Patent classifications
C12N7/02
Chromatography media and method
Adsorptive media for chromatography, particularly ion-exchange chromatography, derived from a shaped fiber. In certain embodiments, the functionalized shaped fiber presents a fibrillated or ridged structure which greatly increases the surface area of the fibers when compared to ordinary fibers. Also disclosed herein is a method to add surface pendant functional groups that provides cation-exchange or anion-exchange functionality to the high surface area fibers. This pendant functionality is useful for the ion-exchange chromatographic purification of biomolecules, such as monoclonal antibodies (mAbs).
HIGH EFFICIENCY PURIFICATION OF DIVERGENT AAV SEROTYPES USING AAVX AFFINITY CHROMATOGRAPHY
Disclosed herein are optimized methods of high efficiency purification of adeno-associated virus (AAV) particles, comprising a step of binding one or more AAV particles with a volume of chromatography resin medium comprising at least one ligand possessing a pan-AAV affinity. The optimized methods are capable of providing purified AAV particles comprising a wide diversity of AAV serotypes without the need to further optimize for a given AAV serotype.
HIGH EFFICIENCY PURIFICATION OF DIVERGENT AAV SEROTYPES USING AAVX AFFINITY CHROMATOGRAPHY
Disclosed herein are optimized methods of high efficiency purification of adeno-associated virus (AAV) particles, comprising a step of binding one or more AAV particles with a volume of chromatography resin medium comprising at least one ligand possessing a pan-AAV affinity. The optimized methods are capable of providing purified AAV particles comprising a wide diversity of AAV serotypes without the need to further optimize for a given AAV serotype.
HIV-1 GP140 immunogens comprising modified NHR1 regions that stabilize pre-fusion envelope conformations
The present invention provides HIV-1 vaccine immunogens. Some of the immunogens contain a soluble gp140-derived protein that harbors a modified N-terminus of the HR1 region in gp41. Some of the immunogens contain an HIV-1 Env-derived trimer protein that is presented on a nanoparticle platform. The invention also provides methods of using the HIV-1 vaccine immunogens for eliciting an immune response or treating HIV infections.
SYSTEMS AND METHODS FOR PRODUCING GENE THERAPY FORMULATIONS
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.
SYSTEMS AND METHODS FOR PRODUCING GENE THERAPY FORMULATIONS
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.
Production of viruses in cell culture
The present invention relates to methods of replicating viruses in vitro. In particular, the invention relates to a genetically modified population of cells, and/or a population of cells treated with an exogenous compound, wherein the cells are capable of producing more virus than cells lacking the genetic modification and/or lacking treatment with the exogenous compound. The invention also relates to methods of producing populations of such cells, as well as the use of the viruses obtained to prepare vaccine compositions.
Production of viruses in cell culture
The present invention relates to methods of replicating viruses in vitro. In particular, the invention relates to a genetically modified population of cells, and/or a population of cells treated with an exogenous compound, wherein the cells are capable of producing more virus than cells lacking the genetic modification and/or lacking treatment with the exogenous compound. The invention also relates to methods of producing populations of such cells, as well as the use of the viruses obtained to prepare vaccine compositions.
PROTEIN-BASED PURIFICATION MATRICES AND METHODS OF USING THE SAME
Provided herein are protein-based purification matrices and methods of use thereof to purify biologics and/or to remove contaminants from a composition. Methods of bringing two or more biologics in close proximity are also provided. The disclosed compositions and methods allow for faster, more efficient purification of a biologic compared to traditional affinity chromatography.
Zika virus vaccine
Described herein are Zika virus vaccines and compositions and methods of producing and administering said vaccines to subjects in need thereof.