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1. (WO2019067786) A MULTI-ARMED POLYROTAXANE PLATFORM FOR PROTECTED NUCLEIC ACID DELIVERY
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Pub. No.: WO/2019/067786 International Application No.: PCT/US2018/053221
Publication Date: 04.04.2019 International Filing Date: 27.09.2018
IPC:
A61K 31/724 (2006.01) ,A61K 47/60 (2017.01) ,C08B 37/00 (2006.01) ,C08G 83/00 (2006.01) ,C12N 15/87 (2006.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
715
Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
716
Glucans
724
Cyclodextrins
[IPC code unknown for A61K 47/60]
C CHEMISTRY; METALLURGY
08
ORGANIC MACROMOLECULAR COMPOUNDS; THEIR PREPARATION OR CHEMICAL WORKING-UP; COMPOSITIONS BASED THEREON
B
POLYSACCHARIDES; DERIVATIVES THEREOF
37
Preparation of polysaccharides not provided for in groups C08B1/-C08B35/110; Derivatives thereof
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
83
Macromolecular compounds not provided for in groups C08G2/-C08G81/104
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
87
Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
Applicants:
THE REGENTS OF THE UNIVERSITY OF CALIFORNIA [US/US]; 1111 Franklin Street, 12th Floor Oakland, California 94607-5200, US
Inventors:
MENG, Huan; US
SPENCER, Melissa J.; US
PYLE, April D.; US
YOUNG, Courtney S.; US
LIU, Xiangsheng; US
JI, Ying; US
REZA EMAMI, Michael; US
Agent:
HUNTER, Tom; US
AUSTIN, James E.; US
SAMPSON, Roger S.; US
VILLENEUVE, Joseph M.; US
WEAVER, Jeffrey K.; US
SCHOLZ, Christian D.; US
Priority Data:
62/566,10029.09.2017US
62/687,71320.06.2018US
Title (EN) A MULTI-ARMED POLYROTAXANE PLATFORM FOR PROTECTED NUCLEIC ACID DELIVERY
(FR) PLATEFORME POLYROTAXANE MULTI-BRAS POUR L'ADMINISTRATION PROTÉGÉE D'ACIDES NUCLÉIQUES
Abstract:
(EN) In various embodiments a polyrotaxane carrier for in vivo delivery of a nucleic acid is provided. In certain embodiments the carrier comprises: a multi-arm polyethylene glycol (PEG) backbone comprising at least three arms; at least one cyclic compound having a cavity, where an arm of said multi-arm PEG backbone is threaded into the cavity of said cyclic compound forming an inclusion complex; a bulky moiety capping the terminal of the arm(s) threaded into said cyclic compound where said moiety inhibits dethreading of the cyclodextrin from the arm(s) of said backbone; and where at least one arm of said PEG backbone is free of cyclic compounds; and where said carrier has a net positive charge.
(FR) Selon divers modes de réalisation, l'invention concerne un véhicule polyrotaxane pour l'administration in vivo d'un acide nucléique. Dans certains modes de réalisation, le véhicule comprend : un squelette de polyéthylène glycol (PEG) multi-bras comprenant au moins trois bras; au moins un composé cyclique comportant une cavité, où un bras dudit squelette PEG multi-bras est enfilé dans la cavité dudit composé cyclique pour former un complexe d'inclusion; un fragment volumineux qui coiffe l'extrémité terminale du ou des bras enfilés dans ledit composé cyclique, où ledit fragment inhibe la désinclusion de la cyclodextrine du ou des bras dudit squelette; au moins un bras dudit squelette PEG est exempt de composés cycliques; et ledit véhicule a une charge nette positive.
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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)