Compositions for the treatment of disease
AssigneeVoyager Therapeutics, Inc.
InventorsSteven Paul, Donna T. Ward
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Abstract
The invention provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody like polypeptides, for the prevention and/or treatment of diseases and/or disorders.
Claims70
An AAV particle comprising a capsid and a viral genome, said viral genome comprising at least one inverted terminal repeat (ITR) region and a payload region, said payload region comprising a regulatory sequence operably linked to at least a first nucleic acid segment, said first nucleic acid segment encoding one or more polypeptides selected from the group consisting of SEQ ID NO:
- 2948-17938, and fragments thereof.
An AAV particle comprising a capsid and a viral genome, said viral genome comprising at least one inverted terminal repeat (ITR) region and a payload region comprising a regulatory sequence operably linked to at least a first nucleic acid segment, said first nucleic acid segment encoding a bispecific antibody derived from SEQ ID NO:
- 2948-17938 or portions or fragments thereof.
A pharmaceutical composition comprising an AAV particle of any of the preceding claims in a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.