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1. (WO2019067872) COMPOSITIONS AND METHODS FOR TTR GENE EDITING AND TREATING ATTR AMYLOIDOSIS
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Pub. No.: WO/2019/067872 International Application No.: PCT/US2018/053382
Publication Date: 04.04.2019 International Filing Date: 28.09.2018
IPC:
C12N 15/113 (2010.01) ,C12N 15/10 (2006.01) ,A61K 48/00 (2006.01) ,A61P 25/28 (2006.01)
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
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
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
10
Processes for the isolation, preparation or purification of DNA or RNA
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
28
for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Applicants:
INTELLIA THERAPEUTICS, INC. [US/US]; 40 Erie Street Cambridge, Massachusetts 02139, US
Inventors:
KANJOLIA, Arti Mahendra Prakash; US
ODATE, Shobu; US
SEITZER, Jessica Lynn; US
LESCARBEAU, Reynald Michael; US
STRAPPS, Walter; 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:
62/566,23629.09.2017US
62/671,90215.05.2018US
Title (EN) COMPOSITIONS AND METHODS FOR TTR GENE EDITING AND TREATING ATTR AMYLOIDOSIS
(FR) COMPOSITIONS ET MÉTHODES POUR L'ÉDITION DU GÈNE TTR ET LE TRAITEMENT DE L'AMYLOÏDOSE ATTR
Abstract:
(EN) Compositions and methods for editing, e.g., introducing double-stranded breaks, within the TTR gene are provided. Compositions and methods for treating subjects having amyloidosis associated with transthyretin (ATTR), are provided.
(FR) L'invention concerne des compositions et des méthodes pour l'édition, par exemple, l'introduction de cassures à double brin dans le gène TTR . L'invention concerne des compositions et des méthodes pour traiter des sujets ayant une amyloïdose associée à la transthyrétine (ATTR).
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)