SG11201808981XA - Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof - Google Patents

Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Info

Publication number
SG11201808981XA
SG11201808981XA SG11201808981XA SG11201808981XA SG11201808981XA SG 11201808981X A SG11201808981X A SG 11201808981XA SG 11201808981X A SG11201808981X A SG 11201808981XA SG 11201808981X A SG11201808981X A SG 11201808981XA SG 11201808981X A SG11201808981X A SG 11201808981XA
Authority
SG
Singapore
Prior art keywords
international
opmd
pct
reagents
treatment
Prior art date
Application number
SG11201808981XA
Inventor
David Suhy
Michael Graham
Capucine Trollet
Alberto Malerba
George J Dickson
Original Assignee
Benitec Biopharma Ltd
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 Benitec Biopharma Ltd filed Critical Benitec Biopharma Ltd
Publication of SG11201808981XA publication Critical patent/SG11201808981XA/en

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    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/41—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a Myc-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/20—Vector systems having a special element relevant for transcription transcription of more than one cistron

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property -, Organization 111111110111101110101011111010111110111010111011111010111011111111101111011111 International Bureau 0.. .... .. ...... ... (10) International Publication Number (43) International Publication Date WO 2017/177277 Al 19 October 2017(19.10.2017) WIPO I PCT (51) International Patent Classification: (74) Agent: FB RICE; Level 23, 44 Market St, Sydney, New C12N 15/113 (2010.01) A61K 48/00 (2006.01) South Wales 2000 (AU). (21) International Application Number: (81) Designated States (unless otherwise indicated, for every PCT/AU2017/050330 kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, (22) International Filing Date: BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, 13 April 2017 (13.04.2017) DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, (25) Filing Language: English HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, (26) Publication Language: English MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, (30) Priority Data: NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, 62/322,745 14 April 2016 (14.04.2016) US 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, (71) Applicant: BENITEC BIOPHARMA LIMITED ZA, ZM, ZW. [AU/AU]; Suite 1201, 99 Mount Street North Sydney, New South Wales 2060 (AU). (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, (72) Inventors: SUHY, David; 32 Hurst Court, San Ramon, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, California 94583 (US). GRAHAM, Michael; Apt 217, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, 1125 Park Place, San Mateo, California 94403 (US). TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, TROLLET, Capucine; c/o Sorbonne Universites, UPMC DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, _ Univ Paris 06, UM76, INSERM U974, Institut de Myolo- LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, — gie, CNRS FRE3617, 47 bd de l'Hopital, 75013 Paris (FR). SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, _ MALERBA, Alberto; c/o School of Biological Sciences, GW, KM, ML, MR, NE, SN, TD, TG). Royal Holloway, University of London, Egham Surrey = TW20 OEX (GB). DICKSON, George J.; c/o School of Published: Biological Sciences, Royal Holloway, University of Lon- — with international search report (Art. 21(3)) don, Egham Surrey TW20 OEX (GB). = — with sequence listing part of description (Rule 5.2(a)) = = = = = = = = (54) Title: REAGENTS FOR TREATMENT THEREOF = = = _ Il Ir ... ' ID Ir ... ' OF OCULOPHARYNGEAL MUSCULAR Figure 1 ROM DYSTROPHY (OPMD) AND USE N 0.00 IN ,-, eJ rn , r•I en < < < Z Z Z < < < Z Z Z N Il C ui 5 5 5 5 5, Si TS -0 \"Ci \"Ci \"Ci IN Il N (57) : The present disclosure relates to RNA interference (RNAi) reagents targeting PABPN1 for treatment of oculopharyn- geal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or 0 which are predisposed thereto.
SG11201808981XA 2016-04-14 2017-04-13 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof SG11201808981XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662322745P 2016-04-14 2016-04-14
PCT/AU2017/050330 WO2017177277A1 (en) 2016-04-14 2017-04-13 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Publications (1)

Publication Number Publication Date
SG11201808981XA true SG11201808981XA (en) 2018-11-29

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SG11201808981XA SG11201808981XA (en) 2016-04-14 2017-04-13 Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof

Country Status (15)

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US (1) US11234994B2 (en)
EP (1) EP3443091A4 (en)
JP (1) JP7236195B2 (en)
KR (1) KR102353847B1 (en)
CN (1) CN109219659B (en)
AU (1) AU2017250017B2 (en)
BR (1) BR112018071186A8 (en)
CA (1) CA3020754C (en)
IL (1) IL262337B2 (en)
MX (1) MX2018012545A (en)
NZ (1) NZ747314A (en)
RU (1) RU2755544C2 (en)
SG (1) SG11201808981XA (en)
WO (1) WO2017177277A1 (en)
ZA (1) ZA201807589B (en)

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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102353847B1 (en) 2016-04-14 2022-01-21 베니텍 바이오파마 리미티드 Reagents for the treatment of oropharyngeal muscular dystrophy (OPMD) and uses thereof
KR20230020586A (en) * 2016-12-14 2023-02-10 베니텍 아이피 홀딩스 아이엔씨. Reagents for treatment of oculopharyngeal muscular dystrophy(OPMD) and use thereof
CN112867511A (en) * 2018-10-17 2021-05-28 贝尼泰克生物制药有限公司 Method for treating oculopharyngeal muscular dystrophy (OPMD)

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KR102353847B1 (en) 2016-04-14 2022-01-21 베니텍 바이오파마 리미티드 Reagents for the treatment of oropharyngeal muscular dystrophy (OPMD) and uses thereof

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KR20180133500A (en) 2018-12-14
US20200138849A1 (en) 2020-05-07
BR112018071186A2 (en) 2019-02-12
CA3020754C (en) 2023-07-25
JP2019513395A (en) 2019-05-30
WO2017177277A1 (en) 2017-10-19
AU2017250017A1 (en) 2018-11-08
MX2018012545A (en) 2019-06-10
EP3443091A4 (en) 2019-11-27
CN109219659A (en) 2019-01-15
IL262337B2 (en) 2023-06-01
ZA201807589B (en) 2023-02-22
CN109219659B (en) 2022-09-09
RU2018138271A3 (en) 2020-09-22
AU2017250017B2 (en) 2022-12-22
JP7236195B2 (en) 2023-03-09
RU2018138271A (en) 2020-05-14
BR112018071186A8 (en) 2023-03-28
EP3443091A1 (en) 2019-02-20
RU2755544C2 (en) 2021-09-17
NZ747314A (en) 2022-07-29
IL262337A (en) 2018-11-29
KR102353847B1 (en) 2022-01-21
CA3020754A1 (en) 2017-10-19
US11234994B2 (en) 2022-02-01

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