CO2021014024A2 - Métodos para tratar la distrofia muscular con casimersin - Google Patents
Métodos para tratar la distrofia muscular con casimersinInfo
- Publication number
- CO2021014024A2 CO2021014024A2 CONC2021/0014024A CO2021014024A CO2021014024A2 CO 2021014024 A2 CO2021014024 A2 CO 2021014024A2 CO 2021014024 A CO2021014024 A CO 2021014024A CO 2021014024 A2 CO2021014024 A2 CO 2021014024A2
- Authority
- CO
- Colombia
- Prior art keywords
- muscular dystrophy
- methods
- casimersin
- treating muscular
- treating
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title abstract 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 1
- 229950009744 casimersen Drugs 0.000 abstract 1
- 101150015424 dmd gene Proteins 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 230000035772 mutation Effects 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- 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
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
-
- 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
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Dermatology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962825573P | 2019-03-28 | 2019-03-28 | |
| US201962902518P | 2019-09-19 | 2019-09-19 | |
| PCT/US2020/024550 WO2020198268A1 (en) | 2019-03-28 | 2020-03-25 | Methods for treating muscular dystrophy with casimersen |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CO2021014024A2 true CO2021014024A2 (es) | 2021-10-29 |
Family
ID=70286009
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CONC2021/0014024A CO2021014024A2 (es) | 2019-03-28 | 2021-10-20 | Métodos para tratar la distrofia muscular con casimersin |
Country Status (15)
| Country | Link |
|---|---|
| US (2) | US20220152086A1 (https=) |
| EP (1) | EP3946376A1 (https=) |
| JP (2) | JP2022526763A (https=) |
| KR (1) | KR20210145192A (https=) |
| CN (1) | CN113660939A (https=) |
| AU (2) | AU2020244803A1 (https=) |
| BR (1) | BR112021018801A2 (https=) |
| CA (1) | CA3134165A1 (https=) |
| CL (1) | CL2021002437A1 (https=) |
| CO (1) | CO2021014024A2 (https=) |
| IL (2) | IL318548A (https=) |
| MA (1) | MA55515A (https=) |
| MX (1) | MX2021011498A (https=) |
| SG (1) | SG11202108757XA (https=) |
| WO (1) | WO2020198268A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | 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 |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2021025899A1 (en) * | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
| 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 |
| WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CH445129A (fr) | 1964-04-29 | 1967-10-15 | Nestle Sa | Procédé pour la préparation de composés d'inclusion à poids moléculaire élevé |
| US3459731A (en) | 1966-12-16 | 1969-08-05 | Corn Products Co | Cyclodextrin polyethers and their production |
| US3453257A (en) | 1967-02-13 | 1969-07-01 | Corn Products Co | Cyclodextrin with cationic properties |
| US3426011A (en) | 1967-02-13 | 1969-02-04 | Corn Products Co | Cyclodextrins with anionic properties |
| US3453259A (en) | 1967-03-22 | 1969-07-01 | Corn Products Co | Cyclodextrin polyol ethers and their oxidation products |
| US4235871A (en) | 1978-02-24 | 1980-11-25 | Papahadjopoulos Demetrios P | Method of encapsulating biologically active materials in lipid vesicles |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| WO1986005519A1 (en) | 1985-03-15 | 1986-09-25 | James Summerton | Polynucleotide assay reagent and method |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US4737323A (en) | 1986-02-13 | 1988-04-12 | Liposome Technology, Inc. | Liposome extrusion method |
| KR0166088B1 (ko) | 1990-01-23 | 1999-01-15 | . | 수용해도가 증가된 시클로덱스트린 유도체 및 이의 용도 |
| EP0786522A2 (en) | 1992-07-17 | 1997-07-30 | Ribozyme Pharmaceuticals, Inc. | Enzymatic RNA molecules for treatment of stenotic conditions |
| US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
| US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
| US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
| IL115849A0 (en) | 1994-11-03 | 1996-01-31 | Merz & Co Gmbh & Co | Tangential filtration preparation of liposomal drugs and liposome product thereof |
| WO1999042091A2 (en) | 1998-02-19 | 1999-08-26 | Massachusetts Institute Of Technology | Use of polycations as endosomolytic agents |
| US7070807B2 (en) | 1999-12-29 | 2006-07-04 | Mixson A James | Branched histidine copolymers and methods for using same |
| WO2001047496A1 (en) | 1999-12-29 | 2001-07-05 | Mixson A James | Histidine copolymer and methods for using same |
| US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
| US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
| US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| WO2017059131A1 (en) * | 2015-09-30 | 2017-04-06 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
| JP2019525742A (ja) * | 2016-06-30 | 2019-09-12 | 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 |
| KR20240006023A (ko) * | 2016-12-19 | 2024-01-12 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체 |
| WO2019067981A1 (en) * | 2017-09-28 | 2019-04-04 | Sarepta Therapeutics, Inc. | POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY |
-
2020
- 2020-03-25 US US17/441,620 patent/US20220152086A1/en not_active Abandoned
- 2020-03-25 BR BR112021018801A patent/BR112021018801A2/pt unknown
- 2020-03-25 MX MX2021011498A patent/MX2021011498A/es unknown
- 2020-03-25 SG SG11202108757X patent/SG11202108757XA/en unknown
- 2020-03-25 CN CN202080022966.9A patent/CN113660939A/zh active Pending
- 2020-03-25 AU AU2020244803A patent/AU2020244803A1/en not_active Abandoned
- 2020-03-25 WO PCT/US2020/024550 patent/WO2020198268A1/en not_active Ceased
- 2020-03-25 CA CA3134165A patent/CA3134165A1/en active Pending
- 2020-03-25 MA MA055515A patent/MA55515A/fr unknown
- 2020-03-25 IL IL318548A patent/IL318548A/en unknown
- 2020-03-25 KR KR1020217034183A patent/KR20210145192A/ko active Pending
- 2020-03-25 EP EP20719076.0A patent/EP3946376A1/en not_active Withdrawn
- 2020-03-25 JP JP2021556752A patent/JP2022526763A/ja not_active Withdrawn
-
2021
- 2021-09-20 CL CL2021002437A patent/CL2021002437A1/es unknown
- 2021-09-23 IL IL286653A patent/IL286653A/en unknown
- 2021-10-20 CO CONC2021/0014024A patent/CO2021014024A2/es unknown
-
2025
- 2025-02-11 US US19/050,625 patent/US20260007690A1/en active Pending
- 2025-04-04 JP JP2025062366A patent/JP2025096396A/ja active Pending
- 2025-10-27 AU AU2025256263A patent/AU2025256263A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2021011498A (es) | 2022-01-04 |
| JP2025096396A (ja) | 2025-06-26 |
| EP3946376A1 (en) | 2022-02-09 |
| BR112021018801A2 (pt) | 2021-11-23 |
| IL286653A (en) | 2021-10-31 |
| IL318548A (en) | 2025-03-01 |
| CL2021002437A1 (es) | 2022-05-06 |
| US20260007690A1 (en) | 2026-01-08 |
| KR20210145192A (ko) | 2021-12-01 |
| CA3134165A1 (en) | 2020-10-01 |
| AU2025256263A1 (en) | 2025-11-27 |
| SG11202108757XA (en) | 2021-10-28 |
| JP2022526763A (ja) | 2022-05-26 |
| US20220152086A1 (en) | 2022-05-19 |
| CN113660939A (zh) | 2021-11-16 |
| AU2020244803A1 (en) | 2021-11-18 |
| MA55515A (fr) | 2022-02-09 |
| WO2020198268A1 (en) | 2020-10-01 |
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