BR112023016183A2 - Conjugados de peptídeo de penetração celular e métodos de uso dos mesmos - Google Patents
Conjugados de peptídeo de penetração celular e métodos de uso dos mesmosInfo
- Publication number
- BR112023016183A2 BR112023016183A2 BR112023016183A BR112023016183A BR112023016183A2 BR 112023016183 A2 BR112023016183 A2 BR 112023016183A2 BR 112023016183 A BR112023016183 A BR 112023016183A BR 112023016183 A BR112023016183 A BR 112023016183A BR 112023016183 A2 BR112023016183 A2 BR 112023016183A2
- Authority
- BR
- Brazil
- Prior art keywords
- amino acid
- acid residues
- methods
- peptide
- oligonucleotide
- Prior art date
Links
- 239000000863 peptide conjugate Substances 0.000 title abstract 2
- 230000035515 penetration Effects 0.000 title 1
- 125000000539 amino acid group Chemical group 0.000 abstract 4
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 3
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 abstract 1
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 abstract 1
- 101001053946 Homo sapiens Dystrophin Proteins 0.000 abstract 1
- 125000002091 cationic group Chemical group 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- 239000000562 conjugate Substances 0.000 abstract 1
- 230000002209 hydrophobic effect Effects 0.000 abstract 1
Classifications
-
- 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
- A61K47/62—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 a protein, peptide or polyamino acid
- A61K47/64—Drug-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
- A61K47/645—Polycationic or polyanionic oligopeptides, polypeptides or polyamino acids, e.g. polylysine, polyarginine, polyglutamic acid or peptide TAT
- A61K47/6455—Polycationic oligopeptides, polypeptides or polyamino acids, e.g. for complexing nucleic acids
-
- 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
- A61K47/62—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 a protein, peptide or polyamino acid
- A61K47/64—Drug-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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
-
- 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
- C07K7/08—Linear peptides containing only normal peptide links having 12 to 20 amino acids
-
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3233—Morpholino-type ring
-
- 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
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2320/32—Special delivery means, e.g. tissue-specific
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Epidemiology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| PCT/GB2021/050357 WO2022171972A1 (en) | 2021-02-12 | 2021-02-12 | Cell-penetrating peptide conjugates and methods of their use |
| PCT/GB2022/050371 WO2022172019A1 (en) | 2021-02-12 | 2022-02-11 | Cell-penetrating peptide conjugates and methods of their use |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| BR112023016183A2 true BR112023016183A2 (pt) | 2023-12-12 |
Family
ID=74669201
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112023016183A BR112023016183A2 (pt) | 2021-02-12 | 2022-02-11 | Conjugados de peptídeo de penetração celular e métodos de uso dos mesmos |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US20240299563A1 (https=) |
| EP (2) | EP4291244B1 (https=) |
| JP (1) | JP2024506099A (https=) |
| KR (1) | KR20240006019A (https=) |
| CN (1) | CN117295525A (https=) |
| AU (2) | AU2021427595A1 (https=) |
| BR (1) | BR112023016183A2 (https=) |
| CA (2) | CA3211038A1 (https=) |
| ES (1) | ES3058927T3 (https=) |
| IL (1) | IL305117A (https=) |
| MX (1) | MX2023009446A (https=) |
| WO (2) | WO2022171972A1 (https=) |
Families Citing this family (10)
| 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 |
| WO2022192749A2 (en) * | 2021-03-12 | 2022-09-15 | Pepgen Inc. | Methods of treating duchenne muscular dystrophy using peptide-oligonucleotide conjugates |
| EP4304657A4 (en) * | 2021-03-12 | 2026-04-01 | Pepgen Inc | METHODS OF TREATMENT OF TYPE 1 MYOTONIC DYSTROPHY USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES |
| TW202442246A (zh) | 2023-04-27 | 2024-11-01 | 美商薩羅塔治療公司 | 用於治療慢性腎病之反義寡聚物 |
| WO2025085810A2 (en) | 2023-10-18 | 2025-04-24 | Sarepta Therapeutics, Inc. | Antisense oligomers for treatment of centronuclear myopathies |
