Orna Therapeutics, Inc.

Amy M Becker

Thomas Barnes

Robert Alexander Wesselhoeft

Allen T Horhota

Junghoon Yang

Gregory Motz

Kevin J Kauffman

Brian Goodman

Jui Dutta-Simmons

Disclosed herein are novel lipids that can be used in combination with other lipid components, such as helper lipids, structural lipids, and cholesterols, to form lipid nanoparticles for delivery of therapeutic agents, such as nucleic acids (e.g., circular polynucleotides), both in vitro and in vivo.

Lipid nanoparticle compositions for delivering circular polynucleotides

Circular RNA, along with related compositions and methods are described herein. In some embodiments, the inventive circular RNA comprises group I intron fragments, spacers, an IRES, duplex forming regions, and an expression sequence. In some embodiments, the expression sequence encodes an antigen. In some embodiments, circular RNA of the invention has improved expression, functional stability, immunogenicity, ease of manufacturing, and/or half-life when compared to linear RNA. In some embodiments, inventive methods and constructs result in improved circularization efficiency, splicing efficiency, and/or purity when compared to existing RNA circularization approaches.

Circular RNA encoding chimeric antigen receptors targeting BCMA

Growing community of inventors

Cambridge, MA, United States of America

Amy M Becker