	US 11,925,675 B2
	Systemic delivery of adeno-associated virus vector expressing gamma-sarcoglycan and the treatment of muscular dystrophy
Louise Rodino-Klapac, Cambridge, MA (US); and Eric Pozsgai, Cambridge, MA (US)
Assigned to Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed by Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed on Sep. 7, 2021, as Appl. No. 17/468,086.
Claims priority of provisional application 63/075,697, filed on Sep. 8, 2020.
Prior Publication US 2022/0088123 A1, Mar. 24, 2022
Int. Cl. A61P 21/00 (2006.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); C07K 14/705 (2006.01); C12N 15/86 (2006.01)

CPC A61K 38/177 (2013.01) [A61K 48/0058 (2013.01); A61K 48/0066 (2013.01); A61P 21/00 (2018.01); C07K 14/705 (2013.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01); C12N 2750/14171 (2013.01)]

14 Claims

1. A method of treating muscular dystrophy in a subject in need thereof, comprising the step of administering a recombinant adeno-associated virus (rAAV) scAAVrh74.MHCK7.hSGCG to the subject, wherein the rAAV is administered using a systemic route of administration and at a dose of about 4.63×10¹²vg/kg, about 1.85×10¹³vg/kg, or about 7.41×10¹³vg/kg based on a linearized plasmid as the quantitation standard, and wherein the rAAV comprises a nucleotide sequence that is identical to SEQ ID NO: 7 or SEQ ID NO: 10.