HRP20230342T1 - Kationski lipid koji se može ionizirati za isporuku rna - Google Patents
Kationski lipid koji se može ionizirati za isporuku rna Download PDFInfo
- Publication number
- HRP20230342T1 HRP20230342T1 HRP20230342TT HRP20230342T HRP20230342T1 HR P20230342 T1 HRP20230342 T1 HR P20230342T1 HR P20230342T T HRP20230342T T HR P20230342TT HR P20230342 T HRP20230342 T HR P20230342T HR P20230342 T1 HRP20230342 T1 HR P20230342T1
- Authority
- HR
- Croatia
- Prior art keywords
- compound according
- consist
- branched alkyl
- cationic lipid
- carbon atoms
- Prior art date
Links
- -1 cationic lipid Chemical class 0.000 title 1
- 150000001875 compounds Chemical class 0.000 claims 10
- 125000000217 alkyl group Chemical group 0.000 claims 7
- 125000004432 carbon atom Chemical group C* 0.000 claims 4
- VGGSQFUCUMXWEO-UHFFFAOYSA-N Ethene Chemical compound C=C VGGSQFUCUMXWEO-UHFFFAOYSA-N 0.000 claims 2
- 239000005977 Ethylene Substances 0.000 claims 2
- 125000004805 propylene group Chemical group [H]C([H])([H])C([H])([*:1])C([H])([H])[*:2] 0.000 claims 2
- 125000001495 ethyl group Chemical group [H]C([H])([H])C([H])([H])* 0.000 claims 1
- 125000001449 isopropyl group Chemical group [H]C([H])([H])C([H])(*)C([H])([H])[H] 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 125000001570 methylene group Chemical group [H]C([H])([*:1])[*:2] 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C323/00—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups
- C07C323/50—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton
- C07C323/51—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
- C07C323/60—Thiols, sulfides, hydropolysulfides or polysulfides substituted by halogen, oxygen or nitrogen atoms, or by sulfur atoms not being part of thio groups containing thio groups and carboxyl groups bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton with the carbon atom of at least one of the carboxyl groups bound to nitrogen atoms
-
- 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
- A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0033—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being non-polymeric
-
- 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
- A61K9/0043—Nose
-
- 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
- A61K9/007—Pulmonary tract; Aromatherapy
- A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/12—Aerosols; Foams
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
-
- 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
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Liposomes
- A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
- A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C333/02—Monothiocarbamic acids; Derivatives thereof
- C07C333/04—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups bound to hydrogen atoms or to acyclic carbon atoms
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07C—ACYCLIC OR CARBOCYCLIC COMPOUNDS
- C07C333/00—Derivatives of thiocarbamic acids, i.e. compounds containing any of the groups, the nitrogen atom not being part of nitro or nitroso groups
- C07C333/02—Monothiocarbamic acids; Derivatives thereof
- C07C333/10—Monothiocarbamic acids; Derivatives thereof having nitrogen atoms of thiocarbamic groups being part of any of the groups, X being a hetero atom, Y being any atom, e.g., N-acyl-thiocarbamates
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J1/00—Normal steroids containing carbon, hydrogen, halogen or oxygen, not substituted in position 17 beta by a carbon atom, e.g. estrane, androstane
- C07J1/0003—Androstane derivatives
- C07J1/0007—Androstane derivatives not substituted in position 17
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J31/00—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring
- C07J31/006—Normal steroids containing one or more sulfur atoms not belonging to a hetero ring not covered by C07J31/003
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07J—STEROIDS
- C07J41/00—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring
- C07J41/0033—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005
- C07J41/0055—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the 17-beta position being substituted by an uninterrupted chain of at least three carbon atoms which may or may not be branched, e.g. cholane or cholestane derivatives, optionally cyclised, e.g. 17-beta-phenyl or 17-beta-furyl derivatives
-
- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- 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
Claims (10)
1. Spoj, naznačen time, da je predstavljen formulom III:
[image]
u kojoj
R1 se sastoji od razgranatog alkila s 12 do 20 ugljika,
R2 se sastoji od razgranatog alkila s 12 do 20 ugljika,
svaki od L1 i L2 se sastoji od veze ili ravnog alkila koji ima 1 do 3 atoma ugljika,
X se sastoji od S ili O,
L3 se sastoji od veze,
R3 se bira od etilena, n-propilena ili izopropilena, i
R4 i R5 su isti ili različiti, od kojih se svaki sastoji od ravnog ili razgranatog alkila koji se sastoji od 1 do 6 ugljika;
ili njegova farmaceutski prihvatljiva sol.
