Advancing Gene-Targeted Therapies for Central Nervous System Disorders Proceedings of a Workshop (2019) / Chapter Skim
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Appendix B: Workshop Agenda
Appendix B: Workshop Agenda
Pages 65-72
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From page 65... ...
, antisense oligonucleotides (ASOs) , and RNA interference, as well as gene producttargeted therapies.
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From page 66... ...
SESSION I: CURRENT LANDSCAPE AND LESSONS LEARNED Objectives: • Provide an overview of the current landscape of gene-targeted therapy approaches for central nervous system disorders. • Explore lessons learned from gene and ASO therapies that have achieved Food and Drug Administration approval -- including trans lation plans and which animal models were used in prelinical c tudies, use of dog models for RPE65, role of natural history tudies s s for SMA therapy, and other lessons learned in translation to clinical development.
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From page 67... ...
Gene Therapy for SMA Petra Kaufmann, AveXis 2:30 p.m. Lessons Learned from Unsuccessful Gene Therapy Trials of Neurotrophins for Neurodegenerative Diseases Jeffrey Kordower, Rush University 2:45 p.m.
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4:05 p.m. Speakers Anastasia Khvorova, University of Massachusetts Medical School Asa Abeliovich, Prevail Therapeutics Sarah DeVos, Denali Therapeutics 4:35 p.m.
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From page 69... ...
• Discuss what it takes to move beyond monogenetic disorders to develop gene therapy approaches for common, heterogeneous dis orders such as Alzheimer's and Parkinson's diseases. • Examine key challenges such as: o cell type-specific transduction; CNS o Regulation of viral gene expression to optimize safety and efficacy; and o Capsid engineering to improve tissue-specific targeting and blood–brain barrier penetration.
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Clinical Michael Panzara, Wave Biosciences Cristina Sampaio, CHDI Foundation 11:05 a.m. Regulatory Pathway Peter Marks, Food and Drug Administration Rune Kjeken, Norwegian Medicines Agency 11:25 a.m.
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Novel, Non-Viral Methods of Gene Therapy, Tunable Vectors, and AAV Manufacturing Capacity Robert Kotin, Generation Bio and University of Massachusetts Medical School 2:05 p.m. Using a Small-Molecule Drug to Modulate Splicing Anu Bhattacharyya, PTC Therapeutics 2:20 p.m.
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From page 72... ...
Synthesis of Key Workshop Themes and Future Directions Story Landis, Co-Chair, Forum on Neuroscience and Nervous System Disorders (Co-Chair) Lamya Shihabuddin, Sanofi (Co-Chair)
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