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
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)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Dermatology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente descripción proporciona, entre otras cosas, composiciones y métodos mejorados para tratar la distrofia muscular. Por ejemplo, la descripción proporciona métodos para tratar pacientes con distrofia muscular de Duchenne que tienen una mutación en el gen DMD que es susceptible a la omisión del exón 45 por la administración de una cantidad efectiva de casimersen.
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 (1) | US20220152086A1 (es) |
EP (1) | EP3946376A1 (es) |
JP (1) | JP2022526763A (es) |
KR (1) | KR20210145192A (es) |
CN (1) | CN113660939A (es) |
AU (1) | AU2020244803A1 (es) |
BR (1) | BR112021018801A2 (es) |
CA (1) | CA3134165A1 (es) |
CL (1) | CL2021002437A1 (es) |
CO (1) | CO2021014024A2 (es) |
IL (1) | IL286653A (es) |
MA (1) | MA55515A (es) |
MX (1) | MX2021011498A (es) |
SG (1) | SG11202108757XA (es) |
WO (1) | WO2020198268A1 (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2021025899A1 (en) * | 2019-08-02 | 2021-02-11 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer pharmaceutical compositions |
WO2023168427A1 (en) | 2022-03-03 | 2023-09-07 | Yale University | Compositions and methods for delivering therapeutic polynucleotides for exon skipping |
Family Cites Families (27)
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 |
DE3687030T2 (de) | 1985-03-15 | 1993-03-11 | James Summerton | Stereoregulare polynukleotiden bindende polymere. |
US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
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 |
EP0654077A4 (en) | 1992-07-17 | 1996-03-13 | Ribozyme Pharm Inc | PROCESS AND REAGENT FOR THE TREATMENT OF DISEASES IN ANIMALS. |
US5820873A (en) | 1994-09-30 | 1998-10-13 | The University Of British Columbia | Polyethylene glycol modified ceramide lipids and liposome uses thereof |
US5753613A (en) | 1994-09-30 | 1998-05-19 | Inex Pharmaceuticals Corporation | Compositions for the introduction of polyanionic materials into cells |
US5885613A (en) | 1994-09-30 | 1999-03-23 | The University Of British Columbia | Bilayer stabilizing components and their use in forming programmable fusogenic liposomes |
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 |
US7163695B2 (en) | 1999-12-29 | 2007-01-16 | Mixson A James | Histidine copolymer and methods for using same |
US8076476B2 (en) | 2007-11-15 | 2011-12-13 | Avi Biopharma, Inc. | Synthesis of morpholino oligomers using doubly protected guanine morpholino subunits |
US8299206B2 (en) | 2007-11-15 | 2012-10-30 | Avi Biopharma, Inc. | Method of synthesis of morpholino oligomers |
US20190262375A1 (en) * | 2016-06-30 | 2019-08-29 | Sarepta Therapeutics, Inc. | Exon skipping oligomers for muscular dystrophy |
SI3554554T1 (sl) * | 2016-12-19 | 2023-02-28 | Sarepta Therapeutics, Inc. | Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo |
US20210145852A1 (en) * | 2017-09-28 | 2021-05-20 | Sarepta Therapeutics, Inc. | Combination Therapies for Treating Muscular Dystrophy |
-
2020
- 2020-03-25 US US17/441,620 patent/US20220152086A1/en active Pending
- 2020-03-25 AU AU2020244803A patent/AU2020244803A1/en active Pending
- 2020-03-25 JP JP2021556752A patent/JP2022526763A/ja active Pending
- 2020-03-25 KR KR1020217034183A patent/KR20210145192A/ko unknown
- 2020-03-25 EP EP20719076.0A patent/EP3946376A1/en active Pending
- 2020-03-25 MX MX2021011498A patent/MX2021011498A/es unknown
- 2020-03-25 MA MA055515A patent/MA55515A/fr unknown
- 2020-03-25 SG SG11202108757X patent/SG11202108757XA/en unknown
- 2020-03-25 CN CN202080022966.9A patent/CN113660939A/zh active Pending
- 2020-03-25 WO PCT/US2020/024550 patent/WO2020198268A1/en unknown
- 2020-03-25 BR BR112021018801A patent/BR112021018801A2/pt unknown
- 2020-03-25 CA CA3134165A patent/CA3134165A1/en active Pending
-
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
Also Published As
Publication number | Publication date |
---|---|
IL286653A (en) | 2021-10-31 |
BR112021018801A2 (pt) | 2021-11-23 |
JP2022526763A (ja) | 2022-05-26 |
CL2021002437A1 (es) | 2022-05-06 |
EP3946376A1 (en) | 2022-02-09 |
US20220152086A1 (en) | 2022-05-19 |
AU2020244803A1 (en) | 2021-11-18 |
MA55515A (fr) | 2022-02-09 |
KR20210145192A (ko) | 2021-12-01 |
MX2021011498A (es) | 2022-01-04 |
CN113660939A (zh) | 2021-11-16 |
WO2020198268A1 (en) | 2020-10-01 |
SG11202108757XA (en) | 2021-10-28 |
CA3134165A1 (en) | 2020-10-01 |
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