2. Spoj prema patentnom zahtjevu 1, naznačena time, da se X sastoji od S.
3. Spoj prema patentnom zahtjevu 1, naznačena time, da se L1 i L2 oba sastoje od ravnog alkila koji ima 1 do 3 atoma ugljika.
4. Spoj prema patentnom zahtjevu 1, naznačena time, da R3 je etilen.
5. Spoj prema patentnom zahtjevu 1, naznačena time, da R3 je n-propilen ili izopropilen.
6. Spoj prema patentnom zahtjevu 1, naznačena time, da se svaki od R4 i R5 sastoji od metila, etila ili izopropila.
7. Spoj prema patentnom zahtjevu 1, naznačena time, da se L1 i L2 oba sastoje od veze.
8. Spoj prema patentnom zahtjevu 1, naznačena time, da se L1 i L2 oba sastoje od metilena.
9. Spoj prema patentnom zahtjevu 1, naznačena time, da se R1 i R2 oba sastoje od razgranatog alkila od 19 ili 20 atoma ugljika.
10. Spoj prema patentnom zahtjevu 1, naznačena time, da se R1 i R2 oba sastoje od razgranatog alkila od 13 ili 14 atoma ugljika.
Applications Claiming Priority (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US14/546,105 US9593077B2 (en) | 2013-11-18 | 2014-11-18 | Ionizable cationic lipid for RNA delivery |
US14/707,796 US9567296B2 (en) | 2013-11-18 | 2015-05-08 | Ionizable cationic lipid for RNA delivery |
US14/707,876 US9365610B2 (en) | 2013-11-18 | 2015-05-08 | Asymmetric ionizable cationic lipid for RNA delivery |
PCT/US2015/030218 WO2016081029A1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
EP15724466.6A EP3221293B1 (en) | 2014-11-18 | 2015-05-11 | Ionizable cationic lipid for rna delivery |
Publications (1)
Publication Number | Publication Date |
---|---|
HRP20230342T1 true HRP20230342T1 (hr) | 2023-06-09 |
Family
ID=56014367
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HRP20230342TT HRP20230342T1 (hr) | 2014-11-18 | 2015-05-11 | Kationski lipid koji se može ionizirati za isporuku rna |
Country Status (20)
Country | Link |
---|---|
EP (1) | EP3221293B1 (hr) |
JP (1) | JP6637988B2 (hr) |
KR (1) | KR102380363B1 (hr) |
CN (1) | CN107207428A (hr) |
AU (1) | AU2015350561B2 (hr) |
CA (1) | CA2968060C (hr) |
DK (1) | DK3221293T3 (hr) |
ES (1) | ES2946110T3 (hr) |
FI (1) | FI3221293T3 (hr) |
HK (1) | HK1244479A1 (hr) |
HR (1) | HRP20230342T1 (hr) |
HU (1) | HUE062130T2 (hr) |
IL (1) | IL252291B (hr) |
LT (1) | LT3221293T (hr) |
NZ (1) | NZ732777A (hr) |
PL (1) | PL3221293T3 (hr) |
PT (1) | PT3221293T (hr) |
RS (1) | RS64078B1 (hr) |
SI (1) | SI3221293T1 (hr) |
WO (1) | WO2016081029A1 (hr) |
Families Citing this family (34)
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WO2015074085A1 (en) | 2013-11-18 | 2015-05-21 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for rna delivery |
US10975388B2 (en) | 2016-12-14 | 2021-04-13 | Ligandal, Inc. | Methods and compositions for nucleic acid and protein payload delivery |
