The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Sep. 09, 2025
Filed:
Nov. 22, 2023
Ascidian Therapeutics, Inc., Boston, MA (US);
Rebekka Krumbach, Boston, MA (US);
Scott Dooley, Boston, MA (US);
Akiko Doi, Boston, MA (US);
Kirk Burkhart, Boston, MA (US);
Jesse Gray, Boston, MA (US);
Lingtao Peng, Boston, MA (US);
Dennis Wu, Boston, MA (US);
Akiko Noma, Boston, MA (US);
Kirk Gosik, Boston, MA (US);
Shimyn Slomovic, Boston, MA (US);
Adam Clemens, Boston, MA (US);
Robert Bell, Boston, MA (US);
Ascidian Therapeutics, Inc., Boston, MA (US);
Abstract
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.