CA3213299A1 - Compositions and methods for targeted delivery to cells - Google Patents

Compositions and methods for targeted delivery to cells Download PDF

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Publication number
CA3213299A1
CA3213299A1 CA3213299A CA3213299A CA3213299A1 CA 3213299 A1 CA3213299 A1 CA 3213299A1 CA 3213299 A CA3213299 A CA 3213299A CA 3213299 A CA3213299 A CA 3213299A CA 3213299 A1 CA3213299 A1 CA 3213299A1
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CA
Canada
Prior art keywords
lipid
lung
subject
cell
therapeutic agent
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
CA3213299A
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English (en)
French (fr)
Inventor
Mirko HENNIG
Vladimir Kharitonov
Brandon Wustman
Jackson EBY
Rumpa BHATTACHARJEE
Daniel SIEGWART
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.)
University of Texas System
Recode Therapeutics Inc
Original Assignee
University of Texas System
Recode Therapeutics Inc
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 University of Texas System, Recode Therapeutics Inc filed Critical University of Texas System
Publication of CA3213299A1 publication Critical patent/CA3213299A1/en
Pending legal-status Critical Current

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    • A61K9/0073—Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
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    • 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
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    • 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)
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    • 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

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  • 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)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
CA3213299A 2021-03-22 2022-03-22 Compositions and methods for targeted delivery to cells Pending CA3213299A1 (en)

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
CA3213299A1 true CA3213299A1 (en) 2022-09-29

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Family Applications (1)

Application Number Title Priority Date Filing Date
CA3213299A Pending CA3213299A1 (en) 2021-03-22 2022-03-22 Compositions and methods for targeted delivery to cells

Country Status (10)

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

Cited By (1)

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CN116983280A (zh) * 2023-07-19 2023-11-03 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

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CN108271360B (zh) * 2015-09-14 2023-01-24 得克萨斯州大学系统董事会 亲脂阳离子树枝状聚合物及其用途
MX2023011104A (es) * 2021-03-22 2024-03-01 Recode Therapeutics Inc Composiciones de polinucleotidos, formulaciones relacionadas y metodos de uso de las mismas.
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2022204219A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Compositions and methods for targeted delivery to cells
EP4313001A4 (en) * 2021-03-22 2025-11-26 Univ Texas Compositions and methods for targeted administration to cells
KR20230175196A (ko) 2021-03-23 2023-12-29 리코드 테라퓨틱스, 인크. 폴리뉴클레오티드 조성물, 관련된 제제, 및 이의 사용 방법
MX2023011230A (es) * 2021-03-23 2023-12-06 Recode Therapeutics Inc Composiciones y metodos para administracion sistemica dirigida a celulas.
WO2022226344A1 (en) * 2021-04-22 2022-10-27 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
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