IL273838B - Peptide and oligonucleotide conjugates, preparations containing them and their uses - Google Patents
Peptide and oligonucleotide conjugates, preparations containing them and their usesInfo
- Publication number
- IL273838B IL273838B IL273838A IL27383820A IL273838B IL 273838 B IL273838 B IL 273838B IL 273838 A IL273838 A IL 273838A IL 27383820 A IL27383820 A IL 27383820A IL 273838 B IL273838 B IL 273838B
- Authority
- IL
- Israel
- Prior art keywords
- conjugate
- alkyl
- occurrence
- independently
- amino acid
- Prior art date
Links
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
-
- 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
- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/42—Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein
-
- 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
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/04—Antibacterial agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
- A61P31/12—Antivirals
- A61P31/14—Antivirals for RNA viruses
- A61P31/16—Antivirals for RNA viruses for influenza or rhinoviruses
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Virology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Communicable Diseases (AREA)
- Oncology (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Biochemistry (AREA)
- Pulmonology (AREA)
- Rheumatology (AREA)
- Pain & Pain Management (AREA)
- Urology & Nephrology (AREA)
- Inorganic Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13/101,942 US20110269665A1 (en) | 2009-06-26 | 2011-05-05 | Compound and method for treating myotonic dystrophy |
| US13/107,528 US9238042B2 (en) | 2010-05-13 | 2011-05-13 | Antisense modulation of interleukins 17 and 23 signaling |
| PCT/US2011/061282 WO2012150960A1 (en) | 2011-05-05 | 2011-11-17 | Peptide oligonucleotide conjugates |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| IL273838A IL273838A (en) | 2020-05-31 |
| IL273838B true IL273838B (en) | 2022-09-01 |
Family
ID=45218873
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL273838A IL273838B (en) | 2011-05-05 | 2011-11-17 | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
| IL229227A IL229227B (en) | 2011-05-05 | 2013-11-04 | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL229227A IL229227B (en) | 2011-05-05 | 2013-11-04 | Peptide and oligonucleotide conjugates, preparations containing them and their uses |
Country Status (8)
| Country | Link |
|---|---|
| EP (1) | EP2704749A1 (enExample) |
| JP (6) | JP6478632B2 (enExample) |
| KR (3) | KR102339196B1 (enExample) |
| CN (2) | CN107693797B (enExample) |
| AU (6) | AU2011367230B2 (enExample) |
| CA (2) | CA2834128A1 (enExample) |
| IL (2) | IL273838B (enExample) |
| WO (1) | WO2012150960A1 (enExample) |
Families Citing this family (90)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3808845A1 (en) | 2004-06-28 | 2021-04-21 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
| ES2852549T3 (es) | 2005-02-09 | 2021-09-13 | Sarepta Therapeutics Inc | Composición antisentido para tratamiento de la atrofia muscular |
| EP2049664B1 (en) | 2006-08-11 | 2011-09-14 | Prosensa Technologies B.V. | Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| CA3066050A1 (en) | 2008-10-24 | 2010-04-29 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
| SI2499249T1 (sl) | 2009-11-12 | 2019-02-28 | The University Of Western Australia, | Protismiselne molekule in postopki zdravljenja patologij |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| HK1201514A1 (en) | 2011-11-18 | 2015-09-04 | Sarepta Therapeutics, Inc. | Functionally-modified oligonucleotides and subunits thereof |
| AU2012345638C1 (en) | 2011-11-30 | 2018-10-18 | Sarepta Therapeutics, Inc. | Induced exon inclusion in spinal muscle atrophy |
| ES2832531T3 (es) | 2011-11-30 | 2021-06-10 | Sarepta Therapeutics Inc | Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones |
| US9326992B2 (en) | 2011-12-08 | 2016-05-03 | Sarepta Therapeutics, Inc. | Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| PL2841578T3 (pl) | 2012-04-23 | 2017-12-29 | Biomarin Technologies B.V. | Oligonukleotydy modulujące RNA o ulepszonych właściwościach do leczenia zaburzeń nerwowo-mięśniowych |
