NZ794954A - Nucleic acids and methods of treatment for cystic fibrosis - Google Patents

Nucleic acids and methods of treatment for cystic fibrosis

Info

Publication number
NZ794954A
NZ794954A NZ794954A NZ79495421A NZ794954A NZ 794954 A NZ794954 A NZ 794954A NZ 794954 A NZ794954 A NZ 794954A NZ 79495421 A NZ79495421 A NZ 79495421A NZ 794954 A NZ794954 A NZ 794954A
Authority
NZ
New Zealand
Prior art keywords
cystic fibrosis
treatment
methods
nucleic acids
protein
Prior art date
Application number
NZ794954A
Other languages
English (en)
Inventor
Carlos G Perez-Garcia
Kiyoshi Tachikawa
Daiki Matsuda
Padmanabh Chivukula
Priya Prakash Karmali
Yanjie Bao
Jerel Boyd Lee Vega
Rajesh Mukthavaram
Amit Sagi
Original Assignee
Arcturus 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 Arcturus Therapeutics Inc filed Critical Arcturus Therapeutics Inc
Publication of NZ794954A publication Critical patent/NZ794954A/en

Links

Classifications

    • 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
    • A61K48/0033—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 non-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
    • A61K9/0043—Nose
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pulmonology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Toxicology (AREA)
  • Otolaryngology (AREA)
  • Physics & Mathematics (AREA)
  • Biomedical Technology (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
NZ794954A 2020-05-01 2021-04-30 Nucleic acids and methods of treatment for cystic fibrosis NZ794954A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063019170P 2020-05-01 2020-05-01
US202063082997P 2020-09-24 2020-09-24
PCT/US2021/030257 WO2021222801A2 (en) 2020-05-01 2021-04-30 Nucleic acids and methods of treatment for cystic fibrosis

Publications (1)

Publication Number Publication Date
NZ794954A true NZ794954A (en) 2026-03-27

Family

ID=78374030

Family Applications (1)

Application Number Title Priority Date Filing Date
NZ794954A NZ794954A (en) 2020-05-01 2021-04-30 Nucleic acids and methods of treatment for cystic fibrosis

Country Status (7)

Country Link
US (2) US12070509B2 (https=)
EP (1) EP4143217A4 (https=)
JP (1) JP2023524071A (https=)
AU (1) AU2021263590A1 (https=)
CA (1) CA3176844A1 (https=)
NZ (1) NZ794954A (https=)
WO (1) WO2021222801A2 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017201347A1 (en) 2016-05-18 2017-11-23 Modernatx, Inc. Polynucleotides encoding cystic fibrosis transmembrane conductance regulator for the treatment of cystic fibrosis
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
US12070509B2 (en) * 2020-05-01 2024-08-27 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
CN116368226A (zh) 2020-09-04 2023-06-30 维乎医疗有限公司 用于加帽rna的组合物和方法
IL303195A (en) 2020-11-25 2023-07-01 Akagera Medicines Inc Lipid nanoparticles for delivery of nucleic acids and related methods of use
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
JP2024511437A (ja) 2021-03-23 2024-03-13 リコード セラピューティクス，インク． ポリヌクレオチド組成物、関連製剤、およびその使用方法
KR20240006603A (ko) 2021-05-07 2024-01-15 헬릭스 나노테크놀로지스, 인코포레이티드 변형된 리보핵산 및 이의 용도
MX2024002726A (es) 2021-09-03 2024-03-20 CureVac SE Nuevas nanoparticulas lipidicas para la administracion de acidos nucleicos.
US20250099614A1 (en) 2022-01-28 2025-03-27 CureVac SE Nucleic acid encoded transcription factor inhibitors
WO2023227608A1 (en) 2022-05-25 2023-11-30 Glaxosmithkline Biologicals Sa Nucleic acid based vaccine encoding an escherichia coli fimh antigenic polypeptide
EP4531819A2 (en) 2022-05-25 2025-04-09 Akagera Medicines, Inc. Lipid nanoparticles for delivery of nucleic acids and methods of use thereof
WO2024089229A1 (en) 2022-10-28 2024-05-02 CureVac SE Improved formulations comprising lipid-based carriers encapsulating rna
KR20250153298A (ko) 2023-03-08 2025-10-24 큐어백 에스이 핵산 전달을 위한 신규의 지질 나노입자 제형
WO2024230934A1 (en) 2023-05-11 2024-11-14 CureVac SE Therapeutic nucleic acid for the treatment of ophthalmic diseases
US20250144237A1 (en) * 2023-11-07 2025-05-08 Cila Therapeutics Inc. Agents, compositions and methods for enhanced delivery of nucleic acids to the cells within the respiratory system
WO2025103803A1 (en) 2023-11-13 2025-05-22 CureVac SE Immunotherapy against neuronal and brain tumors
US12364773B2 (en) 2023-12-01 2025-07-22 Recode Therapeutics, Inc. Lipid nanoparticle compositions and uses thereof
WO2026072721A1 (en) * 2024-09-25 2026-04-02 Carbon Biosciences, Inc. Parvovirus compositions and methods for gene therapy

