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1. (WO2019028192) FACTOR VIII (FVIII) GENE THERAPY METHODS
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Pub. No.: WO/2019/028192 International Application No.: PCT/US2018/044892
Publication Date: 07.02.2019 International Filing Date: 01.08.2018
IPC:
A61K 38/37 (2006.01) ,A61K 45/06 (2006.01) ,A61K 48/00 (2006.01) ,C07K 14/755 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17
from animals; from humans
36
Blood coagulation or fibrinolysis factors
37
Factors VIII
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
45
Medicinal preparations containing active ingredients not provided for in groups A61K31/-A61K41/132
06
Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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
745
Blood coagulation or fibrinolysis factors
755
Factors VIII
Applicants:
SPARK THERAPEUTICS, INC. [US/US]; 3737 Market Street, Suite 1300 Philadelphia, Pennsylvania 19104, US
Inventors:
ANGUELA, Xavier; US
Agent:
BEDGOOD, Robert M.; US
Priority Data:
62/540,05301.08.2017US
62/583,89009.11.2017US
62/596,53508.12.2017US
62/596,67008.12.2017US
Title (EN) FACTOR VIII (FVIII) GENE THERAPY METHODS
(FR) MÉTHODES DE THÉRAPIE GÉNIQUE CIBLANT LE FACTEUR VIII (FVIII)
Abstract:
(EN) Methods of using vvectors comprising nucleic acid and nucleic acid variants encoding FVIII protein are disclosed. In particular embodiments, a method of treating a human having hemophilia A includes administering a recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid encoding Factor VIII (FVIII) or nucleic acid variant encoding Factor VIII (FVIII) having a B domain deletion (hFVIII-BDD). In some aspects, a nucleic acid variant has 95% or greater identity to SEQ ID NO:7 and/or a nucleic acid variant has no more than 2 cytosine-guanine dinucleotides (CpGs). In other aspects, a rAAV vector is administered to the human at a dose of less than about 6x1012 vector genomes per kilogram (vg/kg).
(FR) L'invention concerne des méthodes d'utilisation de vecteurs comprenant l'acide nucléique, et des variants de cet acide nucléique, codant pour la protéine FVIII. Dans des modes de réalisation particuliers, une méthode de traitement d'un humain atteint d'hémophilie A comprend l'administration d'un vecteur de type virus adéno-associé recombinant (rAAV) comprenant l'acide nucléique codant pour le facteur VIII (FVIII) ou un variant de l'acide nucléique codant pour le facteur VIII (FVIII) présentant une délétion du domaine B (hFVIII-BDD). Dans certains aspects, le variant de l'acide nucléique possède 95 % ou plus d'identité avec SEQ ID NO : 7 et/ou le variant de l'acide nucléique ne possède pas plus de 2 dinucléotides de cytosine-guanine (CpGs). Dans d'autres aspects, un vecteur rAAV est administré à l'humain à une dose inférieure à environ 6 x 1012 génomes de vecteur par kilogramme (vg/kg).
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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)