Session Details

Session Details

03:45 PM - 05:30 PM - Room 307-308

Alvin Luk - Huidagene Therapeutics Inc

Co-Chair

03:45 PM - 05:30 PM - Room 307-308

Lisa Stanek - PhD - Affinia Therapeutics

Co-Chair

03:45 PM - 04:00 PM - Room 307-308

Fang Wan - MD, PhD - University of Massachusetts Chan Medical School

303: Optimized Artificial miRNAs Delivered by rAAV9 Dramatically Improve Survival, Respiratory and Motor Functions of SOD1^G93A-ALS Mice

04:00 PM - 04:15 PM - Room 307-308

Giridhar Murlidharan - PhD - Affinia Therapeutics

304: Reduction of Atxn2, a Therapeutic Target for Sporadic ALS, in Non-Human Primates Using a Novel, Intravenously Delivered AAV Capsid

04:15 PM - 04:30 PM - Room 307-308

Rachel Bailey - Ph.D. - UT Southwestern Medical Center

305: Development of Tau Isoform-Specific Reduction Therapy

04:30 PM - 04:45 PM - Room 307-308

Laura Rodriguez-Estevez - Universitat Autònoma de Barcelona (UAB)

306: Towards an AAV-Based Gene Therapy Strategy for Hereditary Spastic Paraplegia Type 52 (SPG52)

04:45 PM - 05:00 PM - Room 307-308

Barbara Terzic - PhD - Spark Therapeutics

307: Establishment of Gene Therapy Drug Development Assays Confirms Antiepileptic Activity of an AAV Encoding Neuropeptide Y (NPY) and Y2 Receptor (Y2R)

05:00 PM - 05:15 PM - Room 307-308

Alvin Luk - HuidaGene Therapeutics Co., Ltd.

308: Long-Term Safety and Feasibility of a Cas13-Based RNA Editing Therapy for MECP2 Duplication Syndrome in Humanized MECP2 Transgenic Mice

05:15 PM - 05:30 PM - Room 307-308

David Dimmock - Creyon Bio

309: AI Enabled ASO Design Can Lead to Rapid Initiation of Treatment for an Ultra-Rare Disorder Leading to Allele Selective Knockdown of a Toxic Protein and Consequent Clinical Improvement