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:
Jul. 29, 2025
Filed:
Dec. 06, 2022
Translate Bio, Inc., Waltham, MA (US);
Anusha Dias, Cambridge, MA (US);
Darshan Parekh, Cambridge, MA (US);
Jeffrey S. Dubins, Cambridge, MA (US);
Christian Cobaugh, Cambridge, MA (US);
Shrirang Karve, Cambridge, MA (US);
Zarna Patel, Cambridge, MA (US);
Sara J. Dunaj, Cambridge, MA (US);
Frank Derosa, Cambridge, MA (US);
Michael Heartlein, Cambridge, MA (US);
TRANSLATE BIO, INC., Waltham, MA (US);
Abstract
The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal heavy chain 5 (DNAH5) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAH5 coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.