AU2018206485B2 - Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment - Google Patents

Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment Download PDF

Info

Publication number
AU2018206485B2
AU2018206485B2 AU2018206485A AU2018206485A AU2018206485B2 AU 2018206485 B2 AU2018206485 B2 AU 2018206485B2 AU 2018206485 A AU2018206485 A AU 2018206485A AU 2018206485 A AU2018206485 A AU 2018206485A AU 2018206485 B2 AU2018206485 B2 AU 2018206485B2
Authority
AU
Australia
Prior art keywords
cell
lnp
target cell
protein
expression
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.)
Active
Application number
AU2018206485A
Other languages
English (en)
Other versions
AU2018206485A1 (en
Inventor
Matthew Rein Scholz
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.)
OISIN BIOTECHNOLOGIES
Original Assignee
OISIN BIOTECHNOLOGIES
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 OISIN BIOTECHNOLOGIES filed Critical OISIN BIOTECHNOLOGIES
Publication of AU2018206485A1 publication Critical patent/AU2018206485A1/en
Application granted granted Critical
Publication of AU2018206485B2 publication Critical patent/AU2018206485B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/16Antivirals for RNA viruses for influenza or rhinoviruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/20Antivirals for DNA viruses
    • A61P31/22Antivirals for DNA viruses for herpes viruses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • 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
    • C12N2720/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsRNA viruses
    • C12N2720/00011Details
    • C12N2720/12011Reoviridae
    • C12N2720/12022New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2720/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsRNA viruses
    • C12N2720/00011Details
    • C12N2720/12011Reoviridae
    • C12N2720/12041Use of virus, viral particle or viral elements as a vector
    • C12N2720/12043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/007Vectors comprising a special translation-regulating system cell or tissue specific
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Dispersion Chemistry (AREA)
  • Pulmonology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2018206485A 2017-01-09 2018-01-09 Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment Active AU2018206485B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762444360P 2017-01-09 2017-01-09
US62/444,360 2017-01-09
PCT/US2018/013033 WO2018129563A1 (en) 2017-01-09 2018-01-09 Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment

Publications (2)

Publication Number Publication Date
AU2018206485A1 AU2018206485A1 (en) 2019-07-11
AU2018206485B2 true AU2018206485B2 (en) 2024-12-05

Family

ID=62790808

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2018206485A Active AU2018206485B2 (en) 2017-01-09 2018-01-09 Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment

Country Status (7)

Country Link
US (2) US11525146B2 (enExample)
EP (1) EP3565607A4 (enExample)
JP (1) JP2020503390A (enExample)
CN (1) CN110678208B (enExample)
AU (1) AU2018206485B2 (enExample)
CA (1) CA3047140A1 (enExample)
WO (1) WO2018129563A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112015024605A2 (pt) * 2013-03-24 2017-07-18 Oisin Biotechnologies sistemas e métodos para a produção visada de proteína terapêutica dentro de célula alvo
EP3235908A1 (en) 2016-04-21 2017-10-25 Ecole Normale Superieure De Lyon Methods for selectively modulating the activity of distinct subtypes of cells
CN110678208B (zh) 2017-01-09 2024-04-05 奥依信生物技术公司 膜融合脂质纳米颗粒和制造方法以及用于产生治疗性蛋白质和用于治疗的用途
CN112312918A (zh) * 2018-04-18 2021-02-02 奥依信生物技术公司 促融合脂质纳米粒和制备其的方法以及其用于靶细胞特异性产生治疗蛋白和治疗与靶细胞有关的疾病、病况或障碍的用途
KR20210043574A (ko) 2018-07-09 2021-04-21 플래그쉽 파이어니어링 이노베이션스 브이, 인크. 푸소좀 조성물 및 이의 용도
CN113631718A (zh) 2018-11-14 2021-11-09 旗舰先锋创新V股份有限公司 用于特定隔室货物递送的组合物和方法
WO2020102503A2 (en) * 2018-11-14 2020-05-22 Flagship Pioneering Innovations V, Inc. Fusosome compositions for t cell delivery
WO2020163408A2 (en) * 2019-02-04 2020-08-13 Oisin Biotechnologies, Inc. Fusogenic lipid nanoparticles for the target cell-specific production of rapamycin inducible therapeutic proteins
CN112521511B (zh) 2020-12-07 2023-03-14 中山大学 一种含EB病毒gB蛋白的自组装纳米颗粒及其制备方法与应用
CA3216353A1 (en) 2021-04-08 2022-10-13 Sana Biotechnology, Inc. Cd8-specific antibody constructs and compositions thereof