| GB202405980D0 (en) * | 2024-04-29 | 2024-06-12 | Univ Oxford Innovation Ltd | Conjugate for treating repeat expansion 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 |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3687808A (en) | 1969-08-14 | 1972-08-29 | Univ Leland Stanford Junior | Synthetic polynucleotides |
| EP2933332A1 (en) | 2004-06-28 | 2015-10-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| WO2009147368A1 (en) * | 2008-06-04 | 2009-12-10 | Medical Research Council | Peptides |
| BR122020021379B1 (pt) | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| TR201816523T4 (tr) | 2009-11-12 | 2018-11-21 | Univ Western Australia | Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler. |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| CA3092114A1 (en) * | 2011-05-05 | 2012-11-08 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US20140315977A1 (en) * | 2013-03-14 | 2014-10-23 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| PT3554552T (pt) * | 2016-12-19 | 2022-10-03 | Sarepta Therapeutics Inc | Conjugados de oligómero de skipping de exão para distrofia muscular |
| KR20240006023A (ko) * | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| KR102810425B1 (ko) * | 2016-12-19 | 2025-05-21 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| US20200131231A1 (en) * | 2017-02-17 | 2020-04-30 | Oxford University Innovation Limited | Cell penetrating peptides |
| EP3687547A1 (en) * | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| WO2019067979A1 (en) * | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| WO2019067981A1 (en) * | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
| US11555189B2 (en) * | 2017-10-18 | 2023-01-17 | Sarepta Therapeutics, Inc. | Antisense oligomer compounds |
| EP3765030A4 (en) * | 2018-03-16 | 2022-01-26 | Sarepta Therapeutics, Inc. | CHIMERIC PEPTIDES FOR ANTISENSE ADMINISTRATION |
| JP2021521794A (ja) * | 2018-04-26 | 2021-08-30 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| JP7579147B2 (ja) * | 2018-06-14 | 2024-11-07 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート |
| TW202449155A (zh) * | 2018-07-27 | 2024-12-16 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| GB201812972D0 (en) | 2018-08-09 | 2018-09-26 | Univ Oxford Innovation Ltd | Cell-penetrating peptides |
| GB201812980D0 (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 |
| WO2020214763A1 (en) * | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| JP2022536495A (ja) * | 2019-06-19 | 2022-08-17 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを治療する方法 |
| GB201911403D0 (en) * | 2019-08-09 | 2019-09-25 | Univ Oxford Innovation Ltd | Conjugate and uses thereof |
-
2021
- 2021-02-12 WO PCT/GB2021/050357 patent/WO2022171972A1/en not_active Ceased
- 2021-02-12 CA CA3211038A patent/CA3211038A1/en active Pending
- 2021-02-12 AU AU2021427595A patent/AU2021427595A1/en active Pending
-
2022
- 2022-02-11 WO PCT/GB2022/050371 patent/WO2022172019A1/en not_active Ceased
- 2022-02-11 CN CN202280028220.8A patent/CN117295525A/zh active Pending
- 2022-02-11 IL IL305117A patent/IL305117A/en unknown
- 2022-02-11 EP EP22704422.9A patent/EP4291244B1/en active Active
- 2022-02-11 MX MX2023009446A patent/MX2023009446A/es unknown
- 2022-02-11 BR BR112023016183A patent/BR112023016183A2/pt unknown
- 2022-02-11 ES ES22704422T patent/ES3058927T3/es active Active
- 2022-02-11 JP JP2023548577A patent/JP2024506099A/ja active Pending
- 2022-02-11 EP EP25221967.0A patent/EP4717702A2/en active Pending
- 2022-02-11 AU AU2022220252A patent/AU2022220252A1/en active Pending
- 2022-02-11 US US18/277,013 patent/US20240299563A1/en active Pending
- 2022-02-11 CA CA3211042A patent/CA3211042A1/en active Pending
- 2022-02-11 KR KR1020237029218A patent/KR20240006019A/ko active Pending
-
2025
- 2025-02-14 US US19/054,315 patent/US20250177543A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL305117A (en) | 2023-10-01 |
| CA3211038A1 (en) | 2022-08-18 |
| AU2022220252A1 (en) | 2023-09-28 |
| EP4717702A2 (en) | 2026-04-01 |
| US20250177543A1 (en) | 2025-06-05 |
| ES3058927T3 (en) | 2026-03-13 |
| WO2022172019A1 (en) | 2022-08-18 |
| EP4291244C0 (en) | 2025-12-10 |
| US20240299563A1 (en) | 2024-09-12 |
| WO2022171972A1 (en) | 2022-08-18 |
| KR20240006019A (ko) | 2024-01-12 |
| JP2024506099A (ja) | 2024-02-08 |
| AU2021427595A1 (en) | 2023-09-28 |
| EP4291244A1 (en) | 2023-12-20 |
| EP4291244B1 (en) | 2025-12-10 |
| CA3211042A1 (en) | 2022-08-18 |
| CN117295525A (zh) | 2023-12-26 |
| MX2023009446A (es) | 2023-10-05 |
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