CA3243078A1 - Antisense therapeutic products conjugate to pre-MIARN splicing modulating peptides for the treatment of diseases - Google Patents
Antisense therapeutic products conjugate to pre-MIARN splicing modulating peptides for the treatment of diseasesInfo
- Publication number
- CA3243078A1 CA3243078A1 CA3243078A CA3243078A CA3243078A1 CA 3243078 A1 CA3243078 A1 CA 3243078A1 CA 3243078 A CA3243078 A CA 3243078A CA 3243078 A CA3243078 A CA 3243078A CA 3243078 A1 CA3243078 A1 CA 3243078A1
- Authority
- CA
- Canada
- Prior art keywords
- mrna
- slice
- diseases
- treatment
- conjugated antisense
- 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
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
-
- 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
- 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
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
-
- 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
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmacology & Pharmacy (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263303577P | 2022-01-27 | 2022-01-27 | |
| US63/303,577 | 2022-01-27 | ||
| PCT/CA2023/050096 WO2023141710A1 (en) | 2022-01-27 | 2023-01-26 | Pre-mrna slice modulating peptide-conjugated antisense therapeutics for the treatment of diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3243078A1 true CA3243078A1 (en) | 2023-08-03 |
Family
ID=87469898
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3243078A Pending CA3243078A1 (en) | 2022-01-27 | 2023-01-26 | Antisense therapeutic products conjugate to pre-MIARN splicing modulating peptides for the treatment of diseases |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20250114464A1 (OSRAM) |
| EP (1) | EP4469576A4 (OSRAM) |
| JP (1) | JP2025508337A (OSRAM) |
| CN (1) | CN119137274A (OSRAM) |
| CA (1) | CA3243078A1 (OSRAM) |
| WO (1) | WO2023141710A1 (OSRAM) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| JP7611825B2 (ja) | 2018-08-02 | 2025-01-10 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体およびそれらの使用 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3137715A1 (en) | 2019-04-25 | 2020-10-29 | Avidity Biosciences, Inc. | Nucleic acid compositions and methods of multi-exon skipping |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9161948B2 (en) * | 2011-05-05 | 2015-10-20 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| US9801948B2 (en) * | 2011-09-21 | 2017-10-31 | Yale University | Antimicrobial compositions and methods of use thereof |
| WO2021003573A1 (en) * | 2019-07-09 | 2021-01-14 | The Governors Of The University Of Alberta | Exons 45-55 skipping using mutation-tailored cocktails of antisense morpholinos in the dmd gene |
| JP7684971B2 (ja) * | 2019-12-20 | 2025-05-28 | ピーワイシー セラピューティクス リミテッド | 新規の細胞内送達方法 |
-
2023
- 2023-01-26 US US18/730,955 patent/US20250114464A1/en active Pending
- 2023-01-26 JP JP2024544912A patent/JP2025508337A/ja active Pending
- 2023-01-26 WO PCT/CA2023/050096 patent/WO2023141710A1/en not_active Ceased
- 2023-01-26 EP EP23745765.0A patent/EP4469576A4/en active Pending
- 2023-01-26 CA CA3243078A patent/CA3243078A1/en active Pending
- 2023-01-26 CN CN202380028701.3A patent/CN119137274A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20250114464A1 (en) | 2025-04-10 |
| EP4469576A4 (en) | 2026-04-15 |
| WO2023141710A1 (en) | 2023-08-03 |
| CN119137274A (zh) | 2024-12-13 |
| JP2025508337A (ja) | 2025-03-26 |
| EP4469576A1 (en) | 2024-12-04 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A00 | Application filed |
Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A00-A101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION RECEIVED - PCT Effective date: 20240717 |
|
| A15 | Pct application entered into the national or regional phase |
Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A15-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: NATIONAL ENTRY REQUIREMENTS DETERMINED COMPLIANT Effective date: 20240717 |
|
| P18 | Priority claim added or amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P18-P105 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIORITY CLAIM REQUIREMENTS DETERMINED COMPLIANT Effective date: 20240718 |
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| W00 | Other event occurred |
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| MFA | Maintenance fee for application paid |
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| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS Effective date: 20260120 |