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:
Oct. 14, 2025
Filed:
May. 24, 2024
Ascidian Therapeutics, Inc., Boston, MA (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Philip R. Johnson, Bryn Mawr, PA (US);
Bruce C. Schnepp, Havertown, PA (US);
Jean Bennett, Bryn Mawr, PA (US);
Scott J. Dooley, Clementon, NJ (US);
Krishna Jawaharlal Fisher, Durham, NC (US);
Junwei Sun, Philadelphia, PA (US);
Ascidian Therapeutics, Inc., Boston, MA (US);
The Trustees of the University of Pennsylvania, Philadelphia, PA (US);
Abstract
The present invention features nucleic acid trans-splicing molecules (e.g., pre-mRNA trans-splicing molecules (RTMs)) capable of correcting one or more mutations in the ABCA4 gene or the CEP290 gene. Such molecules are useful in the treatment of disorders associated with mutations in ABCA4, such as Stargardt Disease (e.g., Stargardt Disease 1) and disorders associated with a mutation in CEP290, such as Leber congenital amourosis 10 (LCA 10). Also provided by the invention described herein are methods of using the nucleic acid trans-splicing molecules for correcting mutations in ABCA4 and CEP290 and for treating disorders associated with mutations in ABCA4 and CEP290, such as Stargardt Disease and LCA 10.