JP2023527747A - 新規脂質及びそれらのナノ粒子組成物 - Google Patents

新規脂質及びそれらのナノ粒子組成物 Download PDF

Info

Publication number
JP2023527747A
JP2023527747A JP2022570302A JP2022570302A JP2023527747A JP 2023527747 A JP2023527747 A JP 2023527747A JP 2022570302 A JP2022570302 A JP 2022570302A JP 2022570302 A JP2022570302 A JP 2022570302A JP 2023527747 A JP2023527747 A JP 2023527747A
Authority
JP
Japan
Prior art keywords
lipid
cedna
itr
ionizable
disease
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.)
Pending
Application number
JP2022570302A
Other languages
English (en)
Japanese (ja)
Other versions
JPWO2021236479A5 (https=
JP2023527747A5 (https=
Inventor
マシュー ジー． スタントン，
バート ノルティング，
Original Assignee
ジェネレーション バイオ カンパニー
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 ジェネレーション バイオ カンパニー filed Critical ジェネレーション バイオ カンパニー
Publication of JP2023527747A publication Critical patent/JP2023527747A/ja
Publication of JPWO2021236479A5 publication Critical patent/JPWO2021236479A5/ja
Publication of JP2023527747A5 publication Critical patent/JP2023527747A5/ja
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61K48/0041—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 polymeric
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • C07C323/23—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 nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton
    • C07C323/24—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 nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton
    • C07C323/25—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 nitrogen atoms, not being part of nitro or nitroso groups, bound to the same carbon skeleton having the sulfur atoms of the thio groups bound to acyclic carbon atoms of the carbon skeleton the carbon skeleton being acyclic and saturated
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/08—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms
    • C07D211/18—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms
    • C07D211/20—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms
    • C07D211/24—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hydrocarbon or substituted hydrocarbon radicals directly attached to ring carbon atoms with substituted hydrocarbon radicals attached to ring carbon atoms with hydrocarbon radicals, substituted by singly bound oxygen or sulphur atoms by sulfur atoms to which a second hetero atom is attached
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
JP2022570302A 2020-05-18 2021-05-17 新規脂質及びそれらのナノ粒子組成物 Pending JP2023527747A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063026479P 2020-05-18 2020-05-18
US63/026,479 2020-05-18
PCT/US2021/032676 WO2021236479A2 (en) 2020-05-18 2021-05-17 Novel lipids and nanoparticle compositions thereof

Publications (3)

Publication Number Publication Date
JP2023527747A true JP2023527747A (ja) 2023-06-30
JPWO2021236479A5 JPWO2021236479A5 (https=) 2024-05-24
JP2023527747A5 JP2023527747A5 (https=) 2024-05-24

Family

ID=78707483

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022570302A Pending JP2023527747A (ja) 2020-05-18 2021-05-17 新規脂質及びそれらのナノ粒子組成物

Country Status (9)

Country Link
US (1) US20230181764A1 (https=)
EP (1) EP4153144A4 (https=)
JP (1) JP2023527747A (https=)
CN (1) CN115968280A (https=)
AU (1) AU2021274481A1 (https=)
CA (1) CA3175957A1 (https=)
IL (1) IL298241A (https=)
MX (1) MX2022014484A (https=)
WO (1) WO2021236479A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023534043A (ja) * 2020-07-17 2023-08-07 ジェネレーション バイオ カンパニー ポリヌクレオチドを縮小サイズの脂質ナノ粒子に封入する方法及びその新規製剤

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
CN114716355B (zh) * 2022-04-02 2023-09-05 华南理工大学 一种脂质化合物、包含其的组合物及应用
CN121532216A (zh) * 2023-04-18 2026-02-13 赛仁生物技术公司 腺相关病毒载体组合物和使用方法
US12311033B2 (en) 2023-05-31 2025-05-27 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN117925729B (zh) * 2024-01-19 2025-06-10 晟迪生物医药(苏州)有限公司 基于空白脂质纳米颗粒的转染试剂、其制备方法和应用
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013073480A1 (ja) * 2011-11-18 2013-05-23 日油株式会社 細胞内動態を改善したカチオン性脂質
WO2016121942A1 (ja) * 2015-01-30 2016-08-04 日油株式会社 カチオン性脂質
WO2017061150A1 (ja) * 2015-10-08 2017-04-13 日油株式会社 Ｏ／ｗ型エマルション
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED
WO2019188867A1 (ja) * 2018-03-27 2019-10-03 日油株式会社 細胞内動態を改善した新規カチオン性脂質
JP2023507465A (ja) * 2019-12-20 2023-02-22 キュアバック エスイー 核酸を送達するための脂質ナノ粒子

