Abstract: The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof). In certain embodiments, the production process uses Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAV particles.

Abstract: The present disclosure relates to pharmaceutical compositions of adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).

Abstract: The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and Baculoviral Infected Insect Cells (BIICs) in the production of AAV particles.

Abstract: The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles and AAV formulations, including recombinant adeno-associated virus (rAAV) particles and formulations. In certain embodiments, the present disclosure presents methods and systems for clarifying, purifying, formulating, filtering and processing AAV particles and AAV formulations. The present disclosure also describes compositions, methods and processes for the design, preparation, manufacture, use and/or formulation of AAV particles comprising modulatory polynucleotides, e.g., polynucleotides encoding small interfering RNA (siRNA) molecules which target the Huntingtin (HTT) gene (e.g., the wild-type or the mutated CAG-expanded HTT gene). Methods for using formulated AAV particles comprising modulatory polynucleotides to inhibit the HTT gene expression in a subject with a neurodegenerative disease (e.g., Huntington's Disease (HD)) are also disclosed.