AU2014346457A1 - Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety - Google Patents

Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety Download PDF

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Publication number
AU2014346457A1
AU2014346457A1 AU2014346457A AU2014346457A AU2014346457A1 AU 2014346457 A1 AU2014346457 A1 AU 2014346457A1 AU 2014346457 A AU2014346457 A AU 2014346457A AU 2014346457 A AU2014346457 A AU 2014346457A AU 2014346457 A1 AU2014346457 A1 AU 2014346457A1
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AU
Australia
Prior art keywords
sina
myostatin
nucleotides
conjugate
molecule
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2014346457A
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English (en)
Inventor
Duncan Brown
Vasant Jadhav
Tayeba KHAN
Laura Sepp-Lorenzino
Walter Richard Strapps
Marija TADIN-STRAPPS
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Sirna Therapeutics Inc
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 Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Publication of AU2014346457A1 publication Critical patent/AU2014346457A1/en
Assigned to SIRNA THERAPEUTICS, INC. reassignment SIRNA THERAPEUTICS, INC. Request for Assignment Assignors: BROWN, DUNCAN, JADHAV, VASANT, SEPP-LORENZINO, LAURA, SIRNA THERAPEUTICS, INC.
Priority to AU2021200392A priority Critical patent/AU2021200392A1/en
Abandoned legal-status Critical Current

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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • A61P21/06—Anabolic agents
    • 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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Endocrinology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Botany (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Toys (AREA)
AU2014346457A 2013-11-11 2014-11-10 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety Abandoned AU2014346457A1 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2021200392A AU2021200392A1 (en) 2013-11-11 2021-01-21 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361902358P 2013-11-11 2013-11-11
US61/902,358 2013-11-11
PCT/US2014/064837 WO2015070158A1 (en) 2013-11-11 2014-11-10 Systemic delivery of myostatin short interfering nucleic acids (sina) conjugated to a lipophilic moiety

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2021200392A Division AU2021200392A1 (en) 2013-11-11 2021-01-21 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety

Publications (1)

Publication Number Publication Date
AU2014346457A1 true AU2014346457A1 (en) 2016-06-02

Family

ID=51999542

Family Applications (2)

Application Number Title Priority Date Filing Date
AU2014346457A Abandoned AU2014346457A1 (en) 2013-11-11 2014-11-10 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
AU2021200392A Abandoned AU2021200392A1 (en) 2013-11-11 2021-01-21 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety

Family Applications After (1)

Application Number Title Priority Date Filing Date
AU2021200392A Abandoned AU2021200392A1 (en) 2013-11-11 2021-01-21 Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety

Country Status (6)

Country Link
US (5) US10004814B2 (https=)
EP (1) EP3068407A1 (https=)
JP (2) JP6672156B2 (https=)
AU (2) AU2014346457A1 (https=)
CA (1) CA2929574A1 (https=)
WO (1) WO2015070158A1 (https=)

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US10004814B2 (en) 2013-11-11 2018-06-26 Sirna Therapeutics, Inc. Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety
HK1243940A1 (zh) 2014-11-06 2018-07-27 Scholar Rock, Inc. 抗原肌肉生长抑制素/潜在肌肉生长抑制素的抗体及其用途
LT3350220T (lt) 2015-09-15 2021-09-27 Scholar Rock, Inc. Anti pro/latentinio miostatino antikūnai ir jų panaudojimas
CN115814077A (zh) 2016-01-08 2023-03-21 供石公司 抗-原肌生长抑制素/潜伏肌生长抑制素抗体及其使用方法
AU2017283546C1 (en) 2016-06-13 2020-11-19 Scholar Rock, Inc. Use of myostatin inhibitors and combination therapies
EP3516062A1 (en) 2016-09-21 2019-07-31 Alnylam Pharmaceuticals, Inc. Myostatin irna compositions and methods of use thereof
EP4035659A1 (en) 2016-11-29 2022-08-03 PureTech LYT, Inc. Exosomes for delivery of therapeutic agents
ES2944357T3 (es) 2017-01-06 2023-06-20 Scholar Rock Inc Tratamiento de enfermedades metabólicas inhibiendo la activación de miostatina
CN112930193A (zh) * 2018-08-02 2021-06-08 达因疗法公司 肌肉靶向复合物及其在治疗肌肉萎缩中的用途
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US20220193250A1 (en) 2018-08-02 2022-06-23 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
TW202039844A (zh) * 2018-12-19 2020-11-01 美商阿尼拉製藥公司 類澱粉前驅蛋白（ＡＰＰ）ＲＮＡｉ劑組成物及其使用方法
EP3995153A4 (en) * 2019-06-26 2023-11-22 KNC Laboratories Co., Ltd. Nucleic acid drug suppressing production of myostatin gene mrna
JP2022553641A (ja) * 2019-10-10 2022-12-26 インサーム（インスティテュ ナシオナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシェ メディカル） 疼痛の処置のためのアンチセンスオリゴヌクレオチド及びその使用
CA3163283A1 (en) * 2020-01-10 2021-07-15 Romesh R. Subramanian Muscle targeting complexes and uses thereof for modulation of genes associated with muscle health
AU2021340710A1 (en) * 2020-09-11 2023-04-06 Arrowhead Pharmaceuticals, Inc. Skeletal muscle delivery platforms and methods of use
US20240024492A1 (en) * 2020-12-07 2024-01-25 Olix Pharmaceuticals, Inc. Nucleic acid molecule for induction of asymmetric rnai for inhibiting expression of ror-beta
EP4355878A4 (en) * 2021-06-16 2025-04-09 Empirico Inc. TREATMENT OF MTRES1-RELATED DISEASES AND DISORDERS
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US20250339553A1 (en) 2022-04-15 2025-11-06 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating myotonic dystrophy
IL321628A (en) 2022-12-22 2025-08-01 Scholar Rock Inc Selective and potent inhibitory antibodies for myostatin activation

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Also Published As

Publication number Publication date
EP3068407A1 (en) 2016-09-21
AU2021200392A1 (en) 2021-03-18
CA2929574A1 (en) 2015-05-14
US11529428B2 (en) 2022-12-20
US20250312490A1 (en) 2025-10-09
JP2020114821A (ja) 2020-07-30
US20230144623A1 (en) 2023-05-11
US10004814B2 (en) 2018-06-26
JP2017500373A (ja) 2017-01-05
JP6672156B2 (ja) 2020-03-25
US20190060488A1 (en) 2019-02-28
WO2015070158A1 (en) 2015-05-14
US20210008226A1 (en) 2021-01-14
US20160256570A1 (en) 2016-09-08
US10729787B2 (en) 2020-08-04

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Date Code Title Description
PC1 Assignment before grant (sect. 113)

Owner name: SIRNA THERAPEUTICS, INC.

Free format text: FORMER APPLICANT(S): SEPP-LORENZINO, LAURA; SIRNA THERAPEUTICS, INC.; BROWN, DUNCAN; JADHAV, VASANT

MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted