JP7348064B2 - 分子を経膜送達するための化合物及び方法 - Google Patents

分子を経膜送達するための化合物及び方法 Download PDF

Info

Publication number
JP7348064B2
JP7348064B2 JP2019536976A JP2019536976A JP7348064B2 JP 7348064 B2 JP7348064 B2 JP 7348064B2 JP 2019536976 A JP2019536976 A JP 2019536976A JP 2019536976 A JP2019536976 A JP 2019536976A JP 7348064 B2 JP7348064 B2 JP 7348064B2
Authority
JP
Japan
Prior art keywords
conjugate
mmol
moiety
sirna
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2019536976A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020514299A (ja
JP2020514299A5 (enExample
Inventor
ジヴ、イーラン
デュブロヴスキー、ヨセフ
グリムベルグ、ハギット
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Aposense Ltd
Original Assignee
Aposense Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Aposense Ltd filed Critical Aposense Ltd
Publication of JP2020514299A publication Critical patent/JP2020514299A/ja
Publication of JP2020514299A5 publication Critical patent/JP2020514299A5/ja
Application granted granted Critical
Publication of JP7348064B2 publication Critical patent/JP7348064B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • 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
    • A61K47/554—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 the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C07J41/0072—Normal steroids containing one or more nitrogen atoms not belonging to a hetero ring not covered by C07J41/0005 the A ring of the steroid being aromatic
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J51/00—Normal steroids with unmodified cyclopenta(a)hydrophenanthrene skeleton not provided for in groups C07J1/00 - C07J43/00
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Botany (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
JP2019536976A 2017-01-09 2018-01-09 分子を経膜送達するための化合物及び方法 Active JP7348064B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762443822P 2017-01-09 2017-01-09
US62/443,822 2017-01-09
PCT/IL2018/050031 WO2018127927A1 (en) 2017-01-09 2018-01-09 Compounds and methods for trans-membrane delivery of molecules

Publications (3)

Publication Number Publication Date
JP2020514299A JP2020514299A (ja) 2020-05-21
JP2020514299A5 JP2020514299A5 (enExample) 2021-02-18
JP7348064B2 true JP7348064B2 (ja) 2023-09-20

Family

ID=62789169

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2019536976A Active JP7348064B2 (ja) 2017-01-09 2018-01-09 分子を経膜送達するための化合物及び方法

Country Status (10)

Country Link
US (1) US11230710B2 (enExample)
EP (1) EP3565603B1 (enExample)
JP (1) JP7348064B2 (enExample)
KR (1) KR20190104392A (enExample)
CN (1) CN110402152B (enExample)
AU (1) AU2018205898B2 (enExample)
CA (1) CA3049640A1 (enExample)
EA (1) EA201991661A1 (enExample)
IL (1) IL267919B2 (enExample)
WO (1) WO2018127927A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024026212A (ja) * 2018-01-01 2024-02-28 アポセンス リミテッド 分子の膜貫通送達のための化合物および方法

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11318206B2 (en) 2014-03-28 2022-05-03 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules
KR20190104392A (ko) 2017-01-09 2019-09-09 아포센스 엘티디. 분자의 막투과 전달을 위한 화합물 및 방법
US10195286B2 (en) * 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
WO2020044349A1 (en) * 2018-08-30 2020-03-05 Aposense Ltd. Compounds, cojugates and compositions for use in the methods for trans-membrane delivery of molecules
WO2023022055A1 (ja) * 2021-08-17 2023-02-23 国立大学法人東海国立大学機構 修飾ポリヌクレオチド
WO2025096244A1 (en) * 2023-10-30 2025-05-08 Olfera Corporation Olfactory delivery scaffolds and methods for making and using same

