C12N2750/14151

METHODS AND COMPOSITIONS FOR PURIFYING ADENO ASSOCIATED VIRUS PARTICLES OR ADENOVIRUSES

The present invention relates to compositions and methods for lysing cells and isolating and/or purifying adeno-associated virus particles or adenovirus particles using a detergent selected from the group of alkyldimethylamine oxides.

Methods and Compositions for Treatment of Age-Related Dysfunction
20230181687 · 2023-06-15 ·

Disclosed herein are methods and compositions for the treatment of age-related dysfunction, particularly, age-related motor impairment.

METHODS AND COMPOSITIONS FOR THE PURIFICATION OF ADENO-ASSOCIATED VIRUS

Provided herein are methods and compositions for the separation of an adeno-associated virus (AAV) particle from a mixture of the AAV and at least one contaminant using anion exchange chromatography. These methods and compositions allow for improved purification of complete AAV particles from contaminants such as AAV particles that lack a complete genome (e.g., empty capsids) and AAV degradation products.

COMPOSITIONS AND METHODS FOR SEQUENTIAL STACKING OF NUCLEIC ACID SEQUENCES INTO A GENOMIC LOCUS

The present invention encompasses compositions and methods for the sequential stacking of donor nucleic acids into a single genomic locus within a cell to allow for the introduction of relatively long nucleic sequences. This allows for insertion into the genome of a donor nucleic acid sequence that exceeds the packaging capacity of a single adeno-associated viral vector.

METHODS FOR PRODUCING PREPARATIONS OF RECOMBINANT AAV VIRIONS SUBSTANTIALLY FREE OF EMPTY CAPSIDS
20220364111 · 2022-11-17 ·

Methods for separating AAV empty capsids from mixtures of AAV vector particles and AAV empty capsids are described. The methods use column chromatography techniques and provide for commercially viable levels of recombinant AAV virions.

CONTROLLED EXPRESSION OF VIRAL PROTEINS

The present disclosure describes methods and systems for use in the production of adeno-associated virus (AAV) particles, including recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the production process and system use Spodoptera frugiperda insect cells (such as Sf9 or Sf21) as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) in the production of AAV particles. In certain embodiments, the production process and system allow for the controlled expression of AAV capsid proteins, such as VP1, VP2 and VP3.

Compositions and methods for preventing and treating coronavirus infection—SARS-CoV-2 vaccines

The invention relates to immunogenic compositions and vaccines containing a coronavirus (e.g., Wuhan coronavirus (2019-nCoV; also referred to as SARS-CoV-2)) protein or a polynucleotide encoding a coronavirus (e.g., Wuhan coronavirus (2019-nCoV; SARS-CoV-2)) protein and uses thereof. The invention also provides methods of treating and/or preventing a coronavirus (e.g., Wuhan coronavirus (2019-nCoV; SARS-CoV-2)) infection by administering an immunogenic composition or vaccine to a subject (e.g., a human). The invention also provides methods of detecting and/or monitoring a protective anti-coronavirus (e.g., Wuhan coronavirus (2019-nCoV; SARS-CoV-2)) antibody response (e.g., anti-coronavirus antibody response, e.g., anti-2019-nCoV antibody response, e.g., anti-Spike antibody response, e.g., anti-Spike neutralizing antibody response). The present invention relates to isolated nucleic and/or recombinant nucleic acid encoding a coronavirus S protein, in particular a SARS-CoV-2 S protein, and to the coronavirus S proteins, as well as to the use of the nucleic acids and/or proteins thereof in vaccines.

Compositions and methods for treating diseases

The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

A METHOD FOR SEPARATION OR DEPLETION OF EMPTY AAV CAPSIDS FROM FULL AAV CAPSIDS
20230167416 · 2023-06-01 ·

A method for the separation or depletion of empty AAV capsids from full AAV capsids in an aqueous mixture comprising empty and full AAV capsids, wherein the mixture is contacted with a primary amino groups bearing solid phase surface in a first alkaline milieu whereby (i) full AAV capsids bind to the solid phase surface whereas empty AAV capsids at least partially do not bind to the solid phase surface,
or (ii) both full and empty AAV capsids bind to the solid phase surface, and subsequently the empty AAV capsids are at least partially eluted by means of a second alkaline milieu of a pH value higher than the pH value of the first alkaline milieu, with the proviso that the second alkaline milieu does not elute full AAV capsids from the solid phase surface.