This year was a notable year for the Angelman syndrome community — one filled with excitement, some growing pains, frustrations, but most importantly, progress towards potential therapeutics for individuals living with Angelman syndrome.
Here’s a review of our year:
NIH announced a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases, Angelman syndrome being one of them.
FAST partnered with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome.
Ionis Pharmaceuticals and Ultragenyx Pharmaceuticals provided promising updates on their respective investigational ASO clinical trials.
FAST has grown its staff, hiring four critical new staffers to support the operationalization of our mission.
The 16th Annual Global Science Summit & Gala was a success for so many reasons — save the date for next year’s event: Nov 8-9, 2024 in Orlando, FL!
We added FAST Poland to our list of global chapters!
Our community did an amazing job raising funds through social media, hosting events, and peer-to-peer campaigns. We look forward to another successful year of Cure Angelman Now (CAN) fundraising — stay tuned in January 2024 for more details!
We could not do what we do without you. We wish you and your family a safe and happy holiday season. See you back here in 2024 when we hit the ground running!
Sincerely,
The FAST Board & Staff