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0610479D0 (en) * 2006-05-26 2006-07-05 Ge Healthcare Bio Sciences Ab A method for generating metal chelating affinity ligands
WO2018237369A2 (en) * 2017-06-23 2018-12-27 Vical Incorporated Lipid nanoparticle (lnp)-mediated delivery of a crispr-expressing plasmid dna for treating chronic hepatitis b virus infection
JP7700101B2 (ja) * 2019-09-06 2025-06-30 ジェネレーション バイオ カンパニー 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
WO2021060440A1 (ja) * 2019-09-26 2021-04-01 日油株式会社 脂質ナノ粒子の凍結乾燥組成物
AU2020385378A1 (en) * 2019-11-22 2022-04-07 Generation Bio Co. Ionizable lipids and nanoparticle compositions thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013073480A1 (ja) * 2011-11-18 2013-05-23 日油株式会社 細胞内動態を改善したカチオン性脂質
WO2016121942A1 (ja) * 2015-01-30 2016-08-04 日油株式会社 カチオン性脂質
WO2017061150A1 (ja) * 2015-10-08 2017-04-13 日油株式会社 Ｏ／ｗ型エマルション
WO2019051255A1 (en) * 2017-09-08 2019-03-14 Generation Bio Co. CLOSED-ENDED DNA (CEDNA) MODIFIED
WO2019188867A1 (ja) * 2018-03-27 2019-10-03 日油株式会社 細胞内動態を改善した新規カチオン性脂質
JP2023507465A (ja) * 2019-12-20 2023-02-22 キュアバック エスイー 核酸を送達するための脂質ナノ粒子

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
AKITA, HIDETAKA ET AL.: ""A Neutral Envelope‐Type Nanoparticle Containing pH‐Responsive and SS‐Cleavable Lipid‐Like Mate", ADVANCED HEALTHCARE MATERIALS, vol. 2, no. 8, JPN6025015118, 2013, pages 1120 - 1125, ISSN: 0005710244 *
AKITA, HIDETAKA ET AL.: ""Molecular Tuning of a Vitamin E-Scaffold pH-Sensitive and Reductive Cleavable Lipid-like Material", ACS BIOMATERIALS SCIENCE & ENGINEERING, vol. 1, no. 9, JPN7025001656, 2015, pages 834 - 844, ISSN: 0005710243 *
JAIN, PRIYESH ET AL.: ""Zwitterionic Hydrogels Based on a Degradable Disulfide Carboxybetaine Cross-Linker"", 2019年, vol. 35, no. 5, JPN6025015115, pages 1864 - 1871, ISSN: 0005710241 *
TANAKA, HIROKI ET AL.: ""In Vivo Introduction of mRNA Encapsulated in Lipid Nanoparticles to Brain Neuronal Cells and Astro", MOLECULAR PHARMACEUTICS, vol. 15, no. 5, JPN6025015120, 2018, pages 2060 - 2067, ISSN: 0005710240 *
TANAKA, HIROKI ET AL.: ""Neutral biodegradable lipid-envelope-type nanoparticle using vitamin A-Scaffold for nuclear target", BIOMATERIALS, vol. 35, no. 5, JPN6025015117, 2014, pages 1755 - 1761, ISSN: 0005710245 *
TANAKA, HIROKI ET AL.: ""Temperature and pH sensitivity of a stabilized self-nanoemulsion formed using an ionizable lipid-l", COLLOIDS AND SURFACES B: BIOINTERFACES, vol. 151, JPN6025015116, 2017, pages 95 - 101, ISSN: 0005710246 *
TOGASHI, RYOHEI ET AL.: ""A hepatic pDNA delivery system based on an intracellular environment sensitive vitamin E-scaffold", JOURNAL OF CONTROLLED RELEASE, vol. 279, JPN6025015119, 2018, pages 262 - 270, ISSN: 0005710242 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023534043A (ja) * 2020-07-17 2023-08-07 ジェネレーション バイオ カンパニー ポリヌクレオチドを縮小サイズの脂質ナノ粒子に封入する方法及びその新規製剤

Also Published As

Publication number Publication date
US20230181764A1 (en) 2023-06-15
WO2021236479A2 (en) 2021-11-25
EP4153144A2 (en) 2023-03-29
MX2022014484A (es) 2022-12-13
AU2021274481A1 (en) 2022-11-03
IL298241A (en) 2023-01-01
CN115968280A (zh) 2023-04-14
EP4153144A4 (en) 2024-07-03
WO2021236479A8 (en) 2022-02-24
CA3175957A1 (en) 2021-11-25
WO2021236479A3 (en) 2021-12-23

Similar Documents

Publication Publication Date Title
US12252470B2 (en) Lipids and nanoparticle compositions thereof
JP7700101B2 (ja) 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
JP2023502576A (ja) イオン化可能な脂質およびそれらのナノ粒子組成物
US20240293574A1 (en) Cationic lipids and compositions thereof
KR20230052895A (ko) 폴리뉴클레오타이드를 감소된 크기의 지질 나노입자로 캡슐화하는 방법 및 신규한 지질 나노입자 제형
JP2023527747A (ja) 新規脂質及びそれらのナノ粒子組成物
KR20230172570A (ko) 양이온성 지질 및 이의 조성물

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240516

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240516

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250415

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20250417

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250714

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250723

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251017

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260119

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260415