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1. (WO2019067840) RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH'S ATAXIA BY INTRAVENOUS DELIVERY
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Pub. No.: WO/2019/067840 International Application No.: PCT/US2018/053312
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
A61K 48/00 (2006.01) ,C07K 14/47 (2006.01) ,C12N 7/00 (2006.01) ,C12N 15/113 (2010.01) ,C12N 15/86 (2006.01) ,C12N 15/861 (2006.01)
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
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435
from animals; from humans
46
from vertebrates
47
from mammals
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
7
Viruses, e.g. bacteriophages; Compositions thereof; Preparation or purification thereof
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
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
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
861
Adenoviral vectors
Applicants:
VOYAGER THERAPEUTICS, INC. [US/US]; 75 Sidney Street Cambridge, Massachusetts 02139, US
INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) [FR/FR]; 101, rue de Tolbiac 75013 Paris, FR
CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS) [FR/FR]; 3, rue Michel Ange 75016 Paris, FR
UNIVERSITÉ DE STRASBOURG [FR/FR]; 4, rue Blaise Pascal 67000 Strasbourg, FR
Inventors:
SAH, Dinah Wen-Yee; US
GOULET, Martin; US
PATZKE, Holger; US
SHU, Yanqun; US
HOU, Jinzhao; US
PUCCIO, Hélène; FR
Agent:
WARD, Donna T.; US
Priority Data:
62/565,84029.09.2017US
62/663,83527.04.2018US
Title (EN) RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH'S ATAXIA BY INTRAVENOUS DELIVERY
(FR) SAUVETAGE DE PHÉNOTYPE NEUROLOGIQUE CENTRAL ET PÉRIPHÉRIQUE DE L'ATAXIE DE FRIEDREICH PAR ADMINISTRATION INTRAVEINEUSE
Abstract:
(EN) Provided herein is an adeno-associated virus (AAV) particle comprising a capsid and a viral genome, wherein said capsid delivers the AAV particle to a nervous system, and wherein said viral genome comprises a polynucleotide sequence encoding Frataxin and one or more microRNA binding sites. Also, provided herein is a method for treating, ameliorating, and/or preventing a disorder in a subject stemming from a loss or partial loss of frataxin protein in the subject, wherein the method comprises: administering to the subject a therapeutically effective amount of a composition comprising an AAV particle comprising a capsid and a viral genome, wherein said capsid delivers the viral genome to a nervous system, and wherein said viral genome comprises a polynucleotide sequence encoding Frataxin and one or more microRNA binding sites, as described herein.
(FR) L'invention concerne une particule de virus adéno-associé (AAV) comprenant une capside et un génome viral, ladite capside administrant la particule d'AAV à un système nerveux, et ledit génome viral comprenant une séquence polynucléotidique codant pour la frataxine et un ou plusieurs sites de liaison de microARN. L'invention concerne également un procédé de traitement, de soulagement, et/ou de prévention d'un trouble chez un sujet provenant d'une perte ou d'une perte partielle de la protéine de frataxine chez le sujet, le procédé comprenant : l'administration au sujet d'une quantité thérapeutiquement efficace d'une composition comprenant une particule d'AAV comprenant une capside et un génome viral, ladite capside administrant le génome viral à un système nerveux, et ledit génome viral comprenant une séquence polynucléotidique codant pour la frataxine et un ou plusieurs sites de liaison de microARN, tels que décrits ici.
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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)