worldwide license agreement with the University of South Florida (USF) for the treatment of Angelman syndrome using gene therapy technology developed by Edwin Weeber, PhD, Director of the Neurobiology of Learning and Memory and Chief Scientific Officer at the USF Health Byrd Alzheimer's Research Institute at the University of South Florida, and one of the world’s foremost Angelman syndrome investigators.
(Read the Press Release). More from FAST...
Australia Funds First Research Fellowship!
In an Australian first, FAST Australia have awarded a research grant
to Professor Joel Mackay and the University of Sydney for a two year project entitled “RNA binding motifs as therapeutic targets forAngelman syndrome”. Cutting edge molecular biology approaches will be used to define the mechanism by which UBE3A, the causative gene for Angelman syndrome, is switched off in those afflicted with the disorder. The insight gained from this program of research will serve as a platform from which molecules that can reactivate the UBE3A gene might be rationally designed. If successful, subsequent work would explore the potential of such inhibitors as a treatment for Angelman syndrome.
Despite Angelman syndrome developments advancing at great speed internationally, research in Australia has been minimal; hence the formation of FAST Australia. Australia has a reputation for a number of notable scientific discoveries and our scientists have a lot to offer the international search for a therapeutic for Angelman syndrome. FAST Australia has announced their first research project in partnership with the Mater Hospital and Mater Medical Research Institute, the Angelman Syndrome Register. The study will be a “first of its kind” register of Angelman syndrome patients and related medical professionals who can be contacted in the event of the development, participation in a study, a therapeutic or a cure. Captured information will empower the community of families affected by Angelman syndrome community by providing a wide-ranging collection of relevant data on the syndrome. FAST Australia will continue to raise funds and promote collaborative research in Australia until a therapeutic to treat Angelman syndrome is discovered and used in practice for all affected individuals.