	US 12,070,510 B2
	Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid
Anthony Donsante, Decatur, GA (US); Karen Kozarsky, Bala Cynwyd, PA (US); Nicholas Matthew Boulis, Atlanta, GA (US); and Jonathan Patrick Riley, Williamsville, NY (US)
Assigned to Emory University, Atlanta, GA (US); and REGENXBIO. Inc., Rockville, MD (US)
Appl. No. 16/075,122
Filed by Emory University, Atlanta, GA (US); and REGENXBIO Inc., Rockville, MD (US)
PCT Filed Jan. 25, 2017, PCT No. PCT/US2017/014914 § 371(c)(1), (2) Date Aug. 2, 2018, PCT Pub. No. WO2017/136202, PCT Pub. Date Aug. 10, 2017.
Claims priority of provisional application 62/292,157, filed on Feb. 5, 2016.
Prior Publication US 2019/0038777 A1, Feb. 7, 2019
Int. Cl. A61K 48/00 (2006.01); A61K 9/00 (2006.01); A61K 45/06 (2006.01); A61P 25/00 (2006.01); A61P 37/00 (2006.01); C12N 15/86 (2006.01)

CPC A61K 48/0075 (2013.01) [A61K 9/0085 (2013.01); A61K 45/06 (2013.01); A61P 25/00 (2018.01); A61P 37/00 (2018.01); C12N 15/86 (2013.01); C12N 2750/14143 (2013.01)]

14 Claims

1. A method of treating a human subject diagnosed with a CNS disorder, comprising:

administering by injection into the cerebrospinal fluid to the human subject via an intracerebroventricular, intrathecal cisternal, or intrathecal lumbar route, an effective amount of a scAAV9 encoding a therapeutic protein at a flat dose of about 1.2×10¹⁴GC, wherein the CNS disorder is Spinal Muscular Atrophy, the therapeutic protein is SMN1, and the human subject is 3 to 12 years old,

thereby treating the CNS disorder and alleviating a symptom of the CNS disorder in the human subject.