CA3173126A1 - Nucleic acid sequences encapsulated in lipid nanoparticles - Google Patents

Nucleic acid sequences encapsulated in lipid nanoparticles Download PDF

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Publication number
CA3173126A1
CA3173126A1 CA3173126A CA3173126A CA3173126A1 CA 3173126 A1 CA3173126 A1 CA 3173126A1 CA 3173126 A CA3173126 A CA 3173126A CA 3173126 A CA3173126 A CA 3173126A CA 3173126 A1 CA3173126 A1 CA 3173126A1
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CA
Canada
Prior art keywords
lipid
aspects
pharmaceutically acceptable
lipid nanoparticle
cedna
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
CA3173126A
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English (en)
French (fr)
Inventor
Matthew G. Stanton
Birte Nolting
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.)
Generation Bio Co
Original Assignee
Generation Bio Co
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Filing date
Publication date
Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of CA3173126A1 publication Critical patent/CA3173126A1/en
Pending legal-status Critical Current

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50Microcapsules 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
    • A61K9/51Nanocapsules; Nanoparticles
    • A61K9/5107Excipients; Inactive ingredients
    • A61K9/5123Organic compounds, e.g. fats, sugars
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic 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
    • C07D211/08Heterocyclic 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
    • C07D211/18Heterocyclic 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
    • C07D211/20Heterocyclic 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
    • C07D211/22Heterocyclic 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 oxygen atoms
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Obesity (AREA)
  • Biochemistry (AREA)
  • Optics & Photonics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nanotechnology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3173126A 2020-03-27 2021-03-26 Nucleic acid sequences encapsulated in lipid nanoparticles Pending CA3173126A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063000990P 2020-03-27 2020-03-27
US63/000,990 2020-03-27
PCT/US2021/024413 WO2021195529A2 (en) 2020-03-27 2021-03-26 Novel lipids and nanoparticle compositions thereof

Publications (1)

Publication Number Publication Date
CA3173126A1 true CA3173126A1 (en) 2021-09-30

Family

ID=77890624

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3173126A Pending CA3173126A1 (en) 2020-03-27 2021-03-26 Nucleic acid sequences encapsulated in lipid nanoparticles

Country Status (11)

Country Link
US (3) US12534435B2 (enExample)
EP (1) EP4125821A4 (enExample)
JP (2) JP7746280B2 (enExample)
KR (1) KR20220160647A (enExample)
CN (1) CN115916163A (enExample)
AU (1) AU2021241696A1 (enExample)
BR (1) BR112022019369A2 (enExample)
CA (1) CA3173126A1 (enExample)
IL (1) IL296763B2 (enExample)
MX (1) MX2022011988A (enExample)
WO (1) WO2021195529A2 (enExample)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
WO2024192528A1 (en) * 2023-03-22 2024-09-26 Nanovation Therapeutics Inc. Ionizable anionic lipids

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JP7746280B2 (ja) 2020-03-27 2025-09-30 ジェネレーション バイオ カンパニー 新規脂質及びそれらのナノ粒子組成物
AU2021307952A1 (en) * 2020-07-17 2023-03-02 Generation Bio Co. Methods for encapsulating polynucleotides into reduced sizes of Lipid nanoparticles and novel formulation thereof
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WO2025043146A2 (en) * 2023-08-24 2025-02-27 Molecular Axiom Inc. Lipid nanoparticle compositions and methods of using same
CN121263400A (zh) * 2023-12-15 2026-01-02 厦门赛诺邦格生物科技股份有限公司 一种含二硫键的可降解阳离子脂质及其应用
CN117964577B (zh) * 2024-03-29 2024-06-21 天津全和诚生物技术有限公司 阳离子脂质化合物、其制备方法、包含其的组合物及应用
WO2026003582A2 (en) 2024-06-27 2026-01-02 Axelyf ehf. Lipids and lipid nanoparticles
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024192528A1 (en) * 2023-03-22 2024-09-26 Nanovation Therapeutics Inc. Ionizable anionic lipids

Also Published As

Publication number Publication date
JP2025181861A (ja) 2025-12-11
US20230159459A1 (en) 2023-05-25
IL296763A (en) 2022-11-01
JP2023518985A (ja) 2023-05-09
IL296763B1 (en) 2025-12-01
BR112022019369A2 (pt) 2022-12-13
MX2022011988A (es) 2023-01-04
EP4125821A2 (en) 2023-02-08
WO2021195529A3 (en) 2021-11-18
KR20220160647A (ko) 2022-12-06
AU2021241696A1 (en) 2022-12-01
JP7746280B2 (ja) 2025-09-30
EP4125821A4 (en) 2024-06-26
CN115916163A (zh) 2023-04-04
US20250361208A1 (en) 2025-11-27
US12252470B2 (en) 2025-03-18
US12534435B2 (en) 2026-01-27
IL296763B2 (en) 2026-04-01
WO2021195529A2 (en) 2021-09-30
US20240294474A1 (en) 2024-09-05

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