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US 12,516,298 B2Granted
Gene therapies for lysosomal disorders
AssigneePrevail Therapeutics, Inc.
InventorsAsa Abeliovich, Laura Heckman, Herve Rhinn, Li Chin Wong, Hsuan-Ni Lin, Franz Hefti
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Abstract
The disclosure relates to compositions and methods for treatment of diseases associated with aberrant lysosomal function, such as fronto-temporal dementia (FTD). The disclosure also provides expression constructs comprising a transgene encoding progranulin or a portion thereof. The disclosure provides methods of treating FTD by administering such expression constructs to a subject in need thereof.
Claims9
A pharmaceutical composition comprising:
- (i) a recombinant adeno-associated virus (rAAV) comprising:
- (a) a rAAV 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;
- and
- (b) an adeno-associated virus (AAV) 9 capsid protein;
- (ii) about 20 mM Tris, pH 8.0,
- (iii) about 1 mM MgCl2,
- (iv) about 200 mM NaCl, and
- (v) about 0.001% w/v poloxamer 188.
6 more elements
A pharmaceutical composition comprising:
- (i) a recombinant adeno-associated virus (rAAV) comprising:
- (a) a rAAV vector comprising a nucleic acid comprising, in 5′ to 3′ order:
- an AAV2 inverted terminal repeat (ITR);
- a cytomegalovirus (CMV) enhancer;
- a chicken beta actin (CBA) promoter;
- a transgene insert encoding a progranulin (PGRN) protein, wherein the transgene insert comprises the nucleotide sequence of SEQ ID NO:
- 68;
- a Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (WPRE);
- a Bovine Growth Hormone polyA signal tail;
- and
- an AAV2 ITR;
- and
- (b) an adeno-associated virus (AAV) 9 capsid protein;
- (ii) about 20 mM Tris, pH 8.0,
- (iii) about 1 mM MgCl2,
- (iv) about 200 mM NaCl, and
- (v) about 0.001% w/v poloxamer 188.
14 more elements
Dependencies are inferred from the claim text.