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69132032T2 (de) 1990-11-23 2000-12-21 Peptech Ltd., North Ryde Verzögerung, verhinderung und/oder rückgängigmachen von zellalterung
US5869337A (en) 1993-02-12 1999-02-09 President And Fellows Of Harvard College Regulated transcription of targeted genes and other biological events
DE69841002D1 (de) 1997-05-14 2009-09-03 Univ British Columbia Hochwirksame verkapselung von nukleinsäuren in lipidvesikeln
CA2303201A1 (en) * 1997-08-20 1999-02-25 Dnavec Research Inc. Vectors for treating cancer
JP2002512002A (ja) 1997-11-07 2002-04-23 ダルハウジー、ユニバーシティー 新規なレオウイルス由来タンパク質、このタンパク質をコードする核酸、およびその使用
US6899870B1 (en) 1998-03-11 2005-05-31 Board Of Regents, The University Of Texas System Induction of apoptic or cytotoxic gene expression by adenoviral mediated gene codelivery
CA2352486A1 (en) 1998-11-25 2000-06-02 Genetica, Inc. Methods and reagents for increasing proliferative capacity and preventing replicative senescence
CA2325088A1 (en) 2000-12-01 2002-06-01 Fusogenix Inc. Novel membrane fusion proteins derived from poikilothermic reovirus
AU2002345658A1 (en) 2001-06-13 2002-12-23 Eastern Virginia Medical School Methods for targeted expression of therapeutic nucleic acid
CN100361710C (zh) 2004-06-07 2008-01-16 成都康弘生物科技有限公司 肿瘤细胞专一表达免疫调节因子gm-csf的溶瘤性腺病毒重组体的构建及其应用
JPWO2006043354A1 (ja) * 2004-10-20 2008-05-22 独立行政法人放射線医学総合研究所 組込型の低線量放射線誘導性ベクター
US20100299770A1 (en) 2007-06-12 2010-11-25 Selkirk Stephen M Targeted cell death
BRPI0913047A2 (pt) 2008-05-23 2013-03-05 Siwa Corp anticorpo monoclonal humanizado, seu uso, bem como aparelho e mÉtodo in vitro para promover processos regenerativos em uma cultura de tecido ou cultura de cÉlula
DK3338765T3 (en) 2009-12-01 2019-03-04 Translate Bio Inc STEROID DERIVATIVE FOR THE SUPPLY OF MRNA IN HUMANGENETIC DISEASES
US9089520B2 (en) * 2010-05-21 2015-07-28 Baylor College Of Medicine Methods for inducing selective apoptosis
EP2600871A2 (en) 2010-08-04 2013-06-12 The Ohio State University Methods for impairing the p53/hdm2 auto-regulatory loop in multiple myeloma development using mir-192, mir-194 and mir-215
WO2012040825A1 (en) 2010-09-29 2012-04-05 Innovascreen Inc. Recombinant polypeptides for membrane fusion and uses thereof
US20140189897A1 (en) 2011-06-21 2014-07-03 Mayo Foundation For Medical Education And Research Transgenic animals capable of being induced to delete senescent cells
WO2013158664A2 (en) 2012-04-17 2013-10-24 Kythera Biopharmaceuticals, Inc. Use of engineered viruses to specifically kill senescent cells
US9687448B2 (en) 2012-12-07 2017-06-27 Alnylam Pharmaceuticals, Inc. Nucleic acid lipid particle formulations
CN105142614A (zh) 2013-03-14 2015-12-09 迪克纳制药公司 用于配制阴离子试剂的方法
BR112015024605A2 (pt) * 2013-03-24 2017-07-18 Oisin Biotechnologies sistemas e métodos para a produção visada de proteína terapêutica dentro de célula alvo
DE112014003136A5 (de) * 2013-07-01 2016-04-21 Charité - Universitätsmedizin Berlin Immortalisierte humane Chorionzelllinie sowie Verfahren zur Immortalisierung humaner Chorionzellen
EP3234144B1 (en) 2014-12-15 2020-08-26 Bellicum Pharmaceuticals, Inc. Methods for controlled elimination of therapeutic cells
GB201503133D0 (en) 2015-02-24 2015-04-08 Ucl Business Plc And Syncona Partners Llp Chimeric protein
EP3297680A2 (en) 2015-05-20 2018-03-28 Yeda Research and Development Co. Ltd Method of targeting senescent cells
CN110678208B (zh) 2017-01-09 2024-04-05 奥依信生物技术公司 膜融合脂质纳米颗粒和制造方法以及用于产生治疗性蛋白质和用于治疗的用途
CN112312918A (zh) 2018-04-18 2021-02-02 奥依信生物技术公司 促融合脂质纳米粒和制备其的方法以及其用于靶细胞特异性产生治疗蛋白和治疗与靶细胞有关的疾病、病况或障碍的用途
WO2020163408A2 (en) 2019-02-04 2020-08-13 Oisin Biotechnologies, Inc. Fusogenic lipid nanoparticles for the target cell-specific production of rapamycin inducible therapeutic proteins

