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1. (WO2018140411) THERAPEUTIC POLYMERIC NANOPARTICLES FOR TAILORED GENE EXPRESSION
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Pub. No.: WO/2018/140411 International Application No.: PCT/US2018/014898
Publication Date: 02.08.2018 International Filing Date: 23.01.2018
IPC:
A61K 48/00 (2006.01) ,A61K 47/69 (2017.01)
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
[IPC code unknown for A61K 47/69]
Applicants:
THE JOHNS HOPKINS UNIVERSITY [US/US]; 3400 North Charles Street Baltimore, Maryland 21218, US
Inventors:
BORN, Louis Joseph; US
HARMON, John W.; US
LAY, Frank; US
MARTI, Guy; US
NG, Christopher; US
ALIKHASSY, Zahra; US
ANSARI, Amir; US
Agent:
LOPEZ, Miguel A.; US
Priority Data:
62/449,88424.01.2017US
Title (EN) THERAPEUTIC POLYMERIC NANOPARTICLES FOR TAILORED GENE EXPRESSION
(FR) NANOPARTICULES POLYMÈRES THÉRAPEUTIQUES POUR UNE EXPRESSION GÉNIQUE PERSONNALISÉE
Abstract:
(EN) A composition for tailored, non-viral polynucleotide delivery to a cell including a plurality of nanoparticles and a polynucleotide attached to an exterior surface of at least one of the plurality of nanoparticles and methods of using and making the composition thereof. Each of the plurality of nanoparticles has a natural or synthetic cationic polymer electrostatically crosslinked with an anionic electrostatic crosslinking agent. Each of the plurality of nanoparticles have a positive zeta potential, and each of the plurality of nanoparticles are between 1 nm - 1000 nm in diameter.
(FR) L'invention concerne une composition pour une administration personnalisée de polynucléotide non viral à une cellule comprenant une pluralité de nanoparticules et un polynucléotide fixé à une surface extérieure d'au moins une nanoparticule parmi la pluralité de nanoparticules et des méthodes d'utilisation et de fabrication de ladite composition. Chaque nanoparticule parmi la pluralité de nanoparticules a un polymère cationique naturel ou synthétique réticulé électrostatiquement avec un agent de réticulation électrostatique anionique. Chaque nanoparticule parmi la pluralité de nanoparticules a un potentiel zêta positif, et chaque nanoparticule parmi la pluralité de nanoparticules présente un diamètre situé dans la plage allant de 1 nm à 1 000 nm.
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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)