| EP3885439A1 (en) | 2012-12-20 | 2021-09-29 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| EP2946013A1 (en) | 2013-01-16 | 2015-11-25 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| EP3760720A1 (en) | 2013-03-14 | 2021-01-06 | Sarepta Therapeutics, Inc. | Exon skipping compositions for treating muscular dystrophy |
| IL280443B (en) | 2013-03-14 | 2022-07-01 | Sarepta Therapeutics Inc | Preparations that skip axon for the treatment of muscular dystrophy |
| MX373959B (es) | 2013-03-15 | 2020-07-13 | Sarepta Therapeutics Inc | Composición para usarse en el tratamiento de la distrofia muscular de duchenne (dmd). |
| CN105228999B (zh) * | 2013-05-24 | 2021-03-02 | 味之素株式会社 | 吗啉代寡核苷酸的制备方法 |
| TWI736514B (zh) * | 2013-09-05 | 2021-08-21 | 美商薩羅塔治療公司 | 酸性α葡萄糖苷酶之反義股誘導之外顯子2包含 |
| WO2015097600A2 (en) | 2013-12-24 | 2015-07-02 | Sentiss Pharma Private Limited | Topical brimonidine tartrate ophthalmic solution |
| WO2015137409A1 (ja) | 2014-03-12 | 2015-09-17 | 日本新薬株式会社 | アンチセンス核酸 |
| KR101661277B1 (ko) * | 2014-03-17 | 2016-09-30 | 제주광어주식회사 | 한 개의 뉴크레오타이드 염기 변화를 통한 약독화 바이러스성 출혈패혈증 바이러스 |
| EP3620178A3 (en) | 2014-05-16 | 2020-07-22 | Oregon State University | Antisense antibacterial compounds and methods |
| US10391098B2 (en) | 2014-05-19 | 2019-08-27 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| JP6994941B2 (ja) | 2014-12-31 | 2022-02-04 | オレゴン ステート ユニバーシティ | アンチセンス抗細菌性化合物および方法 |
| US20180216111A1 (en) * | 2015-02-27 | 2018-08-02 | Sarepta Therapeutics, Inc. | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| MA41795A (fr) | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| WO2016187425A1 (en) * | 2015-05-19 | 2016-11-24 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| MA50829A (fr) | 2015-06-01 | 2018-04-11 | Sarepta Therapeutics Inc | Exclusion d'exon induite pat technologie antisens dans le collagène de type vii |
| US11020417B2 (en) | 2015-06-04 | 2021-06-01 | Sarepta Therapeutics, Inc | Methods and compounds for treatment of lymphocyte-related diseases and conditions |
| KR20250005537A (ko) * | 2015-08-05 | 2025-01-09 | 에자이 알앤드디 매니지먼트 가부시키가이샤 | 균질한 올리고머의 제조를 위한 키랄 시약 |
| HK1257498A1 (zh) * | 2015-08-28 | 2019-10-25 | Sarepta Therapeutics, Inc. | 在脊髓性肌肉萎缩症中用於内显子纳入的改良的反义寡聚体 |
| KR101841241B1 (ko) * | 2015-09-10 | 2018-03-22 | 한양대학교 산학협력단 | 피부 질환에 대한 예방 또는 치료효과를 갖는 펩티드 융합체 및 이를 유효성분으로 하는 약학 조성물 |
| MA45819A (fr) | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
| GB2545898B (en) | 2015-12-21 | 2019-10-09 | Sutura Therapeutics Ltd | Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides |
| US10907158B2 (en) | 2015-12-23 | 2021-02-02 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| AU2016379402B2 (en) | 2015-12-23 | 2023-01-12 | Board Of Regents, The University Of Texas System | Antisense antibacterial compounds and methods |
| KR102522059B1 (ko) | 2016-04-18 | 2023-04-14 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법 |
| SG11201809468XA (en) * | 2016-04-29 | 2018-11-29 | Sarepta Therapeutics Inc | Oligonucleotide analogues targeting human lmna |
| WO2017205879A2 (en) * | 2016-05-24 | 2017-11-30 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| CN109311919A (zh) * | 2016-06-30 | 2019-02-05 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体 |
| MA65895B1 (fr) * | 2016-12-19 | 2024-06-28 | Sarepta Therapeutics, Inc. | Conjugués d'oligomères induisant le saut d'exons pour la dystrophie musculaire |
| MD3554553T2 (ro) * | 2016-12-19 | 2022-10-31 | Sarepta Therapeutics Inc | Conjugați de oligomeri de omitere a exonului, pentru distrofie musculară |
| DK3554552T3 (da) * | 2016-12-19 | 2022-10-24 | Sarepta Therapeutics Inc | Exon-skipping-oligomerkonjugater mod muskeldystrofi |
| TW201919682A (zh) | 2017-08-08 | 2019-06-01 | 西班牙商阿爾米雷爾有限公司 | 活化Nrf2路徑的新穎化合物 |
| WO2019060522A2 (en) * | 2017-09-22 | 2019-03-28 | The Regents Of The University Of Colorado, A Body Corporate | OLIGONUCLEOTIDES THIOMORPHOLINO FOR THE TREATMENT OF MUSCLE DYSTROPHY |
| EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
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| WO2022140535A1 (en) | 2020-12-23 | 2022-06-30 | Sarepta Therapeutics, Inc. | Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy |
| WO2022171972A1 (en) * | 2021-02-12 | 2022-08-18 | Oxford University Innovation Limited | Cell-penetrating peptide conjugates and methods of their use |
| WO2022232478A1 (en) | 2021-04-30 | 2022-11-03 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
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