IL327379A - תרכובות ושיטות לדילוג על אקסון 50 בניוון שרירים דושן - Google Patents
תרכובות ושיטות לדילוג על אקסון 50 בניוון שרירים דושןInfo
- Publication number
- IL327379A IL327379A IL327379A IL32737926A IL327379A IL 327379 A IL327379 A IL 327379A IL 327379 A IL327379 A IL 327379A IL 32737926 A IL32737926 A IL 32737926A IL 327379 A IL327379 A IL 327379A
- Authority
- IL
- Israel
- Prior art keywords
- compounds
- methods
- muscular dystrophy
- duchenne muscular
- skipping exon
- 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
- 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
-
- 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
- C07K7/64—Cyclic peptides containing only normal peptide links
-
- 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)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Physics & Mathematics (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Peptides Or Proteins (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202363585311P | 2023-09-26 | 2023-09-26 | |
| US202363588115P | 2023-10-05 | 2023-10-05 | |
| US202463557125P | 2024-02-23 | 2024-02-23 | |
| US202463640506P | 2024-04-30 | 2024-04-30 | |
| PCT/US2024/048282 WO2025072246A1 (en) | 2023-09-26 | 2024-09-25 | Compounds and methods for skipping exon 50 in duchenne muscular dystrophy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL327379A true IL327379A (he) | 2026-05-01 |
Family
ID=93100244
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL327379A IL327379A (he) | 2023-09-26 | 2024-09-25 | תרכובות ושיטות לדילוג על אקסון 50 בניוון שרירים דושן |
Country Status (3)
| Country | Link |
|---|---|
| AU (1) | AU2024348141A1 (he) |
| IL (1) | IL327379A (he) |
| WO (1) | WO2025072246A1 (he) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20250325693A1 (en) * | 2024-04-03 | 2025-10-23 | Avidity Biosciences, Inc. | Antibody-oligonucleotide conjugate compositions and methods of inducing dmd exon 50 skipping |
Family Cites Families (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| CA2303299C (en) | 1997-09-12 | 2016-02-23 | Exiqon A/S | Oligonucleotide analogues |
| JP2002535247A (ja) | 1998-11-26 | 2002-10-22 | ペンタファルム アクチェンゲゼルシャフト | 輸送系複合体 |
| CA2392490A1 (en) | 1999-11-24 | 2001-05-31 | Mcs Micro Carrier Systems Gmbh | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
| JP4387669B2 (ja) | 2000-10-13 | 2009-12-16 | ザイジェン エス．アー． | 新規なトランスポーターペプチド配列による生物学的エフェクターの細胞内送達 |
| US7033597B2 (en) | 2000-10-13 | 2006-04-25 | Université de Lausanne | Intracellular delivery of biological effectors |
| CN105779453A (zh) * | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
| US10815276B2 (en) | 2014-05-21 | 2020-10-27 | Entrada Therapeutics, Inc. | Cell penetrating peptides and methods of making and using thereof |
| HUE045872T2 (hu) | 2014-05-21 | 2020-01-28 | Entrada Therapeutics Inc | Sejteket penetráló peptidek és ezek elõállítására és alkalmazására szolgáló eljárások |
| WO2017062835A2 (en) * | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions and methods for treating duchenne muscular dystrophy and related disorders |
| GB201711809D0 (en) | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
| MA50269A (fr) * | 2017-09-22 | 2020-07-29 | Avidity Biosciences Inc | Compositions d'acides nucléiques-polypeptides et méthodes d'induction de saut d'exon |
| EP4706689A2 (en) | 2019-12-19 | 2026-03-11 | Entrada Therapeutics, Inc. | Compositions for delivery of antisense compounds |
| US20250243244A1 (en) | 2021-03-31 | 2025-07-31 | Entrada Therapeutics, Inc. | Cyclic cell penetrating peptides |
| AU2022271873A1 (en) | 2021-05-10 | 2024-01-04 | Entrada Therapeutics, Inc. | Compositions and methods for intracellular therapeutics |
| GEAP202416440A (en) * | 2021-06-23 | 2024-04-25 | Entrada Therapeutics Inc | Antisense compounds and methods for targeting cug repeats |
| US20240390508A1 (en) * | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| EP4395829A1 (en) * | 2021-09-01 | 2024-07-10 | Entrada Therapeutics, Inc. | Compounds and methods for skipping exon 44 in duchenne muscular dystrophy |
| JP2024532465A (ja) * | 2021-09-01 | 2024-09-05 | エントラーダ セラピューティクス，インコーポレイティド | デュシェンヌ型筋ジストロフィーにおいてエクソン４５をスキッピングするための組成物及び方法 |
| US20230312653A1 (en) | 2022-03-31 | 2023-10-05 | Entrada Therapeutics, Inc. | Cyclic cell penetrating peptides |
-
2024
- 2024-09-25 IL IL327379A patent/IL327379A/he unknown
- 2024-09-25 WO PCT/US2024/048282 patent/WO2025072246A1/en active Pending
- 2024-09-25 AU AU2024348141A patent/AU2024348141A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| AU2024348141A1 (en) | 2026-05-14 |
| WO2025072246A9 (en) | 2025-05-08 |
| WO2025072246A1 (en) | 2025-04-03 |
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