Science and Pharmaceutical Updates

FAST works with leading researchers and pharmaceutical companies using cutting edge technology to accelerate our mission to find a cure for Angelman syndrome. At the Global Science Summit a few weeks ago, they shared an update on all of their work this past year that continues to confirm our mission to find a cure is possible.

Science Summit Update

https://www.youtube.com/watch?v=C2dDiEm7FFc&list=PLCTi8nkE1YPcaqcWDTrSB_NmVHXPJLo9G&index=1&t=18s

Keynote address: Cell & Gene Therapy Platforms

https://youtu.be/Cwt7BHUyWMcOverview of the Therapeutic Landscape for Angelman Syndromehttps://youtu.be/aA7cfmWr_KwCreation of a Robust Infrastructure Including Molecular, Neurobehavior & Biomarker Testinghttps://youtu.be/khVi7M059qoUpdate on AS Human IPSC Biorepository Project & the Angelman Syndrome Large Deletion Mouse Modelhttps://youtu.be/P2DqRGQTyl4

Engineering Human Cell Models for Multiple Angelman Syndrome (Epi)Genotypes

https://youtu.be/RSM0BepCWwMGene Therapy for Angelman Syndrome Through RNA Interferencehttps://youtu.be/Iw0lfS9pYNcTranslational Research in a Large Animal Model of Angelman Syndromehttps://youtu.be/H_kMcgmA-PY24/7 Emergency Care Hotlinehttps://youtu.be/OmPSqg53Q24Genetic Approaches for Treating Angelman Syndromehttps://youtu.be/2sq0PKQgfxQ

Hematopoietic Cell Gene Therapy for Angelman Syndrome

https://youtu.be/zlapDAdAwJAPanel Discussion with Researchers

Pharmaceutical Update

https://youtu.be/ulk7IKb9rtIKeynote address: Now Is the Time for Molecular Therapyhttps://youtu.be/_d9rpKHacN0Taysha Gene Therapies: Putting Patients at the Centerhttps://youtu.be/gfOgI3VOTGUNeuren Pharmaceuticals Ltd.: NNZ-2591 As A Potential Treatment For Angelman Syndromehttps://youtu.be/432S1RUevKoIonis Pharmaceuticals: Angelman Syndrome Program Updatehttps://youtu.be/qq6E9iMMicEGeneTx Biotherapeutics: GTX 102 Phase 1/2 Clinical Trial Updatehttps://youtu.be/65wZQf-WSI0Roche Pharmaceuticals: Angelman Syndrome Program Updatehttps://youtu.be/8_Qt0SVCHPwUltragenyx Pharmaceutical Inc.: Overcoming Challengeshttps://youtu.be/pm2KOr0xFacUpdate on the UK Natural History Studyhttps://youtu.be/diZkP1QC-W4

Panel Discussion with Pharma