Patent classifications
C12N2750/14151
Recombinant adeno-associated virus particle purification with multiple-step anion exchange chromatography
The invention describes a method for obtaining purified recombinant Adeno-Associated Virus particles (rAAV), comprising performing a depth filtration of a starting material previously obtained from cells producing rAAV particles comprising a cell lysate and/or a culture supernatant, followed by a first step of anion-exchange chromatography, a second step of anion-exchange chromatography and a final step of tangential flow filtration, whereby purified recombinant Adeno-Associated Virus particles (rAAV) are provided.
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.
ANION EXCHANGE CHROMATOGRAPHY FOR RECOMBINANT AAV PRODUCTION
Provided herein are methods for the purification of recombinant Adeno-Associated Virus (rAAV) particles using anion exchange chromatography.
AAV PRODUCTION USING SUSPENSION ADAPTED CELLS
Provided herein are methods of improving rAAV production in cells, the method comprising increasing the salt concentration in the media in which the cells are infected or transfected, cultured, or in which they produce AAV. Aspects of the disclosure relate to improved methods of rAAV production by co-infecting suspension adapted cells (e.g., suspension adapted HEK293 cells) with viruses that encode one or more AAV components for producing rAAV particles within the suspension adapted cells.
PRODUCTION OF LARGE-SIZED QUASIDYSTROPHINS USING OVERLAPPING AAV VECTORS
The present invention concerns a quasidystrophin (QD) having the structure CH1CH2H1R1R2R3H2R8R9 in its N-terminal part and advantageously further comprising the R16 and R17 rod domains, as well as the dual AAV vector system which allows producing it.
COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME
Provided is a recombinant adeno-associated virus (rAAV) having an AAV capsid and a vector genome which comprises a nucleic acid sequence encoding a functional human methyl-CpG-binding protein 2 (hMECP2). Also provided is a production system useful for producing the rAAV, a pharmaceutical composition comprising the rAAV, and a method of treating a subject having Rett Syndrome, or ameliorating symptoms of Rett Syndrome, or delaying progression of Rett Syndrome via administrating an effective amount of the rAAV to a subject in need thereof.
NOVEL AAV CAPSIDS AND COMPOSITIONS CONTAINING SAME
Provided herein are novel AAV capsids and rAAV comprising the same. In one embodiment, vectors employing a novel AAV capsid show increased transduction of a selected target tissue as compared to a prior art AAV.
CLOSED-ENDED LINEAR DUPLEX DNA FOR NON-VIRAL GENE TRANSFER
Aspects of the disclosure relate to a nucleic acid comprising a heterologous nucleic acid insert flanked by interrupted self-complementary sequences, wherein one self-complementary sequence is interrupted by a cross-arm sequence forming two opposing, lengthwise-symmetric stem-loops, and wherein the other of the self-complementary sequences is interrupted by a truncated cross-arm sequence. Methods of delivering the nucleic acid to a cell are also provided.
Virus purification
A spiral tube countercurrent chromatography rotor for separating virus in a two part aqueous solvent is described.
METHODS FOR THE MANUFACTURE OF RECOMBINANT VIRAL VECTORS
The present invention relates to methods for the production of high titer recombinant viral vectors, more particularly recombinant AAV vectors, so that the methods can be effectively employed on a scale that is suitable for the practical application of gene therapy techniques.