Updates and Research for Angelman Syndrome
To see these videos about Angelman Syndrome please see this amazing piece organized by the Foundation for Angelman Syndrome Therapeutics (FAST)
The science is progressing faster than ever before through donations from family, friends, loved ones and biotechnology companies. Between 2014 and 2017 the progress for therapeutic potential has been huge. We are so grateful to all the work done by the FIRE team at FAST for making this dream our soon reality.
In September 2016 FAST received the largest donation in history of Angelman Syndrome to bring 4 therapeutics to clinical trials within 2 years. This donation was $5.8 million. In December, 2016, $1.25 million was raised toward a gene therapy program expected to start in humans, and in 2017 $4.1 million. Since that time we are in IND-enabling studies to develop gene modifying therapies to treat Angelman syndrome and we are working every day to ensure the timelines are as tight as possible. We need your help! DONATE NOW
On International Angelman Day, 2/15/2017 the Orphan Disease Center at the University of Pennsylvania partnered with the Foundation for Angelman Therapeutics (FAST) to bring a novel gene therapy approach to human clinical trials. This program is being run by the pioneer in gene therapy. Dr. Jim Wilson invented the vectors (adeno-associated virus), which is the vector that is used to bring the gene into the brain. We are so hopeful a cure is in sight. This program is currently underway and funding is crucial to see this through. DONATE NOW
In December 2017, the Foundation for Angelman Syndrome Therapeutics (FAST) launched a biotherapeutic company called GeneTx Biotherapeutics to help forward the promising strategy of antisense oligonucleotide therapy (ASOs), through the IND process to a Phase 1/2 clinical trial, and in March 2018, GeneTx was granted Orphan Drug Designation (ODD) for GTX-101, the first ASO granted this title for the treatment of Angelman syndrome.
We have an educational series that has been summarized for those interested in learning more about the research. Please read them at your leisure. It shows you how close we are to bringing therapeutics to children like Quincy. There is a roadmap to a cure, and you can be a part of this! http://cureangelman.org/road-map-to-a-cure/
For those of you that would like to learn about Angelman Syndrome and the current therapeutic pipeline please watch this video: https://www.youtube.com/watch?v=ZQ4MuGTaDWc
Summary of FAST research lectures: http://cureangelman.org/summary-of-fast-2015-research-lectures/
OV-101 as a potential therapeutic (trials started in Adults): http://cureangelman.org/ovid-therapeutics-ov101-as-a-potential-therapeutic/
Gene therapy for the treatment of Angelman Syndrome: Future clinical trials: http://cureangelman.org/gene-therapy-for-the-treatment-of-angelman-syndrome-future-clinical-trials/
The FIRE team at FAST turn on Ube3a in mice with Angelman Syndrome...the future gets MORE promising with Zinc Finger Technology: http://cureangelman.org/the-fire-team-of-fast-turns-on-ube3a-in-mice-with-angelman-syndrome-the-future-gets-even-more-promising-with-zinc-finger-technology/
Ketone esters for the treatment of seizures in AS: clinical trials coming in 2017: http://cureangelman.org/ketone-esters-for-the-treatment-of-seizures-in-as-clinical-trial-coming-soon/