KR20240005692A - 세포로의 표적화 전달을 위한 조성물 및 방법 - Google Patents

세포로의 표적화 전달을 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20240005692A
KR20240005692A KR1020237035962A KR20237035962A KR20240005692A KR 20240005692 A KR20240005692 A KR 20240005692A KR 1020237035962 A KR1020237035962 A KR 1020237035962A KR 20237035962 A KR20237035962 A KR 20237035962A KR 20240005692 A KR20240005692 A KR 20240005692A
Authority
KR
South Korea
Prior art keywords
lipid
cells
lung
subject
sort
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
KR1020237035962A
Other languages
English (en)
Korean (ko)
Inventor
미르코 헨니그
블라디미르 카리토노프
브랜든 우스트만
잭슨 에비
룸파 바타차르지
다니엘 시그와트
Original Assignee
리코드 테라퓨틱스, 인크.
더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템
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 리코드 테라퓨틱스, 인크., 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 filed Critical 리코드 테라퓨틱스, 인크.
Publication of KR20240005692A publication Critical patent/KR20240005692A/ko
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K9/0012—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/14—Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/28—Steroids, e.g. cholesterol, bile acids or glycyrrhetinic acid
    • 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
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • 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
    • A61K47/543—Lipids, e.g. triglycerides; Polyamines, e.g. spermine or spermidine
    • A61K47/544—Phospholipids
    • 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
    • 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
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K9/0078—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy for inhalation via a nebulizer such as a jet nebulizer, ultrasonic nebulizer, e.g. in the form of aqueous drug solutions or dispersions
    • 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—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • 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
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/113—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
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
    • C12Y306/04002—Dynein ATPase (3.6.4.2)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dispersion Chemistry (AREA)
  • Microbiology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Plant Pathology (AREA)
  • Otolaryngology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nanotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Optics & Photonics (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
KR1020237035962A 2021-03-22 2022-03-22 세포로의 표적화 전달을 위한 조성물 및 방법 Pending KR20240005692A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163164523P 2021-03-22 2021-03-22
US63/164,523 2021-03-22
US202163229497P 2021-08-04 2021-08-04
US63/229,497 2021-08-04
US202263305426P 2022-02-01 2022-02-01
US63/305,426 2022-02-01
PCT/US2022/021442 WO2022204219A1 (en) 2021-03-22 2022-03-22 Compositions and methods for targeted delivery to cells

Publications (1)

Publication Number Publication Date
KR20240005692A true KR20240005692A (ko) 2024-01-12

Family

ID=83396024

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020237035962A Pending KR20240005692A (ko) 2021-03-22 2022-03-22 세포로의 표적화 전달을 위한 조성물 및 방법

Country Status (10)

Country Link
US (10) US20240207178A1 (https=)
EP (1) EP4312987A4 (https=)
JP (1) JP2024511463A (https=)
KR (1) KR20240005692A (https=)
AU (1) AU2022246065A1 (https=)
BR (1) BR112023019229A2 (https=)
CA (1) CA3213299A1 (https=)
IL (1) IL305782A (https=)
MX (1) MX2023011227A (https=)
WO (1) WO2022204219A1 (https=)

Families Citing this family (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017048789A1 (en) * 2015-09-14 2017-03-23 The Board Of Regents Of The University Of Texas System Lipocationic dendrimers and uses thereof
WO2022204215A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204043A1 (en) * 2021-03-22 2022-09-29 The Board Of Regents Of The University Of Texas System Compositions and methods for targeted delivery to cells
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) * 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
IL305998A (en) * 2021-03-23 2023-11-01 Recode Therapeutics Inc Formulations and methods for targeted systemic delivery to cells
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
US20240207442A1 (en) * 2021-04-22 2024-06-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
EP4630057A1 (en) 2022-12-08 2025-10-15 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
CN116211831A (zh) * 2022-12-14 2023-06-06 北京剂泰医药科技有限公司 脂质基质局部注射制剂
EP4385523A1 (en) * 2022-12-14 2024-06-19 Beijing Jitai Pharmaceutical Technology Co., Ltd. Lipid-based topical injection formulations
CN120418427A (zh) 2022-12-23 2025-08-01 因特利亚治疗公司 用于基因组编辑的系统和方法
AU2024279278A1 (en) 2023-05-31 2025-12-18 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
CN116983280B (zh) * 2023-07-19 2025-09-30 南通大学 一种具有肺选择性的脂质纳米颗粒及其制备方法与应用
WO2025075396A1 (ko) * 2023-10-06 2025-04-10 한국과학기술연구원 타겟 핵산 검출물질이 담지된 지질 나노입자 및 이를 이용한 막융합 기반의 타겟 핵산 검출 방법
TW202525812A (zh) * 2023-10-24 2025-07-01 南韓商綠十字股份有限公司 可離子化脂質及其用途
WO2025117816A1 (en) * 2023-12-01 2025-06-05 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12364773B2 (en) * 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
US12553042B2 (en) * 2023-12-01 2026-02-17 Recode Therapeutics, Inc. Method for quantifying an amount of capped messenger RNA
WO2025137646A1 (en) * 2023-12-22 2025-06-26 Recode Therapeutics, Inc. Gene editing methods and compositions for treating cystic fibrosis
CN117942406B (zh) * 2024-01-19 2025-07-11 晟迪生物医药(苏州)有限公司 预制载体在制备用于免疫细胞和干细胞的体外基因递送的产品中的应用
CN121015605A (zh) * 2024-01-19 2025-11-28 晟迪生物医药(苏州)有限公司 含有可电离脂质的肺部递送系统、其制备方法和应用
WO2025240355A1 (en) * 2024-05-14 2025-11-20 Recode Therapeutics, Inc. Lipid nanoparticles for extrahepatic delivery

