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US 2026/0232743 A1Published application
Adeno-Associated Virus Vector Formulations and Methods
AssigneeREGENXBIO Inc.
InventorsTristan James Marshall, Jared Bee, Yu Zhang, Roberto DePaz
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Abstract
Provided herein are formulations comprising recombinant AAV particles. In some embodiments, the formulation is a frozen formulation or a lyophilized formulation. Also provided herein are methods for reducing rAAV genome release from rAAV particles.
Claims21
-134. (canceled)
A stable formulation comprising a recombinant adeno-associated virus (rAAV) particle and
- a) between about 1 mM and about 50 mM Tris,
- b) between about 100 mM and about 400 mM of a sugar, and
- c) an amorphous salt having an ionic strength greater than or equal to 30 mM, wherein the amorphous salt is a citrate or a sulfate salt, and
- d) between about 0.0005% and about 0.01% of a nonionic surfactant,
- wherein the formulation is suitable for lyophilization;
- and
- wherein the rAAV particle comprises an AAV9 capsid protein.
4 more elements
A stable formulation comprising a recombinant adeno-associated virus (rAAV) particle and
- a) between about 1 mM and about 50 mM Tris,
- b) between about 100 mM and about 400 mM of a sugar, and
- c) an amorphous salt having an ionic strength greater than or equal to 60 mM, wherein the amorphous salt is a citrate or a sulfate salt, and
- d) between about 0.0005% and about 0.01% of a nonionic surfactant,
- wherein the formulation is suitable for lyophilization;
- and
- wherein the rAAV particle comprises an AAV8 capsid protein.
4 more elements
Dependencies are inferred from the claim text.