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160106855A1 (en) 2014-03-28 2016-04-21 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2252617A1 (en) 1996-05-01 1997-11-06 Imarx Pharmaceutical Corp. Methods for delivering compounds into a cell
US6090800A (en) 1997-05-06 2000-07-18 Imarx Pharmaceutical Corp. Lipid soluble steroid prodrugs
AU6974798A (en) 1997-05-06 1998-11-27 Imarx Pharmaceutical Corp. Novel prodrugs comprising fluorinated amphiphiles
FR2846969A1 (fr) 2002-11-08 2004-05-14 Salles Jean Pierre Nouveaux vecteurs moleculaires amphiphiles fluorocarbones a usage biomedical et medical
SE527131C2 (sv) 2004-02-13 2005-12-27 Innoventus Project Ab Steroider för cancerbehandling
US8101666B2 (en) * 2004-07-08 2012-01-24 Aposense Ltd. Methods and compounds for detection of medical disorders
EP2266622A3 (en) 2004-08-26 2012-06-20 Piramal Life Sciences Limited Prodrugs containing novel bio-cleavable linkers
AP2007003968A0 (en) 2004-10-01 2007-04-30 Prometic Biosciences Inc Medium-chain length fatty alcohols as stimulators of hematopoiesis
BRPI0520032A2 (pt) 2005-02-18 2009-04-14 Angiochem Inc moléculas para transportar um composto através da barreira hematoencefálica
US8252755B2 (en) 2006-09-22 2012-08-28 Dharmacon, Inc. Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
WO2009082607A2 (en) 2007-12-04 2009-07-02 Alnylam Pharmaceuticals, Inc. Targeting lipids
AU2009246327B8 (en) 2008-05-13 2013-10-03 Phaserx, Inc. Diblock copolymers and polynucleotide complexes thereof for delivery into cells
WO2009155335A2 (en) 2008-06-19 2009-12-23 University Of Maryland, Baltimore Conjugates of 19f mr imaging tracers and chemotherapeutic agents for drug quantification and drug dose individualization
US8796443B2 (en) 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
US20120035362A1 (en) 2010-08-03 2012-02-09 Berry And Associates, Inc. Phosphoramidite derivatives of folic acid
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
SI3401400T1 (sl) 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
US20150376645A1 (en) 2012-05-30 2015-12-31 Baylor College Of Medicine Supercoiled minivectors as a tool for dna repair, alteration and replacement
US9662402B2 (en) 2012-10-16 2017-05-30 Endocyte, Inc. Drug delivery conjugates containing unnatural amino acids and methods for using
WO2014127052A1 (en) 2013-02-13 2014-08-21 Zondlo Neal Perfluoro-tert-butyl hydroxyproline
CN110003066B (zh) 2013-11-18 2021-09-03 阿克丘勒斯治疗公司 用于rna递送的可电离的阳离子脂质
US9889202B2 (en) 2014-03-28 2018-02-13 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US9993563B2 (en) 2014-03-28 2018-06-12 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules
US20160016855A1 (en) * 2014-07-21 2016-01-21 Benxin Wu Fabrication of carbon nanotube-nonoxide structural ceramic nanocomposites through laser sintering
KR20190104392A (ko) 2017-01-09 2019-09-09 아포센스 엘티디. 분자의 막투과 전달을 위한 화합물 및 방법
US10195286B2 (en) 2017-07-04 2019-02-05 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160106855A1 (en) 2014-03-28 2016-04-21 Aposense Ltd. Compounds and methods for trans-membrane delivery of molecules

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024026212A (ja) * 2018-01-01 2024-02-28 アポセンス リミテッド 分子の膜貫通送達のための化合物および方法
US12337036B2 (en) 2018-01-01 2025-06-24 Aposense Ltd Compounds and methods for trans-membrane delivery of molecules

Also Published As

Publication number Publication date
IL267919B1 (en) 2023-11-01
AU2018205898B2 (en) 2020-12-10
IL267919A (en) 2019-09-26
US11230710B2 (en) 2022-01-25
JP2020514299A (ja) 2020-05-21
CN110402152A (zh) 2019-11-01
US20200392490A1 (en) 2020-12-17
IL267919B2 (en) 2024-03-01
EP3565603A1 (en) 2019-11-13
EP3565603A4 (en) 2020-09-09
CA3049640A1 (en) 2018-07-12
WO2018127927A1 (en) 2018-07-12
CN110402152B (zh) 2024-03-19
AU2018205898A1 (en) 2019-08-22
KR20190104392A (ko) 2019-09-09
EA201991661A1 (ru) 2019-12-30
EP3565603B1 (en) 2022-12-07

Similar Documents

Publication Publication Date Title
JP7348064B2 (ja) 分子を経膜送達するための化合物及び方法
JP6669719B2 (ja) 分子の膜貫通送達のための化合物および方法
CN108135924B (zh) 用于跨膜递送分子的化合物和方法
JP7191869B2 (ja) 分子を経膜送達するための化合物及び方法
JP2024026212A (ja) 分子の膜貫通送達のための化合物および方法
WO2020044349A1 (en) Compounds, cojugates and compositions for use in the methods for trans-membrane delivery of molecules
HK40034155A (en) Compounds and methods for trans-membrane delivery of molecules
HK40027749A (en) Compounds and methods for trans-membrane delivery of molecules
US20190125884A1 (en) Compounds and methods for trans-membrane delivery of molecules
NZ724764B2 (en) Oligonucleotide compositions and methods of making the same
NZ724764A (en) Oligonucleotide compositions and methods of making the same

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210106

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210106

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20211018

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211026

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220125

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220325

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220404

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220628

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220928

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221122

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221226

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230228

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230623

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20230711

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20230815

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20230907

R150 Certificate of patent or registration of utility model

Ref document number: 7348064

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150