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1. (WO2019113149) CRISPR-CAS9 MODIFIED CD34+ HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS AND USES THEREOF
Latest bibliographic data on file with the International BureauSubmit observation

Pub. No.: WO/2019/113149 International Application No.: PCT/US2018/063973
Publication Date: 13.06.2019 International Filing Date: 05.12.2018
IPC:
A61K 38/46 (2006.01) ,C12N 9/22 (2006.01) ,C12N 15/10 (2006.01) ,C12N 15/113 (2010.01)
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
16
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43
Enzymes; Proenzymes; Derivatives thereof
46
Hydrolases (3)
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
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
22
Ribonucleases
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
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
Applicants:
CRISPR THERAPEUTICS AG [CH/CH]; Baarerstrasse 14 ZUG 6300, CH
VERTEX PHARMACEUTICALS INCORPORATED [US/US]; 50 Northern Avenue Boston, MA 02210, US
Inventors:
MORAWA, Ewelina; US
CHAKRABORTY, Tirtha; US
LUNDBERG, Ante, Sven; US
HO, Tony; US
SANDLER, Laura; US
EUSTACE, Brenda; US
ROSSERT, Jerome; US
KAUFFMAN, Robert; US
Agent:
CHEN, Yahua; US
KROM, Amy, Mcmahon; US
Priority Data:
62/594,68905.12.2017US
62/664,02327.04.2018US
62/671,77015.05.2018US
62/734,43121.09.2018US
62/734,54321.09.2018US
Title (EN) CRISPR-CAS9 MODIFIED CD34+ HUMAN HEMATOPOIETIC STEM AND PROGENITOR CELLS AND USES THEREOF
(FR) CELLULES SOUCHES ET PROGÉNITRICES HÉMATOPOÏÉTIQUES HUMAINES CD34+ MODIFIÉES PAR CRISPR-CAS9 ET UTILISATIONS ASSOCIÉES
Abstract:
(EN) Provided herein, in some embodiments, are methods and compositions for treatment of subjects with β-thalassemia and subjects with severe sickle cell disease using autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells.
(FR) Dans certains modes de réalisation, l'invention concerne des méthodes et des compositions pour le traitement de sujets atteints de β-thalassémie et de sujets atteints d'une drépanocytose grave à l'aide de cellules souches et progénitrices hématopoïétiques humaines CD34+ modifiées par CRISPR-Cas9 autologues.
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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 Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (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)