US10526284B2 (en) | 2016-12-21 | 2020-01-07 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
WO2018119163A1 (en) * | 2016-12-21 | 2018-06-28 | Payne Joseph E | Ionizable cationic lipid for rna delivery |
US10383952B2 (en) * | 2016-12-21 | 2019-08-20 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
US20200046830A1 (en) * | 2017-03-30 | 2020-02-13 | The Government Of The United States, As Represented By The Secretary Of The Army | Nucleic acid vaccine composition comprising a lipid formulation, and method of increasing the potency of nucleic acid vaccines |
WO2019191780A1 (en) * | 2018-03-30 | 2019-10-03 | Arcturus Therapeutics, Inc. | Lipid particles for nucleic acid delivery |
CA3098262A1 (en) | 2018-04-25 | 2019-10-31 | Ethris Gmbh | Cryoprotective agents for particulate formulations |
KR20190127277A (ko) * | 2018-05-04 | 2019-11-13 | 주식회사 삼양바이오팜 | mRNA 전달용 고분자 나노입자 조성물 및 그 제조방법 |
EP3805198B1 (en) * | 2018-06-08 | 2023-09-20 | FUJIFILM Corporation | Compound, salt thereof and lipid particles |
EP3891274B1 (en) | 2018-12-06 | 2023-10-18 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
CN113840926A (zh) * | 2019-03-19 | 2021-12-24 | 阿克丘勒斯治疗公司 | 脂质包封的rna纳米颗粒的制备方法 |
JP7241869B2 (ja) | 2019-06-07 | 2023-03-17 | 富士フイルム株式会社 | 脂質組成物 |
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CA3158293A1 (en) | 2019-11-15 | 2021-05-20 | Fujifilm Corporation | Lipid composition |
CN115667530A (zh) | 2020-03-24 | 2023-01-31 | 世代生物公司 | 非病毒dna载体和其用于表达因子ix治疗剂的用途 |
CA3172591A1 (en) | 2020-03-24 | 2021-09-30 | Douglas Anthony KERR | Non-viral dna vectors and uses thereof for expressing gaucher therapeutics |
JP2023535632A (ja) | 2020-07-27 | 2023-08-18 | アンジャリウム バイオサイエンシズ エージー | Dna分子の組成物、その作製方法、及びその使用方法 |
KR20230065281A (ko) | 2020-09-13 | 2023-05-11 | 아크투루스 쎄라퓨틱스, 인크. | 큰 rna의 지질 나노입자 캡슐화 |
JP2024517427A (ja) | 2021-04-20 | 2024-04-22 | アンジャリウム バイオサイエンシズ エージー | アミロ-α-1,6-グルコシダーゼ、4-α-グルカノトランスフェラーゼをコードするDNA分子の組成物、その作製の方法、及びその使用の方法 |
AU2022264509A1 (en) | 2021-04-27 | 2023-12-14 | Generation Bio Co. | Non-viral dna vectors expressing therapeutic antibodies and uses thereof |
WO2022232286A1 (en) | 2021-04-27 | 2022-11-03 | Generation Bio Co. | Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof |
IL309505A (en) | 2021-09-03 | 2024-02-01 | CureVac SE | Lipid nanoparticles for nucleic acid delivery |
WO2023073228A1 (en) | 2021-10-29 | 2023-05-04 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
WO2023081526A1 (en) | 2021-11-08 | 2023-05-11 | Orna Therapeutics, Inc. | Lipid nanoparticle compositions for delivering circular polynucleotides |
WO2023135273A2 (en) | 2022-01-14 | 2023-07-20 | Anjarium Biosciences Ag | Compositions of dna molecules encoding factor viii, methods of making thereof, and methods of use thereof |
WO2023144330A1 (en) | 2022-01-28 | 2023-08-03 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
