CN119278031A - 包含多价阴离子例如无机聚磷酸根的核酸组合物以及用于制备、储存和使用该核酸组合物的方法 - Google Patents

包含多价阴离子例如无机聚磷酸根的核酸组合物以及用于制备、储存和使用该核酸组合物的方法 Download PDF

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Publication number
CN119278031A
CN119278031A CN202380032503.4A CN202380032503A CN119278031A CN 119278031 A CN119278031 A CN 119278031A CN 202380032503 A CN202380032503 A CN 202380032503A CN 119278031 A CN119278031 A CN 119278031A
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composition
lipid
nucleic acid
steroid
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Chinese (zh)
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斯特芬·潘茨纳
戈兰·马丁·哈姆拉兹
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Debiotech SA
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Debiotech SA
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    • 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
    • A61K9/5115Inorganic compounds
    • 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
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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
    • 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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/39Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
    • 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
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/51Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53DNA (RNA) vaccination
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/555Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511Organic adjuvants
    • A61K2039/55555Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Inorganic Chemistry (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Dispersion Chemistry (AREA)
  • Manufacturing & Machinery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202380032503.4A 2022-04-05 2023-04-05 包含多价阴离子例如无机聚磷酸根的核酸组合物以及用于制备、储存和使用该核酸组合物的方法 Pending CN119278031A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EPPCT/EP2022/059023 2022-04-05
PCT/EP2022/059023 WO2023193892A1 (en) 2022-04-05 2022-04-05 Nucleic acid compositions comprising an inorganic polyphosphate and methods for preparing, storing and using the same
PCT/EP2023/059066 WO2023194508A1 (en) 2022-04-05 2023-04-05 Nucleic acid compositions comprising a multivalent anion, such as an inorganic polyphosphate, and methods for preparing, storing and using the same

Publications (1)

Publication Number Publication Date
CN119278031A true CN119278031A (zh) 2025-01-07

Family

ID=81307329

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380032503.4A Pending CN119278031A (zh) 2022-04-05 2023-04-05 包含多价阴离子例如无机聚磷酸根的核酸组合物以及用于制备、储存和使用该核酸组合物的方法

Country Status (8)

Country Link
US (1) US20250222132A1 (https=)
EP (1) EP4504158A1 (https=)
JP (1) JP2025514649A (https=)
CN (1) CN119278031A (https=)
AU (1) AU2023248681A1 (https=)
CA (1) CA3246753A1 (https=)
IL (1) IL315673A (https=)
WO (2) WO2023193892A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2023413766A1 (en) 2022-12-23 2025-05-29 Biontech Delivery Technologies Gmbh Composition
WO2025133105A1 (en) 2023-12-21 2025-06-26 Biontech Delivery Technologies Gmbh Compositions and methods

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FR2686899B1 (fr) 1992-01-31 1995-09-01 Rhone Poulenc Rorer Sa Nouveaux polypeptides biologiquement actifs, leur preparation et compositions pharmaceutiques les contenant.
US20050287153A1 (en) 2002-06-28 2005-12-29 Genentech, Inc. Serum albumin binding peptides for tumor targeting
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
US7176278B2 (en) 2001-08-30 2007-02-13 Biorexis Technology, Inc. Modified transferrin fusion proteins
US7696320B2 (en) 2004-08-24 2010-04-13 Domantis Limited Ligands that have binding specificity for VEGF and/or EGFR and methods of use therefor
EP1900753B1 (en) 2002-11-08 2017-08-09 Ablynx N.V. Method of administering therapeutic polypeptides, and polypeptides therefor
HUE027902T2 (en) 2004-02-09 2016-11-28 Human Genome Sciences Inc Corp Service Company Albumin fusion proteins
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
US20070269422A1 (en) 2006-05-17 2007-11-22 Ablynx N.V. Serum albumin binding proteins with long half-lives
CN101511868B (zh) 2006-07-24 2013-03-06 比奥雷克西斯制药公司 毒蜥外泌肽融合蛋白
AU2007293614A1 (en) 2006-09-08 2008-03-13 Ablynx N.V. Serum albumin binding proteins with long half-lives
WO2009083804A2 (en) 2007-12-27 2009-07-09 Novartis Ag Improved fibronectin-based binding molecules and their use
EA201001734A1 (ru) 2008-05-02 2011-10-31 Новартис Аг Улучшенные связывающие молекулы на основе фибронектина и их применение
EP2493871B1 (en) 2009-10-30 2014-09-03 Domain Therapeutics Novel oxime derivatives and their use as allosteric modulators of metabotropic glutamate receptors
EP2556087A1 (en) 2010-04-09 2013-02-13 Novozymes Biopharma DK A/S Albumin derivatives and variants
EP2558491B1 (en) 2010-04-13 2018-07-04 Bristol-Myers Squibb Company Fibronectin based scaffold domain proteins that bind pcsk9
EP2758436B1 (en) 2011-09-23 2019-06-12 Universität Stuttgart Serum half-life extension using immunoglobulin binding domains
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WO2016005004A1 (en) 2014-07-11 2016-01-14 Biontech Rna Pharmaceuticals Gmbh Stabilization of poly(a) sequence encoding dna sequences
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HRP20230209T1 (hr) * 2015-10-28 2023-04-14 Acuitas Therapeutics Inc. Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
SI3445850T1 (sl) 2016-04-22 2021-12-31 BioNTech SE Postopki za zagotavljanje enoverižne RNA
EP3468610A1 (en) * 2016-06-09 2019-04-17 OPKO Biologics Ltd. Long-acting oxyntomodulin formulation and methods of producing and administering same
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KR20200143391A (ko) 2018-03-15 2020-12-23 비온테크 알엔에이 파마슈티컬스 게엠베하 5'-캡-트리뉴클레오티드- 또는 고차의 올리고뉴클레오티드 화합물 및 이들의 rna 안정화, 단백질 발현 및 치료에서의 사용
US12419965B2 (en) * 2018-10-01 2025-09-23 BioNTech SE RNA particles comprising polysarcosine
CN115335084A (zh) * 2020-03-25 2022-11-11 生物技术公司 包含聚肌氨酸的rna颗粒

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Publication number Publication date
WO2023193892A1 (en) 2023-10-12
US20250222132A1 (en) 2025-07-10
AU2023248681A1 (en) 2024-09-26
CA3246753A1 (en) 2023-10-12
WO2023194508A1 (en) 2023-10-12
EP4504158A1 (en) 2025-02-12
IL315673A (en) 2024-11-01
JP2025514649A (ja) 2025-05-09

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Inventor after: Steffen Panzner

Inventor after: Golan Martin Hamlet

Inventor after: Martin kishberg

Inventor after: Christian Reinsch

Inventor after: Patrick Kredwig

Inventor before: Steffen Panzner

Inventor before: Golan Martin Hamlet