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US 2025/0018059 A1Published application
Production of recombinant aav vectors for treating muscular dystrophy
AssigneeSarepta Therapeutics, Inc.
InventorsMaroof ALAM, Louise RODINO-KLAPAC
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Abstract
The present disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV), produced in mammalian adherent cells cultured in suspension conditions, for expressing a human micro-dystrophin gene. The present disclosure also provides compositions and methods of using these rAAV to treat muscular dystrophy, such as, e.g., Duchenne Muscular Dystrophy.
Claims48
A method of producing a recombinant adeno-associated virus (rAAV) rAAVrh74.MHCK7.microdystrophin in adherent mammalian cells by a suspension seed process, comprising:
- (a) culturing cells with a first growth medium comprising serum in a N-2 container;
- (b) removing the cells from the first medium;
- (c) inoculating the cells from step (b) into a second medium comprising no serum or serum at a concentration less than the first medium in a N-1 container;
- (d) culturing the cells in the N-1 container under suspension conditions;
- and
- (e) inoculating a third medium in a bioreactor with the cells from step (d).
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Dependencies are inferred from the claim text.