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1. (WO2019043630) ADENO-ASSOCIATED VIRUS (AAV) WITH MODIFIED PHOSPHOLIPASE DOMAIN
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Pub. No.: WO/2019/043630 International Application No.: PCT/IB2018/056651
Publication Date: 07.03.2019 International Filing Date: 30.08.2018
IPC:
C12N 15/35 (2006.01) ,C12N 15/64 (2006.01) ,C12N 15/864 (2006.01) ,C07K 14/015 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; 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
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; 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
64
General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; 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
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
Applicants:
BENITEC BIOPHARMA LIMITED [AU/AU]; 99 Mount Street, Suite 1201 North Sydney, New South Wales 2060, AU
Inventors:
STRINGS-UFOMBAH, Vanessa; AU
KAO, Shih-Chu; AU
ROELVINK, Petrus W.; AU
Priority Data:
62/553,02831.08.2017US
Title (EN) ADENO-ASSOCIATED VIRUS (AAV) WITH MODIFIED PHOSPHOLIPASE DOMAIN
(FR) VIRUS ADÉNO-ASSOCIÉ (AAV) AVEC DOMAINE DE PHOSPHOLIPASE MODIFIÉ
Abstract:
(EN) The present disclosure relates generally to modified adeno-associated virus (AAV) from serotypes other than serotype 2, which have a viral capsid protein with a subunit 1 (VP1) sequence which is modified relative to the corresponding wildtype sequence. In particular, the modified AAVs of the disclosure comprise site-specific amino acid substitutions within the phospholipase A2 (PLA2) domain and flanking sequence relative to the corresponding wild-type sequence which improve functionality of the AAV when produced in insect cells. The present disclosure also relates to methods of producing the modified AAVs, reagents therefor, baculovirus expression systems and insect cells for producing said modified AAVs.
(FR) La présente invention concerne d'une manière générale des virus adéno-associés (AAV) modifiés de sérotypes autres que le sérotype 2, qui ont une protéine de capside virale avec une séquence de sous-unité 1 (VP1) qui est modifiée par rapport à la séquence de type sauvage correspondante. En particulier, les AAV modifiés de l'invention comprennent des substitutions d'acides aminés spécifiques au site à l'intérieur du domaine de phospholipase A2 (PLA2) et de la séquence flanquante par rapport à la séquence de type sauvage correspondante qui améliorent la fonctionnalité des AAV lorsqu'ils sont produits dans des cellules d'insectes. La présente invention concerne également des méthodes de production des AAV modifiés, des réactifs pour celles-ci, des systèmes d'expression de baculovirus et des cellules d'insecte pour produire lesdits AAV modifiés.
Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, 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, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, 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)
Publication Language: English (EN)
Filing Language: English (EN)