Ascidian Therapeutics, Inc.

Rebekka Krumbach

Scott J Dooley

Dennis Wu

Adam Clemens

Jesse Gray

Shimyn Slomovic

Lingtao Peng

Robert Bell

Akiko Doi

Akiko Noma

Kirk Burkhart

Kirk Gosik

Provided herein are nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs); RNA exon editing molecules) capable of correcting mutations in the ABCA4 gene. Such molecules are useful in the treatment of disorders such as ABCA4-associated retinal dystrophies (e.g., Stargardt Disease or cone-rod dystrophy). Also described herein are methods of using the nucleic acid trans-splicing molecules described herein to correct mutations in ABCA4, thereby treating disorders associated with mutations in ABCA4 and use of the nucleic acid trans-splicing molecules described herein for treating disorders associated with mutations in ABCA4 and in the preparation of medicaments for the treatment of disorders associated with mutations in ABCA4.

ABCA4 trans-splicing molecules

Growing community of inventors

Boston, MA, United States of America

Rebekka Krumbach