Also Published As

Publication number Publication date
JP2020503390A (ja) 2020-01-30
EP3565607A4 (en) 2020-11-18
US20190330657A1 (en) 2019-10-31
US11525146B2 (en) 2022-12-13
WO2018129563A1 (en) 2018-07-12
CN110678208B (zh) 2024-04-05
CA3047140A1 (en) 2018-07-12
US20230220422A1 (en) 2023-07-13
AU2018206485A1 (en) 2019-07-11
EP3565607A1 (en) 2019-11-13
CN110678208A (zh) 2020-01-10

Similar Documents

Publication Publication Date Title
AU2018206485B2 (en) Fusogenic lipid nanoparticles and methods for manufacturing and use for therapeutic protein production and for treatment
CN112312918A (zh) 促融合脂质纳米粒和制备其的方法以及其用于靶细胞特异性产生治疗蛋白和治疗与靶细胞有关的疾病、病况或障碍的用途
AU2018306307B2 (en) Enhanced chimeric antigen receptors and use thereof
AU2016232146B2 (en) Optimized liver-specific expression systems for FVIII and FIX
TW202237826A (zh) 基因編輯的自然殺手細胞
KR20220130093A (ko) 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
CN112912112B (zh) 肝特异性核酸调节元件以及其方法及用途
AU2016343979A1 (en) Delivery of central nervous system targeting polynucleotides
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
KR20190100200A (ko) 동종이형 종양 세포 백신
KR20230023641A (ko) Gjb2-연관 난청을 치료하기 위한 조성물 및 방법
KR20240022575A (ko) 아머링된 키메라 수용체 및 이의 사용 방법
KR20240004253A (ko) 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 방법
KR20240021799A (ko) 청력 손실을 치료하기 위한 유전자 치료 전달 조성물 및 방법
CN111733174B (zh) 一种分离的核酸分子及其用途
CN115666722A (zh) 包封的rna复制子和使用方法
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
KR20230041028A (ko) SARS-CoV-2 면역원성 조성물, 백신 및 방법
JP2025519070A (ja) 効率的in vivo送達のための組成物および方法
CN113260704B (zh) CYP4V2和RdCVF在制备药物中的用途
CN107614515A (zh) 用于抗hiv(人免疫缺陷病毒)疗法和/或疫苗的t20构建体
CN114573716A (zh) 一种含ccl1的融合蛋白、制备方法及应用
CN116323942A (zh) 用于基因组编辑的组合物及其使用方法
KR20220142502A (ko) 근육 특이적 핵산 조절 요소 및 이의 방법 및 용도
CA3218783A1 (en) Flexible expression vector systems and application of same to vaccines and immunotherapeutics

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)