HRP20221155T1 - Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju - Google Patents
Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju Download PDFInfo
- Publication number
- HRP20221155T1 HRP20221155T1 HRP20221155TT HRP20221155T HRP20221155T1 HR P20221155 T1 HRP20221155 T1 HR P20221155T1 HR P20221155T T HRP20221155T T HR P20221155TT HR P20221155 T HRP20221155 T HR P20221155T HR P20221155 T1 HRP20221155 T1 HR P20221155T1
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- Croatia
- Prior art keywords
- antisense oligomer
- pharmaceutically acceptable
- oligomer conjugate
- acceptable salt
- conjugate
- Prior art date
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- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 claims 15
- 150000003839 salts Chemical class 0.000 claims 10
- 102000001039 Dystrophin Human genes 0.000 claims 7
- 108010069091 Dystrophin Proteins 0.000 claims 7
- 239000000825 pharmaceutical preparation Substances 0.000 claims 7
- 206010064571 Gene mutation Diseases 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 230000008094 contradictory effect Effects 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 238000002560 therapeutic procedure Methods 0.000 claims 1
Classifications
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- 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
- 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
- A61K47/54—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 organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or 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
- A61K47/62—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
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Genetics & Genomics (AREA)
- Psychology (AREA)
- Neurosurgery (AREA)
- Immunology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (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)
Claims (13)
1. Protusmisleni oligomerni konjugat formule (IV):
[image]
ili njegova farmaceutski prihvatljiva sol.
2. Konjugat protusmislenog oligomera prema zahtjevu 1, naznačen time što je protusmisleni oligomer formule (IVA):
[image]
3. Farmaceutski pripravak, naznačen time što sadrži konjugat protusmislenog oligomera prema zahtjevu 1 ili 2, ili njegovu farmaceutski prihvatljivu sol, i farmaceutski prihvatljiv nosač.
4. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol prema zahtjevu 1 ili 2, naznačen time što se koristi u terapiji.
5. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol prema zahtjevu 1 ili 2, ili farmaceutski pripravak prema zahtjevu 3, za upotrebu u liječenju Duchenneove mišićne distrofije (DMD) kod subjekta kojem je to potrebno, pri čemu subjekt ima mutaciju gena distrofina koji je podložan preskakanju egzona 53.
6. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol prema zahtjevu 1 ili 2, ili farmaceutski pripravak prema zahtjevu 3, za upotrebu u obnavljanju okvira za čitanje mRNA za induciranje proizvodnje distrofina kod subjekta koji ima mutaciju gena za distrofin koja je podložna preskakanju egzona 53.
7. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili 6, naznačen time što upotreba obuhvaća davanje konjugata protusmislenog oligomera jednom tjedno.
8. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili 6 naznačen time što upotreba obuhvaća primjenu konjugata protusmislenog oligomera dva puta tjedno.
9. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili 6 naznačen time što upotreba obuhvaća primjenu konjugata protusmislenog oligomera svaki treći tjedan.
10. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili 6 naznačen time što upotreba obuhvaća primjenu konjugata protusmislenog oligomera jednom mjesečno.
11. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema bilo kojem od zahtjeva 4-10, naznačen time što upotreba obuhvaća primjenu konjugata protusmislenog oligomera u dozi odabranoj od (i) oko 30 mg/kg; (ii) oko 40 mg/kg; (iii) oko 60 mg/kg; (iv) oko 80 mg/kg; i (v) oko 160 mg/kg.
12. Farmaceutski pripravak prema zahtjevu 3, naznačen time što se koristi za isključivanje egzona 53 iz distrofina pre-mRNA tijekom obrade mRNA kod subjekta koji ima mutaciju gena za distrofin koji je podložan preskakanju egzona 53.
13. Farmaceutski pripravak prema zahtjevu 3, naznačen time što se koristi za vezanje egzona 53 distrofina pre-mRNA kod subjekta koji ima mutaciju gena za distrofin koja je podložna preskakanju egzona 53.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662436223P | 2016-12-19 | 2016-12-19 | |
US201762443484P | 2017-01-06 | 2017-01-06 | |
US201762479178P | 2017-03-30 | 2017-03-30 | |
US201762562146P | 2017-09-22 | 2017-09-22 | |
EP17826645.8A EP3554552B1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
PCT/US2017/066509 WO2018118662A1 (en) | 2016-12-19 | 2017-12-14 | Exon skipping oligomer conjugates for muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20221155T1 true HRP20221155T1 (hr) | 2022-12-09 |
Family
ID=60953960
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20221155TT HRP20221155T1 (hr) | 2016-12-19 | 2017-12-14 | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
Country Status (25)
Country | Link |
---|---|
US (2) | US11000600B2 (hr) |
EP (2) | EP3554552B1 (hr) |
JP (3) | JP7160807B2 (hr) |
KR (2) | KR20240006057A (hr) |
CN (1) | CN110636866A (hr) |
AU (1) | AU2017382773A1 (hr) |
BR (1) | BR112019012664A2 (hr) |
CA (1) | CA3047010A1 (hr) |
CO (1) | CO2019007399A2 (hr) |
DK (1) | DK3554552T3 (hr) |
ES (1) | ES2928486T3 (hr) |
HR (1) | HRP20221155T1 (hr) |
HU (1) | HUE059905T2 (hr) |
IL (2) | IL297528A (hr) |
LT (1) | LT3554552T (hr) |
MD (1) | MD3554552T2 (hr) |
MX (1) | MX2019006879A (hr) |
NZ (1) | NZ755438A (hr) |
PL (1) | PL3554552T3 (hr) |
PT (1) | PT3554552T (hr) |
RS (1) | RS63621B1 (hr) |
SA (1) | SA519402226B1 (hr) |
SG (1) | SG10202100557UA (hr) |
TW (2) | TW202332470A (hr) |
WO (1) | WO2018118662A1 (hr) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
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TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
US10875880B2 (en) | 2016-05-24 | 2020-12-29 | Sarepta Therapeutics, Inc. | Processes for preparing oligomers |
US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
CA3047010A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
LT3554554T (lt) | 2016-12-19 | 2022-11-25 | Sarepta Therapeutics, Inc. | Egzoną praleidžiantys oligomero konjugatai nuo raumenų distrofijos |
EA201991450A1 (ru) * | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
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EP3987029A1 (en) * | 2019-06-19 | 2022-04-27 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
WO2021072034A1 (en) * | 2019-10-09 | 2021-04-15 | Children's Medical Center Corporation | Methods and compositions for treating ataxia telangiectasia |
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BR112023027298A2 (pt) | 2021-06-23 | 2024-03-12 | Nat Center Neurology & Psychiatry | Combinação de oligômeros antissenso |
JPWO2023282346A1 (hr) | 2021-07-08 | 2023-01-12 | ||
IL310003A (en) | 2021-07-08 | 2024-03-01 | Nippon Shinyaku Co Ltd | A substance that lowers nephrotoxicity |
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