US 11,655,460 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)
Filed by Prevail Therapeutics, Inc., New York, NY (US)
Filed on Jun. 18, 2020, as Appl. No. 16/904,909.
Application 16/904,909 is a continuation of application No. 16/690,320, filed on Nov. 21, 2019, granted, now 10,689,625.
Application 16/690,320 is a continuation of application No. PCT/US2018/054227, filed on Oct. 3, 2018.
Claims priority of provisional application 62/567,311, filed on Oct. 3, 2017.
Claims priority of provisional application 62/567,310, filed on Oct. 3, 2017.
Claims priority of provisional application 62/567,301, filed on Oct. 3, 2017.
Claims priority of provisional application 62/567,319, filed on Oct. 3, 2017.
Claims priority of provisional application 62/567,296, filed on Oct. 3, 2017.
Prior Publication US 2020/0308554 A1, Oct. 1, 2020
Int. Cl. C12N 15/85 (2006.01); C07H 21/04 (2006.01); C12N 7/00 (2006.01); C07K 14/47 (2006.01); A61P 25/28 (2006.01); A61K 9/00 (2006.01); A61K 35/76 (2015.01); A61K 48/00 (2006.01); C07K 14/005 (2006.01); C07K 14/61 (2006.01); C07K 14/705 (2006.01); A61P 25/16 (2006.01); C07K 14/435 (2006.01); A61K 35/761 (2015.01); C12N 15/861 (2006.01); C12N 15/86 (2006.01); A61K 39/23 (2006.01); C12N 15/864 (2006.01)
CPC C12N 7/00 (2013.01) [A61K 9/0019 (2013.01); A61K 9/0085 (2013.01); A61K 35/76 (2013.01); A61K 35/761 (2013.01); A61K 48/0058 (2013.01); A61K 48/0075 (2013.01); A61P 25/16 (2018.01); A61P 25/28 (2018.01); C07K 14/005 (2013.01); C07K 14/435 (2013.01); C07K 14/47 (2013.01); C07K 14/61 (2013.01); C07K 14/70503 (2013.01); C12N 15/861 (2013.01); A61K 39/23 (2013.01); C07H 21/04 (2013.01); C12N 15/86 (2013.01); C12N 15/8645 (2013.01); C12N 2750/14022 (2013.01); C12N 2750/14043 (2013.01); C12N 2750/14121 (2013.01); C12N 2750/14122 (2013.01); C12N 2750/14143 (2013.01); C12Y 302/01045 (2013.01)] 14 Claims
 
1. A Baculovirus vector comprising a nucleic acid comprising an expression construct comprising a promoter operably linked to a transgene insert encoding a Progranulin (PGRN) protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO: 68.