Family Cites Families (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5976567A (en) * 1995-06-07 1999-11-02 Inex Pharmaceuticals Corp. Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
DK0941066T3 (da) 1996-08-26 2004-02-23 Transgene Sa Kationisk lipid-nukleinsyre-komplekser
US5962429A (en) 1996-11-22 1999-10-05 University Of Iowa Complexes of adenovirus with cationic molecules
ES2340499T3 (es) 2001-06-05 2010-06-04 Curevac Gmbh Arnm de antigeno tumoral estabilizado con un contenido de g/c aumentado.
PL2578685T3 (pl) 2005-08-23 2020-01-31 The Trustees Of The University Of Pennsylvania Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
KR101766408B1 (ko) 2009-06-10 2017-08-10 알닐람 파마슈티칼스 인코포레이티드 향상된 지질 조성물
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
AU2012236099A1 (en) 2011-03-31 2013-10-03 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
AU2013237874B2 (en) 2012-03-29 2018-01-18 Translate Bio, Inc. Lipid-derived neutral nanoparticles
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
EP3083556B1 (en) 2013-12-19 2019-12-25 Novartis AG Lipids and lipid compositions for the delivery of active agents
HUE060907T2 (hu) 2014-06-25 2023-04-28 Acuitas Therapeutics Inc Új lipidek és lipid nanorészecske formulációk nukleinsavak bevitelére
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
WO2016094342A1 (en) 2014-12-08 2016-06-16 The Board Of Regents Of The University Of Texas System Lipocationic polymers and uses thereof
EP3247363A4 (en) 2015-01-21 2018-10-03 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
EP3247398A4 (en) 2015-01-23 2018-09-26 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016205691A1 (en) 2015-06-19 2016-12-22 Massachusetts Institute Of Technology Alkenyl substituted 2,5-piperazinediones and their use in compositions for delivering an agent to a subject or cell
PT3313829T (pt) 2015-06-29 2024-07-08 Acuitas Therapeutics Inc Formulações de lípidos e de nanopartículas lipídicas para a administração de ácidos nucleicos
GB2560250A (en) 2015-06-30 2018-09-05 Ethris Gmbh ATP-Binding cassette family coding polyribonucleotides and formulations thereof
US20180200186A1 (en) 2015-07-09 2018-07-19 Insmed Incorporated Compositions and methods for treating lung diseases and lung injury
WO2017048789A1 (en) 2015-09-14 2017-03-23 The Board Of Regents Of The University Of Texas System Lipocationic dendrimers and uses thereof
HRP20230209T1 (hr) 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
WO2017117528A1 (en) 2015-12-30 2017-07-06 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
LT3436077T (lt) 2016-03-30 2025-06-25 Intellia Therapeutics, Inc. Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams
WO2017180917A2 (en) * 2016-04-13 2017-10-19 Modernatx, Inc. Lipid compositions and their uses for intratumoral polynucleotide delivery
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
CN109414408B (zh) 2016-05-16 2022-03-29 得克萨斯州大学系统董事会 阳离子磺酰胺氨基脂质和两亲性两性离子氨基脂质
CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
CA3103528A1 (en) 2018-06-19 2019-12-26 The Board Of Regents Of The University Of Texas System Lipid nanoparticle compositions for delivery of mrna and long nucleic acids
WO2020051223A1 (en) 2018-09-04 2020-03-12 The Board Of Regents Of The University Of Texas System Compositions and methods for organ specific delivery of nucleic acids
EP3846857A4 (en) * 2018-09-04 2022-10-12 The Board Of Regents Of The University Of Texas System COMPOSITIONS AND METHODS FOR ORGAN-SPECIFIC DELIVERY OF NUCLEIC ACIDS
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
JP2022516356A (ja) 2019-01-07 2022-02-25 トランスレイト バイオ， インコーポレイテッド 原発性線毛機能不全症の治療のための組成物および方法
WO2021011504A1 (en) * 2019-07-12 2021-01-21 Duke University Nanoparticle systems for targeted delivery of crispr/cas13 and methods of using same
JP2023512519A (ja) 2020-01-31 2023-03-27 ベス イスラエル ディーコネス メディカル センター インコーポレイテッド コロナウイルス感染症を予防および処置するための組成物および方法－ｓａｒｓ－ｃｏｖ－２ワクチン
CN115427021A (zh) * 2020-02-25 2022-12-02 翻译生物公司 制备负载mrna的脂质纳米颗粒的改进方法
WO2021205032A1 (en) 2020-04-09 2021-10-14 Technische Universität München Targeted delivery of an inhibitor of mir-21 to macrophages for the treatment of pulmonary fibrosis
US20240293317A1 (en) 2020-04-20 2024-09-05 Board Of Regents, The University Of Texas System Lipid nanoparticle (lnp) delivery systems and uses thereof
CA3176844A1 (en) * 2020-05-01 2021-11-04 Carlos G. PEREZ-GARCIA Nucleic acids and methods of treatment for cystic fibrosis
EP4146680A1 (en) 2020-05-07 2023-03-15 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
MA71659A (fr) 2020-08-06 2025-05-30 Modernatx, Inc. Compositions pour l'administration de molécules de charge utile à l'épithélium des voies respiratoires
JP7849049B2 (ja) * 2020-08-21 2026-04-21 ザ ボード オブ リージェンツ オブ ザ ユニヴァーシティー オブ テキサス システム 機能性のイオン化可能なリン脂質
IL305021A (en) 2021-02-08 2023-10-01 Univ Texas Unsaturated Dendrimer Compositions, Related Formulations, and Methods of Using Them
US20240123087A1 (en) 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204053A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204215A1 (en) 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) 2021-03-22 2024-03-13 リコード セラピューティクス，インク． 細胞への標的送達のための組成物および方法
IL305998A (en) 2021-03-23 2023-11-01 Recode Therapeutics Inc Formulations and methods for targeted systemic delivery to cells
CA3213107A1 (en) 2021-03-23 2022-09-29 Mirko HENNIG Polynucleotide compositions, related formulations, and methods of use thereof
US20250281580A1 (en) 2021-04-05 2025-09-11 The Board Of Regents Of The University Of Texas System Compositions, methods and uses for treating cystic fibrosis and related disorders
US20240207442A1 (en) 2021-04-22 2024-06-27 The Board Of Regents Of The University Of Texas System All-in-one dendrimer-based lipid nanoparticles enable precise hdr-mediated gene editing in vivo
JP2024520834A (ja) 2021-06-09 2024-05-24 レコード・セラピューティクス・インコーポレイテッド ポリヌクレオチド組成物、関連する製剤及びその使用方法

