JP2024511955A - ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法 - Google Patents

ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法 Download PDF

Info

Publication number
JP2024511955A
JP2024511955A JP2023555748A JP2023555748A JP2024511955A JP 2024511955 A JP2024511955 A JP 2024511955A JP 2023555748 A JP2023555748 A JP 2023555748A JP 2023555748 A JP2023555748 A JP 2023555748A JP 2024511955 A JP2024511955 A JP 2024511955A
Authority
JP
Japan
Prior art keywords
seq
peptide
alkyl
terminus
complex
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.)
Pending
Application number
JP2023555748A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024511955A5 (https=
Inventor
キャロライン ゴドフリー,
ソニア ブレイスガードル,
アシュリング ホランド,
スミタ グンヌー,
Original Assignee
ペプゲン インコーポレイテッド.
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 ペプゲン インコーポレイテッド. filed Critical ペプゲン インコーポレイテッド.
Publication of JP2024511955A publication Critical patent/JP2024511955A/ja
Publication of JP2024511955A5 publication Critical patent/JP2024511955A5/ja
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/62Medicinal 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 a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3233Morpholino-type ring

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2023555748A 2021-03-12 2022-03-11 ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法 Pending JP2024511955A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163160705P 2021-03-12 2021-03-12
US63/160,705 2021-03-12
US202163194039P 2021-05-27 2021-05-27
US63/194,039 2021-05-27
PCT/US2022/020061 WO2022192749A2 (en) 2021-03-12 2022-03-11 Methods of treating duchenne muscular dystrophy using peptide-oligonucleotide conjugates

Publications (2)

Publication Number Publication Date
JP2024511955A true JP2024511955A (ja) 2024-03-18
JP2024511955A5 JP2024511955A5 (https=) 2025-04-03

Family

ID=83228540

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023555748A Pending JP2024511955A (ja) 2021-03-12 2022-03-11 ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法

Country Status (4)

Country Link
US (1) US20240200062A1 (https=)
EP (1) EP4304628A4 (https=)
JP (1) JP2024511955A (https=)
WO (1) WO2022192749A2 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20200131231A1 (en) 2017-02-17 2020-04-30 Oxford University Innovation Limited Cell penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US12465646B2 (en) 2018-12-07 2025-11-11 Oxford University Innovation Limited Linkers
EP4702143A2 (en) 2023-04-24 2026-03-04 BioMarin Pharmaceutical Inc. Compositions and methods for treating stxbp1 disorders
WO2025264554A1 (en) 2024-06-17 2025-12-26 Pepgen Inc. Antisense oligonucleotide conjugates targeting peripheral myelin protein 22 (pmp22) and methods of use thereof
WO2026080471A2 (en) 2024-10-07 2026-04-16 Flagship Pioneering Innovations Vi, Llc Compositions of trem conjugates and uses thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103998458B (zh) * 2011-08-30 2018-10-09 医学研究理事会 具有中央疏水域的细胞穿透肽
WO2015161255A1 (en) * 2014-04-18 2015-10-22 Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of mir124 and mir29 in subjects with muscular dystrophy
EP3858993A1 (en) * 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
GB201812972D0 (en) * 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
US12465646B2 (en) * 2018-12-07 2025-11-11 Oxford University Innovation Limited Linkers
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use

Also Published As

Publication number Publication date
WO2022192749A3 (en) 2022-10-27
EP4304628A2 (en) 2024-01-17
WO2022192749A8 (en) 2022-11-17
US20240200062A1 (en) 2024-06-20
EP4304628A4 (en) 2026-04-01
WO2022192749A2 (en) 2022-09-15

Similar Documents

Publication Publication Date Title
JP2024511955A (ja) ペプチド-オリゴヌクレオチド複合体を用いるデュシェンヌ型筋ジストロフィーの治療法
EP4291244B1 (en) Cell-penetrating peptide conjugates and methods of their use
JP2024511954A (ja) ペプチド-オリゴヌクレオチド複合体を用いる筋強直性ジストロフィー1型の治療法
JP2025538552A (ja) 細胞透過性ペプチド、その複合体、及びそれらの使用方法
JP7678654B2 (ja) 複合体及びその使用
JP2025063139A (ja) リンカー
AU2022354260A1 (en) Conjugated oligonucleotides and uses thereof
HK40106175A (zh) 使用肽-寡核苷酸缀合物治疗强直性肌营养不良1型的方法
HK40105562A (zh) 细胞穿透肽缀合物及其使用方法
JP2026510866A (ja) ミトコンドリアアミドキシム還元成分1(MARC1)の発現を阻害するためのRNAi剤、その医薬組成物、及び使用方法
HK40130357A (zh) 细胞穿透肽、其缀合物及其使用方法

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20250310

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250326