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The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.

The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.

Patent No.:

US 10898585 B1

PDFFull Text
Date of Patent:
Jan. 26, 2021

Filed:

Apr. 12, 2018

Gene therapy for aadc deficiency

Applicant:

National Taiwan University, Taipei, TW;

Inventors:

Mark Pykett, Cambridge, MA (US);

Richard Thorn, Mendon, MA (US);

Wuh-Liang (“Paul”) Hwu, Taipei, TW;

Assignees:

PTC THERAPEUTICS .INC., South Plainfield, NJ (US);

NATIONAL TAIWAN UNIVERSTY, Taipei, TW;

Attorney:
Primary Examiner:

Marcia S Noble

Int. Cl.
CPC ...
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);
U.S. Cl.
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);
Abstract

The present invention is directed to compositions and methods for treating aromatic-amino acid decarboxylase (AADC) deficiency. This invention includes a method of treating AADC deficiency in a pediatric subject, comprising the steps of: (a) providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, (b) stereotactically delivering the pharmaceutical formulation to at least one target site in the brain of the subject in a dose of an amount at least about 1.8×10vg; wherein delivering the pharmaceutical formulation to the brain is optionally by frameless stereotaxy, and optionally wherein the dose is an amount of at least about 2.4×10vg and in some embodiments wherein the pharmaceutical formulation comprises a rAAV2-hAADC vector concentration of about 5.7×10vg/mL. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally further comprises the step of administering a therapeutically effective dose of dopamine-antagonist to the subject such as risperidone. This invention is also directed to methods for treating aromatic-amino acid decarboxylase (AADC) deficiency, wherein the method optionally comprises providing a pharmaceutical formulation comprising an rAAV2-hAADC vector, and empty capsids.

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