Granted Patent
B2
US 12,611,436 · App. 17/072,637 · Granted Apr 28, 2026
AAV transfer cassette
Assignee:
Sarepta Therapeutics, Inc.
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Abstract
Described herein are AAV transfer cassettes and plasmids used in the production of recombinant adeno-associated viral (rAAV) vectors. The disclosed cassettes and plasmids comprise one or more transgenes having therapeutic efficacy in the amelioration, treatment and/or prevention of one or more diseases or disorders, such as Niemann-Pick Disease, type C1 (NPC1).
Claims (12)
1 . A method for treating a subject having Neimann-Pick Disease Type C1 (NPCI) in need thereof, comprising:
systemically administering to the subject a recombinant AAV vector comprising a protein capsid and a nucleic acid encapsidated by the protein capsid,
wherein the nucleic acid comprises a transfer cassette comprising the sequence of SEQ ID NO: 14 and the protein capsid comprises a capsid protein subunit from an AAV9 serotype,
and
wherein treatment reduces cholesterol accumulation in cells of the subject transduced by the recombinant AAV vector.
2 . The method of claim 1 , wherein the subject is a human subject.
3 . A method for treating a subject having Neimann-Pick Disease Type C1 (NPCI) in need thereof, comprising:
systemically administering to the subject a recombinant AAV vector comprising a protein capsid and a nucleic acid encapsidated by the protein capsid,
wherein the nucleic acid comprises a transfer cassette comprising the sequence of any one of SEQ ID NOS: 15-19 and the protein capsid comprises a capsid protein subunit from an AAV9 serotype,
and
wherein treatment reduces cholesterol accumulation in cells of the subject transduced by the recombinant AAV vector.
4 . The method of claim 3 , wherein the subject is a human subject.
Assignments (3)
SECURITY INTEREST
Recorded May 7, 2025
From: SAREPTA THERAPEUTICS, INC.
To: JPMORGAN CHASE BANK, N.A. AS ADMINISTRATIVE AGENT
Reel/Frame 071218/0445 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Feb 7, 2023
From: STRIDEBIO, INC.
To: SAREPTA THERAPEUTICS, INC.
Reel/Frame 062608/0243 →
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded May 5, 2022
From: DISMUKE, DAVID
To: STRIDEBIO, INC.
Reel/Frame 059832/0110 →
Continuity (3)
Provisional Application
62923253
· Oct 18, 2019
Provisional Application
62916749
· Oct 17, 2019
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· 2013
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