Session Details

Session Details

03:45 PM - 05:30 PM - Ballroom 1

Isabel Aznarez - Stoke Therapeutics

Co-Chair

03:45 PM - 05:30 PM - Ballroom 1

Afrooz Rashnonejad - PhD - Center for Gene Therapy at Abigale Wexner Research Institute of Nationwide Children's Hospital

Co-Chair

03:45 PM - 04:00 PM - Ballroom 1

Beltran Borges - MD - University of California San Francisco

331: Prenatal Intra-Amniotic Administration of an Antisense Oligonucleotide Achieves Widespread Biodistribution with Therapeutic Levels in Fetal Lambs

04:00 PM - 04:15 PM - Ballroom 1

Roni Klein - Rosalind Franklin University of Medicine and Science

332: Targeting NLRP3 Splicing Variants with Antisense Oligonucleotides to Control Pathological Inflammation

04:15 PM - 04:30 PM - Ballroom 1

Sandra Smieszek - PhD - Vanda Pharmaceuticals Inc.

333: First in Class ASO Targeting IGHMBP2 Cryptic Splice Variant: Efficacy and Safety

04:30 PM - 04:45 PM - Ballroom 1

Wei Liu - Wave Life Sciences

334: Potent, Durable mRNA Knockdown in Extrahepatic Tissues Using siRNAs with Novel Phosphoryl Guanidine Backbone Variants

04:45 PM - 05:00 PM - Ballroom 1

Alex Felix - University of Pennsylvania

335: miRNA Site-Blocking Oligonucleotides as a Novel Therapeutic Strategy for STXBP1 Encephalopathy

05:00 PM - 05:15 PM - Ballroom 1

Jillian Belgrad - UMass Chan Medical School

336: Modulation of Somatic Repeat Expansion with Small Interfering RNAs

05:15 PM - 05:30 PM - Ballroom 1

Rachael Miller - UMass Chan Medical School

337: Therapeutic Oligonucleotides Induce Acute Toxicity in CNS, Preventable by the Addition of Divalent Cations to Formulation