Abstract: Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) ?-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).

Abstract: Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

Abstract: The present disclosure relates to using size exclusion chromatography to isolate AAV genome, to determine the vector genome size purity of a composition comprising isolated rAAV particles, to assess the folding or secondary structure of vector genomes inside the capsids, and to determine vector genome titer (Vg) of a composition comprising isolated rAAV particles.

Abstract: Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) ?-L-iduronidase (ID-UA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).

Abstract: The present disclosure relates to using spectrophotometry to estimate genome copies and full/empty ratios adeno-associated virus particles.

Abstract: Compositions and methods are described for the delivery of a fully human post-translationally modified (HuPTM) therapeutic VEGF-Trap (VEGF-TrapHuPTM)—to a human subject diagnosed with an ocular disease or condition or cancer associated with neovascularization and indicated for treatment with the therapeutic mAb. Delivery may be advantageously accomplished via gene therapy—e.g., by administering a viral vector or other DNA expression construct encoding the VEGF-TrapHuPTM to a patient (human subject) diagnosed with an ocular condition or cancer indicated for treatment with the VEGF-Trap—to create a permanent depot in a tissue or organ of the patient that continuously supplies the VEGF-TrapHuPTM, i.e., a human-glycosylated transgene product. Alternatively, the VEGF-TrapHuPTM, for example, produced in cultured human cell culture, can be administered to the patient for treatment of the ocular disease or cancer.

Abstract: Compositions and methods are described for the delivery of a fully human post-translationally modified (HuPTM) monoclonal antibody (“mAb”) or the antigen-binding fragment of a mAb against human vascular endothelial growth factor (“hVEGF”)—such as, e.g., a fully human-glycosylated (HuGly) anti-hVEGF antigen-binding fragment—to the retina/vitreal humour in the eye(s) of human subjects diagnosed with ocular diseases caused by increased neovascularization, for example, neovascular age-related macular degeneration (“nAMD”), also known as “wet” age-related macular degeneration (“WAMD”), age-related macular degeneration (“AMD”), and diabetic retinopathy.

Abstract: Compositions and methods are described for the delivery of recombinant human iduronate-2-sulfatase (IDS) produced by human neuronal or glial cells to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis II (MPS II).

Abstract: Provided are methods and compositions for the delivery of fully human post-translationally modified therapeutic monoclonal antibodies and antigen-binding fragments thereof. The fully human post-translationally modified therapeutic monoclonal antibodies may be preferably delivered by gene therapy methods, particularly as a recombinant adeno-associated virus (rAAV) vector to the appropriate tissue. Methods of manufacture of the AAV vectors, pharmaceutical compositions and methods of treatment are also provided. In addition, provided are methods of producing therapeutic antibodies that are “biobetters” as fully human post-translationally modified. These fully human post-translationally modified therapeutic antibodies may be administered to a subject in need of treatment with the therapeutic antibody.

Abstract: Compositions and methods are described for the delivery of a fully human-glycosylated (HuGly) ?-L-iduronidase (IDUA) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject diagnosed with mucopolysaccharidosis I (MPS I).