2021 FAST Science Summit

• Development of Rare Disease Therapies: Overcoming Challenges
  Explore the journey of developing rare disease therapies with Emil Kakkis, founder of Ultragenyx. Learn about the challenges, solutions, and the importance of perseverance in this field.
• Engineering Human Stem Cell Models for Multiple Angelman Syndrome (Epi)Genotypes
  Explore the groundbreaking research at North Carolina State University on engineering human stem cell models for multiple Angelman Syndrome genotypes and epigenotypes. Discover our unique approach and dedication to understanding and addressing this complex condition.
• GTX-102 Phase 1/2 Clinical Trial Update, Development of an ASO for Angelman Syndrome: Science and Regulation
  Get the latest on the GTX-102 Phase 1/2 clinical trial for Angelman syndrome treatment. Discover trial protocol, safety concerns, preliminary results, and future directions.
• Gene Therapy for Angelman Syndrome through RNA Interference
  Explore the potential of RNA gene therapy for Angelman syndrome. This one-time injection could last a lifetime, potentially increasing UBE3A expression and alleviating symptoms.
• Genetic Approaches for Treating Angelman Syndrome
  Explore the latest genetic approaches to treat Angelman Syndrome, including gene therapy, genome editing, and RNA interference. Learn about the challenges, clinical trials, and future directions.
• Hematopoietic Stem Cell Gene Therapy for Angelman Syndrome: Progress and Process
  Explore the progress of hematopoietic stem cell gene therapy for Angelman syndrome. Learn about the process, preclinical studies, future clinical trials, and the potential benefits of this promising treatment.
• Ionis Pharmaceuticals Angelman Syndrome Program Update
  Explore Ionis Pharmaceuticals’ progress in their Angelman Syndrome program, including a clinical trial initiation, collaborations for research, and an introduction to the Halos trial. Stay updated with the latest developments.
• NNZ-2591 as a Potential Treatment for Angelman Syndrome
  Explore Neuron Pharmaceuticals’ NNZ-2591, a promising treatment for Angelman syndrome. Learn about its development stages, how it works, and its potential benefits in addressing this rare neurodevelopmental disorder.
• Now Is the Time for Molecular Therapies for Angelman Syndrome
  Explore the potential of molecular therapies for Angelman Syndrome. Learn about gene therapy, viral-based therapies, and antisense oligonucleotides, and how they can address this genetic disorder.
• Overview of the Therapeutic Landscape for Angelman Syndrome
  Explore the therapeutic landscape for Angelman Syndrome with Allyson Berent, Chief Science Officer at FAST. Discover potential treatments, genetic insights, and the importance of research collaboration.
• Panel Discussion with Pharma
  Get the latest updates on clinical trials for Angelman Syndrome from the 2021 FAST Science Summit. Learn about trial progress, patient selection, drug access, and future plans from leading pharma representatives.
• Panel Discussion with Researchers
  Explore key insights from the 2021 FAST Science Summit panel discussion, where experts discuss advancements and potential treatments for Angelman syndrome, a rare genetic disorder.
• Putting Patients at the Center
  Explore Taysha Gene Therapies’ approach to developing treatments for rare neurological diseases, their partnership with UT Southwestern, and their commitment to patient-centered care.
• Roche Pharmaceuticals Angelman Syndrome Program Update to the FAST Community
  “Explore the progress of Roche’s Angelman Syndrome Program, including updates on the TANGELO Phase 1 drug study and the FREESIAS observational study. Learn about our commitment to improving lives.”
• Stem Cell and Gene Therapy Platforms
  Explore the latest advancements in stem cell and gene therapy platforms with Jan A. Nolta at the 2021 FAST Science Summit. Discover ongoing research, clinical trials, and future directions at UC Davis.
• The Creation of a Robust Infrastructure Including Molecular, Neurobehavior, and Biomarker Testing for Pre-Clinical Drug Evaluation in Angelman Syndrome
  Explore the creation of a robust infrastructure for pre-clinical drug evaluation in Angelman syndrome, focusing on molecular, neurobehavioral, and biomarker testing. Discover potential treatments and advances in research.
• Translational Research in a Large Animal Model of Angelman Syndrome
  Explore the development and importance of a pig model for Angelman Syndrome research. Discover how this model aids in understanding the condition’s pathophysiology and testing potential therapies.
• Update on Angelman Syndrome Human iPSC Biorepository Project and the Angelman Syndrome Large Deletion Mouse Model
  Advancing understanding and treatment of Angelman Syndrome through iPSC cell lines and mouse models. Valuable resources for researchers.
• Update on the UK Natural History Study and Wearable Devices
  Explore the latest updates on the UK Natural History Study and the role of wearable devices in medical research. Discover the importance of these studies and the challenges faced in conducting them.
• We’re There When You Need Us: A 24-7 Emergency Care Hotline for the AS Community
  Explore our 24-7 emergency care hotline for Angelman Syndrome (AS), providing standardized, expert care for children with AS. Join us in revolutionizing rare disease treatment.