HRP20220943T1 - Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju - Google Patents
Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju Download PDFInfo
- Publication number
- HRP20220943T1 HRP20220943T1 HRP20220943TT HRP20220943T HRP20220943T1 HR P20220943 T1 HRP20220943 T1 HR P20220943T1 HR P20220943T T HRP20220943T T HR P20220943TT HR P20220943 T HRP20220943 T HR P20220943T HR P20220943 T1 HRP20220943 T1 HR P20220943T1
- Authority
- HR
- Croatia
- Prior art keywords
- antisense oligomer
- pharmaceutically acceptable
- acceptable salt
- oligomer conjugate
- exon
- Prior art date
Links
- 201000006938 muscular dystrophy Diseases 0.000 title 1
- 230000000692 anti-sense effect Effects 0.000 claims 13
- 150000003839 salts Chemical class 0.000 claims 9
- 102000001039 Dystrophin Human genes 0.000 claims 7
- 108010069091 Dystrophin Proteins 0.000 claims 7
- 239000000825 pharmaceutical preparation Substances 0.000 claims 5
- 206010064571 Gene mutation Diseases 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 239000008194 pharmaceutical composition Substances 0.000 claims 4
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
-
- 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
- A61K47/50—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
-
- 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
-
- 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
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
Claims (12)
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 51.
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 51.
7. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili zahtjevu 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 zahtjevu 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 zahtjevu 6, naznačen time što upotreba obuhvaća davanje konjugata protusmislenog oligomera svaki treći tjedan.
10. Konjugat protusmislenog oligomera ili njegova farmaceutski prihvatljiva sol, ili farmaceutski pripravak, za upotrebu prema zahtjevu 5 ili zahtjevu 6, naznačen time što upotreba obuhvaća davanje konjugata protusmislenog oligomera jednom mjesečno.
11. Farmaceutski pripravak prema zahtjevu 3, naznačen time što se koristi za isključivanje egzona 51 iz pre-mRNA distrofina tijekom obrade mRNA kod subjekta koji ima mutaciju gena za distrofin koji je podložan preskakanju egzona 51.
12. Farmaceutski pripravak prema zahtjevu 3, naznačen time što se koristi za vezanje egzona 51 pre-mRNA distrofina kod subjekta koji ima mutaciju gena distrofina koja je podložna preskakanju egzona 51.
Applications Claiming Priority (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662436182P | 2016-12-19 | 2016-12-19 | |
US201762443476P | 2017-01-06 | 2017-01-06 | |
US201762479173P | 2017-03-30 | 2017-03-30 | |
US201762562080P | 2017-09-22 | 2017-09-22 | |
EP17832401.8A EP3554553B1 (en) | 2016-12-19 | 2017-12-13 | Exon skipping oligomer conjugates for muscular dystrophy |
PCT/US2017/066222 WO2018118599A1 (en) | 2016-12-19 | 2017-12-13 | Exon skipping oligomer conjugates for muscular dystrophy |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20220943T1 true HRP20220943T1 (hr) | 2022-10-28 |
Family
ID=61006315
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20220943TT HRP20220943T1 (hr) | 2016-12-19 | 2017-12-13 | Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju |
Country Status (27)
Country | Link |
---|---|
US (4) | US10888578B2 (hr) |
EP (1) | EP3554553B1 (hr) |
JP (3) | JP7118063B2 (hr) |
KR (2) | KR102552428B1 (hr) |
CN (1) | CN110290812B (hr) |
AU (1) | AU2017382723A1 (hr) |
BR (1) | BR112019012647A2 (hr) |
CA (1) | CA3046793A1 (hr) |
CO (1) | CO2019007397A2 (hr) |
DK (1) | DK3554553T3 (hr) |
ES (1) | ES2927079T3 (hr) |
HR (1) | HRP20220943T1 (hr) |
HU (1) | HUE059843T2 (hr) |
IL (2) | IL267244B (hr) |
LT (1) | LT3554553T (hr) |
MD (1) | MD3554553T2 (hr) |
MX (1) | MX2019006882A (hr) |
MY (1) | MY195801A (hr) |
NZ (1) | NZ755416A (hr) |
PL (1) | PL3554553T3 (hr) |
PT (1) | PT3554553T (hr) |
RS (1) | RS63610B1 (hr) |
SA (1) | SA519402154B1 (hr) |
SG (1) | SG10202012839TA (hr) |
TW (1) | TWI760402B (hr) |
WO (1) | WO2018118599A1 (hr) |
ZA (1) | ZA201904092B (hr) |
Families Citing this family (18)
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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 |
CN109563114B (zh) | 2016-05-24 | 2022-08-12 | 萨勒普塔医疗公司 | 用于制备寡聚物的方法 |
CN109311920B (zh) | 2016-05-24 | 2021-11-09 | 萨勒普塔医疗公司 | 制备磷酸二酰胺吗啉代寡聚物的方法 |
BR112019012647A2 (pt) * | 2016-12-19 | 2019-11-19 | Sarepta Therapeutics Inc | conjugados de oligômero de salto de éxon para distrofia muscular |
WO2018118627A1 (en) | 2016-12-19 | 2018-06-28 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
AU2017382773A1 (en) * | 2016-12-19 | 2019-08-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
GB201711809D0 (en) * | 2017-07-21 | 2017-09-06 | Governors Of The Univ Of Alberta | Antisense oligonucleotide |
GB201821269D0 (en) | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
WO2020214763A1 (en) * | 2019-04-18 | 2020-10-22 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
WO2020257489A1 (en) * | 2019-06-19 | 2020-12-24 | Sarepta Therapeutics, Inc. | Methods for treating muscular dystrophy |
KR20220122673A (ko) | 2019-12-26 | 2022-09-02 | 니뽄 신야쿠 가부시키가이샤 | 엑손 50의 스키핑을 유도하는 안티센스 핵산 |
JPWO2021172498A1 (hr) | 2020-02-28 | 2021-09-02 | ||
EP4330395A1 (en) | 2021-04-30 | 2024-03-06 | Sarepta Therapeutics, Inc. | Treatment methods for muscular dystrophy |
TW202307208A (zh) | 2021-06-23 | 2023-02-16 | 日商日本新藥股份有限公司 | 反義寡聚物之組合 |
AU2022306820A1 (en) | 2021-07-08 | 2024-01-04 | Nippon Shinyaku Co., Ltd. | Nephrotoxicity reducing agent |
WO2023282346A1 (ja) | 2021-07-08 | 2023-01-12 | 日本新薬株式会社 | 析出抑制剤 |
TW202308664A (zh) | 2021-07-08 | 2023-03-01 | 日商日本新藥股份有限公司 | 腎毒性減輕劑 |
WO2023178230A1 (en) | 2022-03-17 | 2023-09-21 | Sarepta Therapeutics, Inc. | Phosphorodiamidate morpholino oligomer conjugates |
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