	US 11,779,655 B2
	AAV-ABCD1 constructs and use for treatment or prevention of adrenoleukodystrophy (ALD) and/or adrenomyeloneuropathy (AMN)
Sean Clark, Princeton, NJ (US); Karen Kozarsky, Bala Cynwyd, PA (US); Tugba Guven-Ozkan, Plymouth Meeting, PA (US); and Anna Tretiakova, Philadelphia, PA (US)
Assigned to SwanBio Therapeutics Limited, London (GB)
Filed by SwanBio Therapeutics Limited, London (GB)
Filed on Dec. 21, 2021, as Appl. No. 17/557,409.
Application 17/557,409 is a continuation of application No. PCT/US2020/067664, filed on Dec. 31, 2020.
Claims priority of provisional application 62/955,667, filed on Dec. 31, 2019.
Prior Publication US 2022/0175965 A1, Jun. 9, 2022 Prior Publication US 2022/0362403 A2, Nov. 17, 2022
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/005 (2013.01) [A61K 9/0019 (2013.01); C12N 15/86 (2013.01); C12N 2800/107 (2013.01)]

15 Claims

1. A recombinant adeno-associated viral (AAV) vector comprising:

(a) an AAV9 capsid; and

(b) a recombinant AAV vector genome comprising, in a 5′ to 3′ direction, a truncated 5′ AAV2 inverted terminal repeat sequence, a cytomegalovirus enhancer, a chicken beta-actin promoter, a beta-actin exon, a chimeric intron, a rabbit beta-globin exon, a human ATP-binding cassette, sub-family D, member 1 (ABCD1) 5′ untranslated region, a human ABCD1 coding sequence, a human ABCD1 3′ untranslated region, an SV40 polyadenylation signal sequence, a bovine growth hormone polyadenylation signal sequence, and a truncated 3′ AAV2 inverted terminal repeat sequence,

wherein the recombinant AAV vector genome does not comprise a woodchuck post-transcriptional regulatory element.