发明公开
- 专利标题: POLYNUCLEOTIDE COMPOSITIONS, RELATED FORMULATIONS, AND METHODS OF USE THEREOF
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申请号: US18596141申请日: 2024-03-05
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公开(公告)号: US20240299310A1公开(公告)日: 2024-09-12
- 发明人: Vladimir Kharitonov , Brandon Wustman , Jackson Eby , Rumpa Bhattacharjee , Daniella Ishimaru , Mirko Hennig
- 申请人: ReCode Therapeutics, Inc.
- 申请人地址: US CA Menlo Park
- 专利权人: ReCode Therapeutics, Inc.
- 当前专利权人: ReCode Therapeutics, Inc.
- 当前专利权人地址: US CA Menlo Park
- 主分类号: A61K9/51
- IPC分类号: A61K9/51 ; A61K9/00 ; A61K38/17 ; A61K48/00 ; A61P11/00
摘要:
Compositions of polynucleotide(s) are disclosed. A polynucleotide may encode for a polypeptide, protein, or functional fragment thereof associated with primary ciliary dyskinesia (PCD), such as dynein axonemal intermediate chain 1 (DNAI1). Pharmaceutical compositions, kits, and methods for treating a disease or condition associated with cilia maintenance and function, and impaired function of the axoneme are also disclosed. The polynucleotide may be assembled with a lipid composition for delivery to an organ, such as the lung, of a subject. The lipid composition may comprise an ionizable cationic lipid. The polynucleotide can be expressed within cells of the organ of the subject.
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