Family Cites Families (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4511069A (en) 1981-06-04 1985-04-16 The Pharmasol Corporation Dispensing system
US4778810A (en) 1987-01-08 1988-10-18 Nastech Pharmaceutical Co., Inc. Nasal delivery of caffeine
JP3257675B2 (ja) 1990-10-12 2002-02-18 マックス−プランク−ゲゼルシャフト ツール フェルデルング デル ビッセンシャフテン エー．ファウ． 修飾リボザイム
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
KR20040051450A (ko) * 2002-12-12 2004-06-18 주식회사 인비보사이언스 알엔에이의 생체내 안정성을 증가시키는 벡터
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
WO2010048536A2 (en) 2008-10-23 2010-04-29 Alnylam Pharmaceuticals, Inc. Processes for preparing lipids
HUE037082T2 (hu) 2008-11-10 2018-08-28 Arbutus Biopharma Corp Új lipidek és készítmények terápiás hatóanyagok szállítására
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
KR20180094137A (ko) 2009-05-05 2018-08-22 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
SG186085A1 (en) 2010-06-03 2013-01-30 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
CA2868391A1 (en) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
WO2015051169A2 (en) 2013-10-02 2015-04-09 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
US9593077B2 (en) * 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
AU2015338923B2 (en) * 2014-11-02 2021-10-21 Arcturus Therapeutics, Inc. Messenger UNA molecules and uses thereof
ES2964690T3 (es) 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
US10487105B2 (en) 2016-10-19 2019-11-26 Arcturus Therapeutics, Inc. Trinucleotide MRNA cap analogs
WO2018119163A1 (en) * 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
EA201991747A1 (ru) * 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
WO2018160592A1 (en) 2017-02-28 2018-09-07 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
ES3039647T3 (en) * 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
US12070509B2 (en) * 2020-05-01 2024-08-27 Arcturus Therapeutics, Inc. Nucleic acids and methods of treatment for cystic fibrosis
EP4426266A4 (en) * 2021-11-03 2025-11-05 Arcturus Therapeutics Inc LIPID FORMULATIONS CONTAINING NUCLEIC ACIDS AND METHODS OF TREATMENT OF CYSTIC FIBROSIS

Also Published As

Publication number Publication date
WO2021222801A2 (en) 2021-11-04
US12070509B2 (en) 2024-08-27
US20220023442A1 (en) 2022-01-27
US20250049950A1 (en) 2025-02-13
EP4143217A2 (en) 2023-03-08
JP2023524071A (ja) 2023-06-08
WO2021222801A3 (en) 2021-12-09
CA3176844A1 (en) 2021-11-04
AU2021263590A1 (en) 2023-01-19
EP4143217A4 (en) 2024-10-02

Similar Documents

Publication Publication Date Title
NZ794954A (en) Nucleic acids and methods of treatment for cystic fibrosis
JP7235702B2 (ja) 核酸生成及び送達のための方法及び製品
ZA202103233B (en) Lipid nanoparticle formulations
Merckx et al. Surfactant protein B (SP-B) enhances the cellular siRNA delivery of proteolipid coated nanogels for inhalation therapy
MX2022012630A (es) Composicion de nanoparticulas lipidicas.
Zuris et al. Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
Kormann et al. Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
MX2022009018A (es) Nanoparticulas lipidas.
PH12023500013A1 (en) Ionizable cationic lipids and lipi nanoparticles, and methods of synthesis and use thereof
PH12022552327A1 (en) Compositions and methods for targeted rna delivery
AU2018240515A1 (en) Nucleic acids encoding CRISPR-associated proteins and uses thereof
EP4406585A3 (en) Nucleic acid products and methods of administration thereof
AU2018268859A1 (en) Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
ZA202300131B (en) Lipid nanoparticles
Zhang et al. Sustained release of PKR inhibitor C16 from mesoporous silica nanoparticles significantly enhances mRNA translation and anti-tumor vaccination
BR112015024869A8 (pt) uso de um agente terapêutico e/ou de diôxido de carbono para tratar um paciente e aparelhos e métodos para tratar terapeuticamente um paciente
MX2023005203A (es) Nanoparticulas lipidicas para administrar vacunas de arnm.
AU2020328611A8 (en) Extracellular vesicle-ASO constructs targeting STAT6
EP3818991A3 (en) Compositions and methods for treating diseases
MX2025010536A (es) Nuevas formulaciones de nanoparticulas lipidicas para la administracion de acidos nucleicos
MX2024013583A (es) Formulaciones de nanopartículas lipídicas para suministro al sistema nervioso central
Muralidharan et al. Electrotransfer for nucleic acid and protein delivery
Wang et al. Morphology–and adhesion–dual biomimetic nanovaccine boosts antigen cross-presentation through subcellular transport regulation
Anjum et al. Advanced Therapeutic Approaches Based on Small Extracellular Vehicles (sEVs) For the Regeneration of Spinal Cord Injuries
MX2025002413A (es) Nuevos lipidos ionizables y nanoparticulas de lipidos y metodos de uso de los mismos