Enabling Targeted Muscle Delivery Through Aptamer-siRNA Conjugates Beyond Antibody Conjugates

• Repurposing transferrin receptor 1 (TfR1)-binding aptamers as targeting ligands for systemic siRNA delivery
• Achieves selective and durable siRNA activity in skeletal muscle following a single systemic dose
• Establishes aptamer–siRNA conjugates as a promising platform for next-generation targeted oligonucleotide therapeutics

Speakers:

Thomas Ormsby

Associate Professor

University of Massachusetts Medical School