SAREPTA THERAPEUTICS, INC.

A package for a dose includes an outer box including a housing including a bottom panel and one or more side wall defining an open segment in the perimeter of the bottom panel. A flap is coupled to the bottom panel and encloses the one or more sidewalls when the outer box is in a closed position. A lid is coupled to the housing. The packaging includes least one tray assembly disposed within the housing including a tray defining an interior region, a support partitioning the interior region into a plurality of slots, and a cover sheet. The cover sheet includes a main sheet overlaying the support and a plurality of openings aligned with at least a subset of the plurality of slots extending through the main sheet to allow one or more slots of the subset to receive a container.

Methods for treating muscular dystrophy by administering a pharmaceutical composition comprising about 80 to about 300 mg/kg of an antisense oligomer, or pharmaceutically acceptable salt thereof, are described. In one embodiment, the compositions are administered less frequently than occurs in existing methods of treatment

Disclosed herein are transgenic non-human animal models of Pompe disease, methods of making, and methods of using the same. Disclosed herein are also nucleic acid molecules useful for making the non-human animal models of Pompe disease.

Provided are methods to characterize the VP1, VP2 and VPS capsid proteins in an adeno-associated virus (AAV) particle using liquid chromatography mass spectrometry, and/or ultraviolet (UV)-visible spectroscopy. The methods generally include the steps of (a) subjecting an AAV particle to liquid chromatography to denature and then separate the VP1, VP2 and VPS capsid proteins, and (b) subjecting the separated VP1, VP2 and VPS capsid proteins produced in step (a) to UV and mass spectrometry to determine the ratio and masses of the VP1, VP2 and VPS capsid proteins in the AAV particle. In another aspect, the disclosure provides an AAV composition comprising a post-translation modification. The disclosure also provides methods for characterizing the purity of AAV compositions using liquid chromatography mass spectrometry.

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.

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).

The present disclosure provides gene therapy vectors, such as recombinant adeno-associated virus (rAAV) 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. The present disclosure also provides genotyping a subject's DMD gene to determine if rAAV gene therapy should be contraindicated.

Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomersynthesis while maintaining overall yield and purity of a synthesized oligomer.

The disclosure is directed to a method for seed train expansion of adherent cells comprising culturing cells with a serum-supplemented growth medium in a N-2 vessel; removing the cells from the serum-supplemented medium; inoculating the cells from step into a serum-free growth medium in a N-1 vessel; culturing the cells in the N-1 vessel under suspension conditions; and inoculating a growth medium in a bioreactor with the suspension-cultured cells. In some aspects, the adherent cells are not suspension-adapted. In some aspects, the adherent cells are suspension-adapted. In some aspects, the adherent cells produced by the seed train expansion method are used to produce viral vectors. In some aspects, the viral vectors are AAV vectors.