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1. (WO2018222925) THERAPEUTICS FOR PHENYLKETONURIA
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Pub. No.: WO/2018/222925 International Application No.: PCT/US2018/035476
Publication Date: 06.12.2018 International Filing Date: 31.05.2018
IPC:
A61K 48/00 (2006.01) ,C07K 14/47 (2006.01) ,C12N 15/00 (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
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
Applicants:
ULTRAGENYX PHARMACEUTICAL INC. [US/US]; 60 Leveroni Court Novato, California 94949, US
Inventors:
TACHIKAWA, Kiyoshi; US
PEREZ-GARCIA, Carlos Gustavo; US
CHIVUKULA, Padmanabh; US
BHASKARAN, Hari Prakash; US
COBAUGH, Christian W.; US
DAUGHERTY, Sean Christopher; US
Agent:
ROY, Madhuri; US
WU, Nan; US
WANG, Yi; US
TUSCAN, Michael S.; US
Priority Data:
62/513,36331.05.2017US
Title (EN) THERAPEUTICS FOR PHENYLKETONURIA
(FR) AGENTS THÉRAPEUTIQUES POUR LA PHÉNYLCÉTONURIE
Abstract:
(EN) This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.
(FR) La présente invention concerne une gamme de molécules de polynucléotides et d'oligomères pouvant être translatées pour exprimer une phénylalanine hydroxylase humaine (PAH), ou un fragment associé ayant une activité de PAH. Les molécules de polynucléotides et d'oligomères peuvent être exprimées pour fournir une HAP humaine ou un fragment associé ayant une activité HAP. Les molécules peuvent être utilisées en tant qu'agents actifs pour exprimer une protéine ou un polypeptide actif dans des cellules ou chez des sujets. Les agents peuvent être utilisés dans des méthodes destinées à améliorer, prévenir, retarder l'apparition ou traiter une maladie ou un état associé à la phénylcétonurie, un métabolisme réduit de phénylalanine, ou des niveaux accrus de phénylalanine chez un sujet.
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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)