- 专利标题： AAV-ABCD1 constructs and use for treatment or prevention of adrenoleukodystrophy (ALD) and/or adrenomyeloneuropathy (AMN)
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申请号： US17557409申请日： 2021-12-21
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公开(公告)号： US11779655B2公开(公告)日： 2023-10-10
- 发明人: Sean Clark , Karen Kozarsky , Tugba Guven-Ozkan , Anna Tretiakova
- 申请人： SwanBio Therapeutics Limited
- 申请人地址： GB London
- 专利权人： SwanBio Therapeutics Limited
- 当前专利权人： SwanBio Therapeutics Limited
- 当前专利权人地址： GB London
- 代理机构： Wolf, Greenfield & Sacks, P.C.
- 主分类号： A61K48/00
- IPC分类号： A61K48/00 ; A61K9/00 ; C12N15/86
摘要：
The present disclosure generally relates to polynucleotides and AAV vectors that provide for the expression of ALD protein in target (e.g., neurons or glial) cells when administered to subjects in need thereof. The present disclosure further relates to compositions comprising such a polynucleotide or vector. These polynucleotides, vectors, and compositions may be used for the treatment and prevention of ALD or AMN in subjects in need thereof.
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