SG11202104960PA - Exon skipping oligomer conjugates for muscular dystrophy - Google Patents
Exon skipping oligomer conjugates for muscular dystrophyInfo
- Publication number
- SG11202104960PA SG11202104960PA SG11202104960PA SG11202104960PA SG11202104960PA SG 11202104960P A SG11202104960P A SG 11202104960PA SG 11202104960P A SG11202104960P A SG 11202104960PA SG 11202104960P A SG11202104960P A SG 11202104960PA SG 11202104960P A SG11202104960P A SG 11202104960PA
- Authority
- SG
- Singapore
- Prior art keywords
- muscular dystrophy
- exon skipping
- oligomer conjugates
- skipping oligomer
- conjugates
- Prior art date
Links
Classifications
-
- 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
-
- 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
-
- 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
- A61K47/68—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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
- A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
- A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
- A61K47/6807—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug or compound being a sugar, nucleoside, nucleotide, nucleic acid, e.g. RNA antisense
-
- 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
- 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
- C07K7/06—Linear peptides containing only normal peptide links having 5 to 11 amino acids
-
- 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
- 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
- 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
-
- 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)
- Bioinformatics & Cheminformatics (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Biotechnology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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 (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862779028P | 2018-12-13 | 2018-12-13 | |
| PCT/US2019/065581 WO2020123574A1 (en) | 2018-12-13 | 2019-12-11 | Exon skipping oligomer conjugates for muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| SG11202104960PA true SG11202104960PA (en) | 2021-06-29 |
Family
ID=69570813
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| SG11202104960PA SG11202104960PA (en) | 2018-12-13 | 2019-12-11 | Exon skipping oligomer conjugates for muscular dystrophy |
Country Status (17)
| Country | Link |
|---|---|
| US (3) | US20200190516A1 (https=) |
| EP (1) | EP3894558A1 (https=) |
| JP (2) | JP7658900B2 (https=) |
| KR (1) | KR20210104759A (https=) |
| CN (1) | CN113412330A (https=) |
| AR (1) | AR117320A1 (https=) |
| AU (2) | AU2019397461A1 (https=) |
| BR (1) | BR112021011018A2 (https=) |
| CA (1) | CA3122281A1 (https=) |
| CL (1) | CL2021001523A1 (https=) |
| CO (1) | CO2021008891A2 (https=) |
| EA (1) | EA202191601A1 (https=) |
| IL (3) | IL318474A (https=) |
| MX (1) | MX2021006737A (https=) |
| SG (1) | SG11202104960PA (https=) |
| TW (1) | TW202035695A (https=) |
| WO (1) | WO2020123574A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN114901823A (zh) | 2019-12-26 | 2022-08-12 | 日本新药株式会社 | 诱导外显子50的跳读的反义核酸 |
| CN119013401A (zh) * | 2022-03-17 | 2024-11-22 | 萨勒普塔医疗公司 | 二氨基磷酸酯吗啉代寡聚物缀合物 |
| WO2025212708A2 (en) * | 2024-04-03 | 2025-10-09 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 50 skipping |
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-
2019
- 2019-12-11 IL IL318474A patent/IL318474A/en unknown
- 2019-12-11 JP JP2021531988A patent/JP7658900B2/ja active Active
- 2019-12-11 AU AU2019397461A patent/AU2019397461A1/en not_active Abandoned
- 2019-12-11 EP EP19850744.4A patent/EP3894558A1/en active Pending
- 2019-12-11 WO PCT/US2019/065581 patent/WO2020123574A1/en not_active Ceased
- 2019-12-11 MX MX2021006737A patent/MX2021006737A/es unknown
- 2019-12-11 SG SG11202104960PA patent/SG11202104960PA/en unknown
- 2019-12-11 US US16/710,144 patent/US20200190516A1/en not_active Abandoned
- 2019-12-11 CN CN201980081396.8A patent/CN113412330A/zh active Pending
- 2019-12-11 EA EA202191601A patent/EA202191601A1/ru unknown
- 2019-12-11 BR BR112021011018-6A patent/BR112021011018A2/pt unknown
- 2019-12-11 KR KR1020217021183A patent/KR20210104759A/ko active Pending
- 2019-12-11 IL IL321667A patent/IL321667A/en unknown
- 2019-12-11 CA CA3122281A patent/CA3122281A1/en active Pending
- 2019-12-11 IL IL283646A patent/IL283646B2/en unknown
- 2019-12-12 AR ARP190103646A patent/AR117320A1/es unknown
- 2019-12-12 TW TW108145500A patent/TW202035695A/zh unknown
-
2021
- 2021-06-10 CL CL2021001523A patent/CL2021001523A1/es unknown
- 2021-06-11 US US17/345,624 patent/US20220017901A1/en not_active Abandoned
- 2021-07-06 CO CONC2021/0008891A patent/CO2021008891A2/es unknown
-
2024
- 2024-02-28 JP JP2024028361A patent/JP2024051108A/ja active Pending
- 2024-06-14 US US18/744,061 patent/US20250179487A1/en active Pending
-
2026
- 2026-03-24 AU AU2026202254A patent/AU2026202254A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| US20220017901A1 (en) | 2022-01-20 |
| CL2021001523A1 (es) | 2022-01-21 |
| JP2022511055A (ja) | 2022-01-28 |
| IL321667A (en) | 2025-08-01 |
| AU2019397461A1 (en) | 2021-07-29 |
| US20250179487A1 (en) | 2025-06-05 |
| IL283646B2 (en) | 2025-11-01 |
| IL318474A (en) | 2025-03-01 |
| EP3894558A1 (en) | 2021-10-20 |
| CO2021008891A2 (es) | 2021-10-29 |
| JP7658900B2 (ja) | 2025-04-08 |
| CN113412330A (zh) | 2021-09-17 |
| CA3122281A1 (en) | 2020-06-18 |
| KR20210104759A (ko) | 2021-08-25 |
| JP2024051108A (ja) | 2024-04-10 |
| TW202035695A (zh) | 2020-10-01 |
| MX2021006737A (es) | 2021-09-23 |
| US20200190516A1 (en) | 2020-06-18 |
| IL283646A (en) | 2021-07-29 |
| EA202191601A1 (ru) | 2022-01-19 |
| BR112021011018A2 (pt) | 2021-08-31 |
| AR117320A1 (es) | 2021-07-28 |
| IL283646B1 (en) | 2025-07-01 |
| WO2020123574A1 (en) | 2020-06-18 |
| AU2026202254A1 (en) | 2026-04-16 |
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