	US 11,865,188 B2
	Gene therapy for AADC deficiency
Mark Pykett, Cambridge, MA (US); Richard Thorn, Mendon, MA (US); and Wuh-Liang (“Paul”) Hwu, Taipei (TW)
Assigned to National Taiwan University, Taipei (TW)
Filed by National Taiwan University, Taipei (TW)
Filed on Jan. 25, 2021, as Appl. No. 17/157,745.
Application 17/157,745 is a continuation of application No. 15/951,270, filed on Apr. 12, 2018, granted, now 10,898,585.
Claims priority of provisional application 62/485,658, filed on Apr. 14, 2017.
Prior Publication US 2021/0236653 A1, Aug. 5, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 48/00 (2006.01); A61P 25/00 (2006.01); A61K 9/51 (2006.01); A61K 31/5513 (2006.01); C12N 15/62 (2006.01); C12N 15/85 (2006.01); C12N 15/113 (2010.01); A61K 31/4515 (2006.01); C12N 15/86 (2006.01); C12N 9/88 (2006.01); C12N 15/864 (2006.01)

CPC A61K 48/005 (2013.01) [A61K 9/5184 (2013.01); A61K 31/4515 (2013.01); A61K 31/5513 (2013.01); A61K 48/00 (2013.01); A61K 48/0075 (2013.01); A61K 48/0083 (2013.01); A61P 25/00 (2018.01); C12N 9/88 (2013.01); C12N 15/113 (2013.01); C12N 15/625 (2013.01); C12N 15/8509 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); C12Y 401/01028 (2013.01)]

15 Claims

1. A method of treating aromatic L-amino acid decarboxylase (AADC) deficiency in a pediatric subject, the method comprising:

(a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector comprising (i) a wild type AAV2 capsid, and (ii) a recombinant DNA dopa decarboxylase (DDC) gene insert comprising a nucleic acid sequence encoding hAADC; and

(b) delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount of at least 1.8×10¹¹vg; wherein the pharmaceutical formulation further comprises empty AAV2 capsids at a percentage from about 50% cp/cp up to about 90% cp/cp, wherein the pharmaceutical formulation is delivered by stereotaxy.