Aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)
AssigneeSwanBio Therapeutics Limited
InventorsSean Clark, Karen Kozarksy, Tugba Guven-Ozkan, Anna Tretiakova
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Abstract
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.
Claims31
-86. (canceled)
A recombinant adeno-associated virus (rAAV) vector genome comprising a promoter operably linked to a polynucleotide encoding a human ATP-binding cassette, sub-family D, member 1 (ABCD1) polypeptide, wherein the rAAV vector genome does not comprise a woodchuck post-transcriptional regulatory element (WPRE).
A recombinant AAV (rAAV) vector comprising:
- an AAV9 capsid;
- and
- an rAAV vector genome comprising, 5′ to 3′, a truncated 5′ AAV2 ITR, a CMV enhancer, a chicken β actin promoter, a β-actin exon, a chimeric intron, and a rabbit β-globin exon, a polynucleotide encoding a human ATP-binding cassette, sub-family D, member 1 (ABCD1) polypeptide, a SV40 polyA signal, a bovine growth hormone polyA signal, and a truncated 3′ AAV2 ITR, and wherein the rAAV vector genome does not comprise a woodchuck post-transcriptional regulatory element (WPRE).
A polynucleotide comprising a sequence having at least 95% identity to the sequence of SEQ ID NO:
- 11050.
A polynucleotide comprising a sequence having at least 95% identity to the sequence of SEQ ID NO:
- 11060.
Dependencies are inferred from the claim text.