CN114213295B (zh) * | 2022-02-22 | 2022-05-20 | 中国科学院基础医学与肿瘤研究所(筹) | 一种阳离子化合物、制备方法及其复合物和用途 |
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WO2023239756A1 (en) | 2022-06-07 | 2023-12-14 | Generation Bio Co. | Lipid nanoparticle compositions and uses thereof |
WO2024040222A1 (en) | 2022-08-19 | 2024-02-22 | Generation Bio Co. | Cleavable closed-ended dna (cedna) and methods of use thereof |
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-
2015
- 2015-05-11 WO PCT/US2015/030218 patent/WO2016081029A1/en active Application Filing
- 2015-05-11 DK DK15724466.6T patent/DK3221293T3/da active
- 2015-05-11 AU AU2015350561A patent/AU2015350561B2/en active Active
- 2015-05-11 HU HUE15724466A patent/HUE062130T2/hu unknown
- 2015-05-11 EP EP15724466.6A patent/EP3221293B1/en active Active
- 2015-05-11 PL PL15724466.6T patent/PL3221293T3/pl unknown
- 2015-05-11 ES ES15724466T patent/ES2946110T3/es active Active
- 2015-05-11 KR KR1020177016742A patent/KR102380363B1/ko active IP Right Grant
- 2015-05-11 CA CA2968060A patent/CA2968060C/en active Active
- 2015-05-11 NZ NZ732777A patent/NZ732777A/en unknown
- 2015-05-11 FI FIEP15724466.6T patent/FI3221293T3/fi active
- 2015-05-11 HR HRP20230342TT patent/HRP20230342T1/hr unknown
- 2015-05-11 SI SI201531935T patent/SI3221293T1/sl unknown
- 2015-05-11 JP JP2017545520A patent/JP6637988B2/ja active Active
- 2015-05-11 PT PT157244666T patent/PT3221293T/pt unknown
- 2015-05-11 CN CN201580073670.9A patent/CN107207428A/zh active Pending
- 2015-05-11 RS RS20230231A patent/RS64078B1/sr unknown
- 2015-05-11 LT LTEPPCT/US2015/030218T patent/LT3221293T/lt unknown
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2017
- 2017-05-15 IL IL252291A patent/IL252291B/en unknown
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2018
- 2018-03-21 HK HK18103903.0A patent/HK1244479A1/zh unknown
Also Published As
Publication number | Publication date |
---|---|
CA2968060A1 (en) | 2016-05-26 |
AU2015350561B2 (en) | 2020-01-30 |
SI3221293T1 (sl) | 2023-05-31 |
CA2968060C (en) | 2023-06-20 |
WO2016081029A1 (en) | 2016-05-26 |
IL252291A0 (en) | 2017-07-31 |
HUE062130T2 (hu) | 2023-09-28 |
JP2017535610A (ja) | 2017-11-30 |
KR20170095241A (ko) | 2017-08-22 |
RS64078B1 (sr) | 2023-04-28 |
FI3221293T3 (fi) | 2023-04-05 |
LT3221293T (lt) | 2023-03-27 |
CN107207428A (zh) | 2017-09-26 |
JP6637988B2 (ja) | 2020-01-29 |
IL252291B (en) | 2021-08-31 |
NZ732777A (en) | 2023-06-30 |
EP3221293A1 (en) | 2017-09-27 |
AU2015350561A1 (en) | 2017-07-06 |
PL3221293T3 (pl) | 2023-07-10 |
ES2946110T3 (es) | 2023-07-12 |
HK1244479A1 (zh) | 2018-08-10 |
KR102380363B1 (ko) | 2022-03-29 |
PT3221293T (pt) | 2023-03-16 |
EP3221293B1 (en) | 2023-03-01 |
DK3221293T3 (da) | 2023-04-03 |
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