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1. (WO2019067910) POLYNUCLEOTIDES, COMPOSITIONS, AND METHODS FOR GENOME EDITING
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Pub. No.: WO/2019/067910 International Application No.: PCT/US2018/053439
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
A61K 48/00 (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
Applicants:
INTELLIA THERAPEUTICS, INC. [US/US]; 40 Erie Street Cambridge, Massachusetts 02139, US
Inventors:
DOMBROWSKI, Christian; US
FINN, Jonathan Douglas; US
SMITH, Amy Madison Rhoden; US
ALEXANDER, Seth C.; US
Agent:
BAUR, Amelia Feulner; US
DOHERTY, Elizabeth A.; US
MCNEILL, Rebecca M.; US
SCARR, Rebecca B.; US
HENNINGER, Mary R.; US
BREIER, Adam M.; US
LEE, Victoria S.; US
PRYBYLOWSKI, Kate; US
KELLEY, Lara C.; US
HERZFELD, Deborah M.; US
SCHOENHARD, Amy E.L.; US
CHO, Hojung; US
Priority Data:
62/566,14429.09.2017US
Title (EN) POLYNUCLEOTIDES, COMPOSITIONS, AND METHODS FOR GENOME EDITING
(FR) POLYNUCLÉOTIDES, COMPOSITIONS ET PROCÉDÉS POUR L'ÉDITION GÉNOMIQUE
Abstract:
(EN) Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
(FR) L’invention concerne des compositions et méthodes pour l'édition génétique. Dans certains modes de réalisation, l'invention concerne un polynucléotide codant pour Cas9 qui peut fournir un ou plusieurs éléments parmi une efficacité d'édition améliorée, une immunogénicité réduite, ou d'autres avantages.
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)