	US 12,146,150 B2
	Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
Dinah Wen-Yee Sah, Hopkinton, MA (US); Martin Goulet, Weston, MA (US); Holger Patzke, Belmont, MA (US); Yanqun Shu, Winchester, MA (US); Jinzhao Hou, Lexington, MA (US); and Hélène Puccio, Illkirch (FR)
Assigned to VOYAGER THERAPEUTICS, INC., Cambridge, MA (US); INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), Paris (FR); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), Paris (FR); and UNIVERSITÉ DE STRASBOURG, Strasbourg (FR)
Filed by VOYAGER THERAPEUTICS, INC., Cambridge, MA (US); INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE), Paris (FR); CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), Paris (FR); and UNIVERSITÉ DE STRASBOURG, Strasbourg (FR)
Filed on Sep. 13, 2022, as Appl. No. 17/931,725.
Application 17/931,725 is a continuation of application No. 16/651,617, abandoned, previously published as PCT/US2018/053312, filed on Sep. 28, 2018.
Claims priority of provisional application 62/663,835, filed on Apr. 27, 2018.
Claims priority of provisional application 62/565,840, filed on Sep. 29, 2017.
Prior Publication US 2023/0044444 A1, Feb. 9, 2023
Int. Cl. C12N 15/86 (2006.01); A61K 48/00 (2006.01); A61P 25/00 (2006.01); C07K 14/47 (2006.01); C12N 7/00 (2006.01)

CPC C12N 15/86 (2013.01) [A61K 48/005 (2013.01); A61P 25/00 (2018.01); C07K 14/47 (2013.01); C12N 7/00 (2013.01); C12N 2750/14123 (2013.01); C12N 2750/14143 (2013.01)]

20 Claims

1. A method of delivering a payload to a tissue of the central nervous system (CNS) in a mammalian subject, wherein the method comprises: administering a first adeno-associated virus (AAV) particle to the subject by intravenous (IV) administration at a dose of at least 2.00×10¹³vg/kg; wherein the first AAV particle comprises:

(i) an AAV capsid comprising the amino acid sequence of SEQ ID NO: 2; and

(ii) a viral genome comprising at least one inverted terminal repeat (ITR) and a polynucleotide sequence encoding the payload.