Also Published As

Publication number Publication date
CA3213299A1 (en) 2022-09-29
WO2022204219A1 (en) 2022-09-29
US20240374512A1 (en) 2024-11-14
US20240374514A1 (en) 2024-11-14
IL305782A (en) 2023-11-01
US20230338411A1 (en) 2023-10-26
US20240374513A1 (en) 2024-11-14
BR112023019229A2 (pt) 2023-11-28
AU2022246065A1 (en) 2023-10-12
US12121610B2 (en) 2024-10-22
US20230293429A1 (en) 2023-09-21
EP4312987A1 (en) 2024-02-07
US20240207178A1 (en) 2024-06-27
US20240390272A1 (en) 2024-11-28
MX2023011227A (es) 2023-12-06
US20230302138A1 (en) 2023-09-28
JP2024511463A (ja) 2024-03-13
US20240374511A1 (en) 2024-11-14
EP4312987A4 (en) 2025-02-26
US20240390271A1 (en) 2024-11-28

Similar Documents

Publication Publication Date Title
US12121610B2 (en) Compositions and methods for targeted delivery to cells
JP7815196B2 (ja) 核酸を臓器特異的送達するための組成物および方法
US11766408B2 (en) Compositions and methods for organ specific delivery of nucleic acids
US20240216515A1 (en) Compositions and methods for targeted delivery to cells
AU2022425201A1 (en) Compositions and methods for targeted delivery to cells
US20240197641A1 (en) Compositions and methods for targeted systemic delivery to cells
CN117835967A (zh) 用于靶向递送至细胞的组合物和方法
CN117750948A (zh) 用于靶向全身性递送至细胞的组合物和方法
HK40108980A (zh) 用於靶向递送至细胞的组合物和方法
HK40111533A (zh) 用於靶向全身性递送至细胞的组合物和方法

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20231019

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20250321

Comment text: Request for Examination of Application