US 11,807,849 B2
Gene therapies for lysosomal disorders
Asa Abeliovich, New York, NY (US); Laura Heckman, New York, NY (US); and Herve Rhinn, New York, NY (US)
Assigned to Prevail Therapeutics, Inc., New York, NY (US)
Appl. No. 16/753,018
Filed by Prevail Therapeutics, Inc., New York, NY (US)
PCT Filed Oct. 3, 2018, PCT No. PCT/US2018/054223
§ 371(c)(1), (2) Date Apr. 2, 2020,
PCT Pub. No. WO2019/070891, PCT Pub. Date Apr. 11, 2019.
Claims priority of provisional application 62/567,303, filed on Oct. 3, 2017.
Claims priority of provisional application 62/567,305, filed on Oct. 3, 2017.
Prior Publication US 2020/0231970 A1, Jul. 23, 2020
Int. Cl. C12N 15/113 (2010.01); A61P 25/28 (2006.01); A61K 9/00 (2006.01); C07K 14/005 (2006.01); C12N 7/00 (2006.01); C12N 9/24 (2006.01); C12N 15/86 (2006.01); A61P 25/02 (2006.01); A61P 25/16 (2006.01); A61K 48/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 48/0075 (2013.01); A61P 25/02 (2018.01); A61P 25/16 (2018.01); A61P 25/28 (2018.01); C07K 14/005 (2013.01); C12N 7/00 (2013.01); C12N 9/2402 (2013.01); C12N 15/86 (2013.01); C12Y 302/01045 (2013.01); A61K 48/00 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2710/14043 (2013.01); C12N 2750/14133 (2013.01); C12N 2750/14143 (2013.01); C12N 2770/32022 (2013.01)] 41 Claims
 
1. An isolated nucleic acid comprising
(i) an expression construct comprising a transgene encoding a single gene product, wherein the gene product is an inhibitory nucleic acid targeting α-Synuclein, wherein the inhibitory nucleic acid is encoded by the sequence set forth in SEQ ID NO: 3; and
(ii) two adeno-associated virus (AAV) inverted terminal repeats (ITR) sequences flanking the expression construct, wherein the first ITR sequence is a 5′ ITR, and the second ITR sequence is a 3′ ITR.