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SG11201809468XA - Oligonucleotide analogues targeting human lmna - Google Patents

Oligonucleotide analogues targeting human lmna

Info

Publication number
SG11201809468XA
SG11201809468XA SG11201809468XA SG11201809468XA SG11201809468XA SG 11201809468X A SG11201809468X A SG 11201809468XA SG 11201809468X A SG11201809468X A SG 11201809468XA SG 11201809468X A SG11201809468X A SG 11201809468XA SG 11201809468X A SG11201809468X A SG 11201809468XA
Authority
SG
Singapore
Prior art keywords
international
lmna
suite
office
rule
Prior art date
Application number
SG11201809468XA
Inventor
Michael R Erdos
Kan Cao
Ryszard Kole
Richard Keith Bestwick
Francis S Collins
Leslie B Gordon
Original Assignee
Sarepta Therapeutics Inc
Us Health
Univ Maryland
The Progeria Res Foundation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sarepta Therapeutics Inc, Us Health, Univ Maryland, The Progeria Res Foundation filed Critical Sarepta Therapeutics Inc
Publication of SG11201809468XA publication Critical patent/SG11201809468XA/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04—Linear peptides containing only normal peptide links
    • C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
    • C12N2310/3145—Phosphoramidates with the nitrogen in 3' or 5'-position
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
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  • Veterinary Medicine (AREA)
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  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 11111111111111111111111111111111111111111111111111111111111111111111111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2017/190041 Al 02 November 2017 (02.11.2017) W I PO I PCT (51) International Patent Classification: 0133 Cole Student Activities Building, College Park, Mary- C12N 15/113 (2010.01) C07H 21/00 (2006.01) land 20742 (US). A61K 31/712 (2006.01) (72) Inventors: ERDOS, Michael R.; Office of Technology (21) International Application Number: Transfer, NIH, 6011 Executive Boulevard, Suite 325, MSC PCT/US2017/030174 7660, Bethesda, Maryland 20892 (US). COLLINS, Fran- cis S.; Office of Technology Transfer, NIH, 6011 Exec- (22) International Filing Date: utive Boulevard, Suite 325, MSC 7660, Bethesda, Mary- 28 April 2017 (28.04.2017) land 20892 (US). CAO, Kan; 2215 Davit Court, Bowie, (25) Filing Language: English Maryland 20721 (US). KOLE, Ryszard; 136 Southwest (26) Publication Language: English Washington Avenue #405, Corvallis, Oregon 97333 (US). BESTWICK, Richard Keith; 3905 NW Clarence Circle, (30) Priority Data: Corvallis, Oregon 97330 (US). 62/330,027 29 April 2016 (29.04.2016) US (74) Agent: SUN, Eileen S. et al.; Seed Intellectual Property (71) Applicants: SAREPTA THERAPEUTICS, INC. Law Group LLP, Suite 5400, 701 Fifth Avenue, Seattle, [US/US]; 215 First Street, Suite 415, Cambridge, Mass- Washington 98104-7064 (US). achusetts 02142 (US). THE UNITED STATES OF (81) Designated States (unless otherwise indicated, for every AMERICA, AS REPRESENTED BY THE SEC- kind of national protection available): AE, AG, AL, AM, RETARY, DEPT. OF HEALTH AND HUMAN AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, SERVICES [US/US]; Office of Technology Transfer, NIH, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, 6011 Executive Boulevard, Suite 325, MSC 7660, Bethes- DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, da, Maryland 20892 (US). UNIVERSITY OF MARY- HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, LAND [[JS/US]; Office of Technology Commercialization, 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, = (54) Title: OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA = ddPCR assay 12-week PPMO pilot study: heart 250 = = 200 E Progerin copies/p1 B Wt LMNA copies/pi 1 2 Progerin/LMNA (%) = = = = 150 = 0) 2 = 0_ < 1 0 4 ,, 7 4 ior /e = z _ 50 11 ,—, ii. 0 / /• /7 ''''' 0 '< • — / / % % %. . , er fr ?;.... 4,... ... 11 Or d r: 0. ,,,.. 0 1 0,11 C5) 2 0 CONTROL PPMO1 PPMO1 PPMO1 PPMO2 PPMO2 PPMO2 0 60 mg/kg 20 mg/kg 7 mg/kg 60 mg/kg 20 mg/kg 7 mg/kg 01 ,-1 FIG. 1 IN ,-1 © (57) : Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced f'1 LMNA mRNA isoforms that encode progerin. o [Continued on next page] WO 2017/190041 Al IIMEDIMOMOIDEIROIDEMOMOHMONNIVOIIIMEMOIMIE 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. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (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), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Declarations under Rule 4.17: — as to the applicant's entitlement to claim the priority of the earlier application (Rule 4.17(iii)) Published: — with international search report (Art. 21(3)) — before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) — with sequence listing part of description (Rule 5.2(a))
SG11201809468XA 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna SG11201809468XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662330027P 2016-04-29 2016-04-29
PCT/US2017/030174 WO2017190041A1 (en) 2016-04-29 2017-04-28 Oligonucleotide analogues targeting human lmna

Publications (1)

Publication Number Publication Date
SG11201809468XA true SG11201809468XA (en) 2018-11-29

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US (2) US10822608B2 (en)
EP (1) EP3449000A1 (en)
JP (2) JP7042255B2 (en)
KR (1) KR102329187B1 (en)
CN (1) CN109477109B (en)
AU (2) AU2017258642B2 (en)
BR (1) BR112018072279A2 (en)
CA (1) CA3022303A1 (en)
CO (1) CO2018012873A2 (en)
EA (1) EA201892467A1 (en)
MA (1) MA45616A (en)
MX (1) MX2018013157A (en)
NZ (1) NZ747685A (en)
SG (1) SG11201809468XA (en)
WO (1) WO2017190041A1 (en)

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