1. WO2026174398 - PLASMID FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS (AAV)

Publication Number WO/2026/174398

Publication Date 27.08.2026

International Application No. PCT/CA2026/050270

International Filing Date 20.02.2026

IPC

C12N 15/864 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
63	Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79	Vectors or expression systems specially adapted for eukaryotic hosts
85	for animal cells
86	Viral vectors
864	Parvoviral vectors

C07K 14/015 2006.1

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
14	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005	from viruses
01	DNA viruses
015	Parvoviridae, e.g. feline panleukopenia virus, human parvovirus

C12N 5/10 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5	Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10	Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells

C12N 7/01 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
7	Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
01	Viruses, e.g. bacteriophages, modified by introduction of foreign genetic material

C12N 15/35 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof
31	Genes encoding microbial proteins, e.g. enterotoxins
33	Genes encoding viral proteins
34	Proteins from DNA viruses
35	Parvoviridae, e.g. feline panleukopenia virus, human parvovirus

C12N 15/864 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
63	Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79	Vectors or expression systems specially adapted for eukaryotic hosts
85	for animal cells
86	Viral vectors
864	Parvoviral vectors

C07K 14/015 2006.1

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
14	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
005	from viruses
01	DNA viruses
015	Parvoviridae, e.g. feline panleukopenia virus, human parvovirus

C12N 5/10 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5	Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10	Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells

C12N 7/01 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
7	Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
01	Viruses, e.g. bacteriophages, modified by introduction of foreign genetic material

C12N 15/35 2006.1

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
11	DNA or RNA fragments; Modified forms thereof
31	Genes encoding microbial proteins, e.g. enterotoxins
33	Genes encoding viral proteins
34	Proteins from DNA viruses
35	Parvoviridae, e.g. feline panleukopenia virus, human parvovirus

View less classifications

Applicants

• VVECTOR BIO INC. [CA]/[CA]

Inventors

• MARTINS FERNANDES PAES, Barbara Cristina
• VENEREO SANCHEZ, Alina
• FARNOS VILLAR, Omar

Agents

• LAVERY, DE BILLY, LLP

Priority Data

63/760,789		20.02.2025		US
63/889,656		29.09.2025		US

Publication Language English (en)

Filing Language English (en)

Designated States

View all

AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CV, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IQ, IR, IS, IT, JM, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, MG, MK, MN, MU, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UY, UZ, VC, VN, WS, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, CV, GH, GM, KE, LR, LS, MU, MW, MZ, NA, RW, SC, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, ME, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)

Hide all

Title

(EN) PLASMID FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS (AAV)

(FR) PLASMIDE POUR PRODUIRE UN VIRUS ADÉNO-ASSOCIÉ (VAA)

Abstract

(EN) Adeno-associated virus (AAV) is a promising tool for gene therapy, but its high manufacturing costs, driven by the complexity of the traditional triple plasmid transfection process, limit its widespread use. Even though traditional AAV production have relied in the transfection of three plasmids encoding essential components, like the inverted terminal repeats (ITRs), with the Rep and Cap genes, and the helper genes; putting these elements together in a single plasmid can reduce process complexity. Thus, simplifying the AAV production process by using a single plasmid could reduce costs and streamline production, making gene therapies more accessible. The present application discloses a single-plasmid system for the production of a recombinant parvovirus in a host cells. Host cells and kits comprising the plasmid, as well as methods for producing recombinant parvovirus using the single-plasmid system, are also disclosed.

(FR) Le virus adéno-associé (VAA) est un outil prometteur en thérapie génique, mais ses coûts de fabrication élevés, entraînés par la complexité du procédé de transfection de plasmide triple classique, limitent son utilisation généralisée. Bien que la production classique de VAA repose sur la transfection de trois plasmides codant des composants essentiels, comme les répétitions terminales inversées (ITR), avec les gènes Rep et Cap, et les gènes auxiliaires ; le regroupement de ces éléments ensemble dans un plasmide unique peut réduire la complexité du procédé. Ainsi, la simplification du procédé de production de VAA à l'aide d'un plasmide unique pourrait réduire les coûts et rationaliser la production, rendant des thérapies géniques plus accessibles. La présente demande concerne un système monoplasmidique pour la production d'un parvovirus recombinant dans des cellules hôtes. L'invention concerne également des cellules hôtes et des kits comprenant le plasmide, ainsi que des procédés de production de parvovirus recombinant à l'aide du système monoplasmidique.

Latest bibliographic data on file with the International Bureau

The publication of an international application under the Patent Cooperation Treaty in PATENTSCOPE (which constitutes an element of the Gazette) does not imply the expression of any opinion whatsoever on the part of the International Bureau of WIPO concerning the legal status of any country, territory, city or area or of its authorities, or concerning the delimitation of its frontiers or boundaries.