Invention Grant
- Patent Title: Treatment of primary ciliary dyskinesia with synthetic messenger RNA
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Application No.: US16941028Application Date: 2020-07-28
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Publication No.: US11642421B2Publication Date: 2023-05-09
- Inventor: David J. Lockhart , Brandon Wustman , Mirko Hennig , Daniella Ishimaru
- Applicant: TranscripTx, Inc.
- Applicant Address: US CA Sunnyvale
- Assignee: TranscripTx, Inc.
- Current Assignee: TranscripTx, Inc.
- Current Assignee Address: US CA Sunnyvale
- Agency: Wilson Sonsini Goodrich & Rosati
- Main IPC: A61K48/00
- IPC: A61K48/00 ; A61K9/127 ; A61K31/7105 ; A61K31/7115 ; A61P11/06 ; C12N15/113 ; A61K47/69 ; A61P11/00

Abstract:
Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with cilia maintenance and function, impaired function of the axoneme, such as DNAI1 or DNAH5.
Public/Granted literature
- US20210162068A1 TREATMENT OF PRIMARY CILIARY DYSKINESIA WITH SYNTHETIC MESSENGER RNA Public/Granted day:2021-06-03
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