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1. (WO2017151623) BIODEGRADABLE ACTIVATED POLYMERS FOR THERAPEUTIC DELIVERY
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Pub. No.: WO/2017/151623 International Application No.: PCT/US2017/019962
Publication Date: 08.09.2017 International Filing Date: 28.02.2017
IPC:
A61K 48/00 (2006.01) ,C08G 63/00 (2006.01) ,C08G 63/91 (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
08
ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
G
MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
63
Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
C CHEMISTRY; METALLURGY
08
ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
G
MACROMOLECULAR COMPOUNDS OBTAINED OTHERWISE THAN BY REACTIONS ONLY INVOLVING CARBON-TO-CARBON UNSATURATED BONDS
63
Macromolecular compounds obtained by reactions forming a carboxylic ester link in the main chain of the macromolecule
91
Polymers modified by chemical after-treatment
Applicants:
ALEXION PHARMACEUTICALS, INC. [US/US]; 100 College Street New Haven, CT 06510, US
YALE UNIVERSITY [US/US]; Two Whitney Avenue New Haven, CT 06510, US
Inventors:
CHENG, Christopher, J.; US
SALTZMAN, W., Mark; US
ZHANG, Junwei; US
Agent:
MOORE, Chester, Gunn; McBee Moore Woodward & Vanik IP, LLC 510 South Market Street Frederick, Maryland 21701, US
Priority Data:
62/301,91601.03.2016US
62/308,31915.03.2016US
Title (EN) BIODEGRADABLE ACTIVATED POLYMERS FOR THERAPEUTIC DELIVERY
(FR) POLYMÈRES ACTIVÉS BIODÉGRADABLES POUR ADMINISTRATION THÉRAPEUTIQUE
Abstract:
(EN) Activated polymers comprising one or more backbone ester(s) are disclosed. In particular, activated poly(amine-co-ester) (aPACE) terpolymers and methods of making and using these aPACE terpolymers are disclosed. These aPACE terpolymers can be used to safely and efficiently deliver biomolecules, in particular nucleic acids, to cells, both in vitro and in vivo. Methods for making activated polymers are also provided. Furthermore, methods for delivering mRNA and methods of gene therapy using activated polymers, in vitro and/or in vivo are further disclosed.
(FR) L’invention concerne des polymères activés comportant un ou plusieurs esters à squelette. L’invention concerne en particulier des terpolymères poly(amine-co-ester) activés (aPACE) et des procédés de fabrication et d'utilisation de ces terpolymères aPACE. Ces terpolymères aPACE peuvent être utilisés pour administrer sans danger et efficacement des biomolécules, en particulier des acides nucléiques à des cellules, in vitro et in vivo. L'invention concerne également des procédés de fabrication de ces polymères activés. L’invention concerne en outre, des procédés d'administration d'ARNm et des procédés de thérapie génique utilisant des polymères activés, in vitro et/ou in vivo.
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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, 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)