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1. (WO2018122610) EXON SKIPPING BY PEPTIDE NUCLEIC ACID DERIVATIVES
Latest bibliographic data on file with the International Bureau    Submit observation

Pub. No.: WO/2018/122610 International Application No.: PCT/IB2017/001725
Publication Date: 05.07.2018 International Filing Date: 29.12.2017
IPC:
C07K 14/00 (2006.01) ,C12N 15/113 (2010.01) ,A61K 38/00 (2006.01)
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38
Medicinal preparations containing peptides
Applicants:
OLIPASS CORPORATION [KR/KR]; 20th Floor, Ace Dongbaek Tower 16 Beon-gil, 16-4 Dongbaek Jungang-ro Giheung-gu, Yongin-si Gyeonggi-do 17105, KR
Inventors:
CHUNG, Shin; KR
JUNG, Daram; KR
CHO, Bongjun; KR
JANG, Kangwon; KR
YOON, Heungsik; KR
Priority Data:
62/440,92930.12.2016US
Title (EN) EXON SKIPPING BY PEPTIDE NUCLEIC ACID DERIVATIVES
(FR) SAUT D'EXON PAR DES DÉRIVÉS D'ACIDE NUCLÉIQUE PEPTIDIQUE
Abstract:
(EN) A peptide nucleic acid derivative of Formula (I) is provided to tightly bind to a splice site withiun a pre-mRNA in a sequence specific manner. Given with excellent cell membrane permeability and strong affinity for RNA, the said peptide nucleic acid derivative induces exon skipping in cells treated with the peptide nucleic acid at sub-femtomolar concentration as "naked" oligonucleotide. The said compound shows therapeutic activity in subjects upon systemic administration even at 1 μg/Kg or less, and therefore is useful to treat a disease or symptom at affordable treatment cost.
(FR) Cette invention concerne un dérivé d'acide nucléique peptidique de Formule I conçu pour se lier étroitement à un site d'épissage de pré-ARNm d'une manière spécifique de séquence. Bénéficiant d'une excellente perméabilité de la membrane cellulaire et d'une forte affinité pour l'ARN, ledit dérivé d'acide nucléique peptidique induit un saut d'exon dans les cellules traitées avec l'acide nucléique peptidique à une concentration sous-femtomolaire à titre d'oligonucléotide "nu". Ledit composé présente une activité thérapeutique chez les sujets après une administration par voie systémique même à 1 μg/Kg ou moins, et par conséquent est utile pour traiter une maladie ou un symptôme à un coût de traitement abordable.
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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)