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1. (WO2019006043) TROPISM-MODIFIED RECOMBINANT VIRAL VECTORS AND USES THEREOF FOR THE TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS
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Pub. No.: WO/2019/006043 International Application No.: PCT/US2018/039874
Publication Date: 03.01.2019 International Filing Date: 27.06.2018
IPC:
C07K 14/015 (2006.01) ,C12N 15/35 (2006.01) ,C12N 15/864 (2006.01) ,A61K 35/76 (2015.01) ,A61K 48/00 (2006.01) ,C12N 5/10 (2006.01) ,C07K 16/18 (2006.01) ,C07K 16/28 (2006.01)
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
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
79
Vectors or expression systems specially adapted for eukaryotic hosts
85
for animal cells
86
Viral vectors
864
Parvoviral vectors
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35
Medicinal preparations containing material or reaction products thereof with undetermined constitution
66
Materials from micro-organisms
76
Viruses
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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
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
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
16
Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18
against material from animals or humans
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
16
Immunoglobulins, e.g. monoclonal or polyclonal antibodies
18
against material from animals or humans
28
against receptors, cell surface antigens or cell surface determinants
Applicants:
REGENERON PHARMACEUTICALS, INC. [US/US]; 777 Old Saw Mill River Road Tarrytown, New York 10591, US
Inventors:
KYRATSOUS, Christos; US
MURPHY, Andrew J.; US
WANG, Cheng; US
SABIN, Leah; US
Agent:
WU, Rita; US
Priority Data:
62/525,70427.06.2017US
Title (EN) TROPISM-MODIFIED RECOMBINANT VIRAL VECTORS AND USES THEREOF FOR THE TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS
(FR) VECTEURS VIRAUX RECOMBINÉS À TROPISME MODIFIÉ ET UTILISATIONS ASSOCIÉES POUR L'INTRODUCTION CIBLÉE DE MATÉRIEL GÉNÉTIQUE DANS DES CELLULES HUMAINES
Abstract:
(EN) Provided herein are compositions and methods for retargeting recombinant viral capsid proteins/capsids/vectors, e.g., in vivo, with a multispecific binding molecule, such as a bispecific antibody, that specifically binds a heterologous epitope displayed by the capsid protein and a protein expressed on the cell of interest for the targeted delivery of a nucleotide of interest.
(FR) L'invention concerne des compositions et des procédés de reciblage de protéines de capside/capsides/vecteurs viraux recombinés, par exemple, in vivo, avec une molécule de liaison multispécifique, telle qu'un anticorps bispécifique, qui se lie spécifiquement à un épitope hétérologue présenté par la protéine de capside et à une protéine exprimée sur la cellule d'intérêt pour l'administration ciblée d'un nucléotide d'intérêt.
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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)