The present disclosure presents methods for producing baculovirus infected insect cells (BIICs). The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions and formulations, including recombinant adeno-associated viruses (rAAV). In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs. In certain embodiments, the present disclosure presents methods and systems for designing, producing, clarifying, purifying, formulating, filtering and processing rAAVs and rAAV formulations. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs).

SYSTEMS AND METHODS FOR PRODUCING BACULOVIRAL INFECTED INSECT CELLS (BIICs) IN BIOREACTORS

The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, compositions and formulations, including recombinant adeno-associated viruses (rAAV). The present disclosure presents cell culture mediums for use in producing adeno-associated viruses (AAV), such as AAV which comprise a polynucleotide encoding a payload. In certain embodiments, the cell culture medium comprises a hydrolysate mixture, L-glutamine, poloxamer 188 (e.g. 10% pluronic F- 68), a lipid emulsion, and a cholesterol mixture. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells (VPCs). In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAVs.

Cell culture medium for use in producing gene therapy products in bioreactors

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

Compositions and methods for treating huntington's disease

Methods for screening for an adeno-associated virus (AAV) capsid protein that can bind to a target protein (e.g., Ly6 protein) and related compositions are provided in aspects of the disclosure.

Systems for evolved adeno-associated viruses (aavs) for targeted delivery

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 eurodegenerative disease (e.g., Huntington's Disease (HD)) are also disclosed.

Systems and methods for producing gene therapy formulations

The disclosure relates to compositions, methods, and processes for the preparation, use, and/or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.

Aav variants with enhanced tropism

The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.

Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord

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Systems and methods for producing gene therapy formulations

Aav variants with enhanced tropism

Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord