C12N2750/14151

A PROCESS FOR PRODUCING A PLURALITY OF SUMOYLATION TARGET-SITE MODIFIED AAV VECTOR AND PRODUCT THEREOF
20230061069 · 2023-03-02 ·

The present invention provides for a process for producing a plurality of SUMOylation target-site modified AAV vectors, SUMOylation target-site modified AAV vectors, and application of the SUMOylation target-site modified AAV vectors in gene therapy. The present invention provides for manipulation of SUMOylation specific amino acids on AAV2 capsid protein, thereby regulating role of the same. Furthermore, the development of SUMOylation target modified AAV2 vectors presents an exciting opportunity for hepatic or ocular gene transfer with the safest AAV vector in human gene therapy applications. The plurality of SUMOylation target-site modified AAV vectors are not immunogenic in comparison to wildtype AAV2 vectors and possess significantly higher gene expression, with respect to wild type, thereby improving efficiency of hepatic and ocular gene transfer.

ENGINEERED ADENO-ASSOCIATED VIRUS CAPSIDS
20220325296 · 2022-10-13 ·

Described herein are methods of generating engineered viral capsid variants. Also described herein are engineered viral capsid variants, engineered viral particles and formulations and cells thereof. Also described herein are vector systems containing an engineered viral capsid polynucleotide and uses thereof.

Polyethylene Glycol Conjugates of Polyethyleneimine and Their Use in Gene Therapy
20230120141 · 2023-04-20 ·

The present invention is directed to improved methods for conjugating polyethylene glycol to polyethyleneimine, and to the use of such polyethylene glycol—polyethyleneimine conjugates to improve the efficiency with which viral and non-viral nucleic acid vectors transfect cells to provide gene therapy, and to improve the efficiency of producing viral particles that comprise therapeutic polynucleotides for use in gene therapy.

DUAL BIFUNCTIONAL VECTORS FOR AAV PRODUCTION

The present invention relates novel combinations of nucleic acid constructs for the production of recombinant parvoviral gene therapy vectors. In particular the invention relates a combination preferably no more than two construct, the first construct expressing both the parvoviral Cap and Rep proteins, and the second construct at least comprising the transgene flanked ITRs and optionally again comprising an expression cassette forthe Cap proteins. The nucleic acid constructs are preferably baculoviral vectors for the production of rAAV in insect cells.

COMPOSITIONS AND METHODS FOR PRODUCTION OF RECOMBINANT ADENO-ASSOCIATED VIRUS

The present disclosure provides methods for producing recombinant adeno-associated virus (rAAV) virions. The present disclosure provides compositions for producing rAAV virions.

MANUFACTURING AND USE OF RECOMBINANT AAV VECTORS

Presented herein are technologies and methods for improved production of AAV vectors.

PRODUCTION OF ANTI-VIRAL MONOCLONAL ANTIBODIES BY HYDRODYNAMIC-BASED TRANSFECTION
20220332798 · 2022-10-20 ·

The present disclosure relates to methods of producing monoclonal antibodies in animals. In particular, the disclosure provides a method of producing, in vivo, antibodies against viral capsids (VCs) derived from a non-enveloped virus (NEV). The method includes administering to a subject, by hydrodynamic-based transfection, a first set of genetic material encoding NEV structural proteins to induce the subject's intracellular translation and assembly of the proteins into viral capsids. The method also includes administering a second set of genetic material encoding NEV non-structural proteins to facilitate the intracellular assembly of the NEV structural proteins. Thus, this method may be used to produce subject-generated antibody-producing cells that secrete anti-VC antibodies that may be harvested and screened for monoclonal anti-VC antibodies.

COMPOSITIONS AND METHODS FOR REDUCING CHROMATIN CONTENT OF BIOLOGICAL PREPARATIONS
20220315622 · 2022-10-06 ·

A method for removing chromatin from a cell culture harvest comprising the steps: providing a cell culture harvest containing a desired biological product selected from the group consisting of a parvovirus or an adeno-associated virus, incubating the cell culture harvest in an aqueous medium at a pH value within the range of 3.0 to 4.0 with an ionic strength corresponding to NaCl at a concentration within the range of 3.0 M to saturation and separating the desired biological product from solids produced.

NONINVASIVE QUANTITATION OF FULL VERSUS EMPTY CAPSIDS USING WATER PROTON NMR
20220317073 · 2022-10-06 ·

A method of using the relaxation rate (R.sub.1 and/or R.sub.2) of solvent NMR signal to noninvasively assess whether viral capsids in a capsid preparation are full or empty, and the percentage of full capsids if the vial contains a mixture of full and empty capsids. The method can simply, rapidly, and non-invasively prove the safety and potency of the capsid preparation and thus whether the capsid preparation can be approved for clinical use, without requiring any sample preparation or reagent addition.

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.