PL3923971T3 - Analizowanie wpływu polirybonukleotydu na ciliogenezę - Google Patents

Analizowanie wpływu polirybonukleotydu na ciliogenezę

Info

Publication number
PL3923971T3
PL3923971T3 PL20704031.2T PL20704031T PL3923971T3 PL 3923971 T3 PL3923971 T3 PL 3923971T3 PL 20704031 T PL20704031 T PL 20704031T PL 3923971 T3 PL3923971 T3 PL 3923971T3
Authority
PL
Poland
Prior art keywords
ciliogenesis
polyribonucleotide
analyzing
effect
Prior art date
Application number
PL20704031.2T
Other languages
English (en)
Inventor
Carsten Rudolph
Verena Mummert
Rebekka Kubisch-Dohmen
Christian Dohmen
Johannes Geiger
Manish ANEJA
Ludwig Weiss
Heymut OMRAN
Petra PENNEKAMP
Kai WOHLGEMUTH
Sandra CINDRIC
Niki Tomas LOGES
Johanna RAIDT
Adrian TER STEEGE
Original Assignee
Ethris Gmbh
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 Ethris Gmbh filed Critical Ethris Gmbh
Publication of PL3923971T3 publication Critical patent/PL3923971T3/pl

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
    • 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
    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/195—Carboxylic acids, e.g. valproic acid having an amino group
    • A61K31/197—Carboxylic acids, e.g. valproic acid having an amino group the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid
    • A61K31/198—Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K9/0012—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5044—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics involving specific cell types
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00

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)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pulmonology (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Zoology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Otolaryngology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Dispersion Chemistry (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Hematology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Analytical Chemistry (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • General Physics & Mathematics (AREA)
PL20704031.2T 2019-02-14 2020-02-13 Analizowanie wpływu polirybonukleotydu na ciliogenezę PL3923971T3 (pl)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP19157210 2019-02-14
PCT/EP2020/053774 WO2020165352A1 (en) 2019-02-14 2020-02-13 Treatment of ciliopathies

Publications (1)

Publication Number Publication Date
PL3923971T3 true PL3923971T3 (pl) 2024-03-25

Family

ID=65440876

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20704031.2T PL3923971T3 (pl) 2019-02-14 2020-02-13 Analizowanie wpływu polirybonukleotydu na ciliogenezę

Country Status (14)

Country Link
US (1) US20220211807A1 (pl)
EP (2) EP3923971B1 (pl)
JP (2) JP7607340B2 (pl)
KR (1) KR20210127718A (pl)
CN (2) CN118949075A (pl)
AU (1) AU2020220636A1 (pl)
BR (1) BR112021014528A2 (pl)
CA (2) CA3225057A1 (pl)
ES (1) ES2974368T3 (pl)
HU (1) HUE065017T2 (pl)
MX (1) MX2021009388A (pl)
PL (1) PL3923971T3 (pl)
WO (1) WO2020165352A1 (pl)
ZA (1) ZA202106769B (pl)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3025626A1 (en) 2016-05-27 2017-11-30 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
EP4240396B1 (en) * 2020-11-04 2025-01-01 Ethris GmbH Ifn-lambda mrna for treating viral infections
EP4308703A4 (en) * 2021-03-19 2025-06-04 Recode Therapeutics, Inc. Polynucleotide compositions, associated formulations and methods of using the same
WO2022204053A1 (en) * 2021-03-22 2022-09-29 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
WO2023014887A1 (en) * 2021-08-04 2023-02-09 The Trustees Of Indiana University Methods of treating compromised lung function and assessing clinical improvement
CN121335708A (zh) 2023-05-01 2026-01-13 耶鲁大学 用于疫苗接种的抗原特异性生理性树突状细胞
JP2026516460A (ja) 2023-05-01 2026-05-25 エール・ユニバーシティ 療法用の抗原特異的生理的樹状細胞
WO2024240962A1 (en) 2023-05-25 2024-11-28 Ethris Gmbh Gm-csf-encoding nucleic acids, pharmaceutical compositions, methods and uses thereof
CN121712488A (zh) 2023-08-14 2026-03-20 埃泽瑞斯公司 非全身性mRNA施用
CN122295574A (zh) 2023-10-17 2026-06-26 特兰西穆内有限公司 用于分析免疫应答和抗原谱分析的方法
JPWO2025178019A1 (pl) * 2024-02-19 2025-08-28
KR20260025059A (ko) * 2024-08-13 2026-02-23 전남대학교산학협력단 신규한 치환된 피리딘 유도체 화합물, 이의 제조방법, 및 이를 유효성분으로 포함하는 호흡기 질환의 예방 또는 치료용 약학적 조성물

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
WO1999008997A1 (de) 1997-08-13 1999-02-25 Biontex Laboratories Gmbh Neue lipopolyamine, deren darstellung und anwendung
WO2001000708A1 (de) 1999-06-25 2001-01-04 Christian Plank Kombinationen zur einführung von nucleinsäuren in zellen
CA2569664C (en) 2004-06-07 2013-07-16 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
FR2925491B1 (fr) 2007-12-19 2010-09-03 Oz Biosciences Sas Nouvelle classe de lipides cationiques pour le transport d'agents actifs dans les cellules
EA201492055A1 (ru) * 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
EA037448B1 (ru) * 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция
WO2014020723A1 (ja) 2012-08-01 2014-02-06 株式会社コナミデジタルエンタテインメント 処理装置、処理装置の制御方法、及び、処理装置のプログラム
KR102285326B1 (ko) * 2013-06-28 2021-08-04 에트리스 게엠베하 Rna를 세포에 도입하기 위한 조성물
US12263229B2 (en) 2014-11-10 2025-04-01 Ethris Gmbh Induction of osteogenesis by delivering BMP encoding RNA
CA3025626A1 (en) * 2016-05-27 2017-11-30 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
GB201707212D0 (en) 2017-05-05 2017-06-21 Ucl Business Plc Gene therapy for ciliopathies

Also Published As

Publication number Publication date
CA3225057A1 (en) 2020-08-20
MX2021009388A (es) 2021-09-10
CA3129912A1 (en) 2020-08-20
JP2022522412A (ja) 2022-04-19
BR112021014528A2 (pt) 2021-10-13
EP4223306A3 (en) 2023-11-29
EP3923971C0 (en) 2023-12-20
KR20210127718A (ko) 2021-10-22
JP7607340B2 (ja) 2024-12-27
CN113423418B (zh) 2025-01-10
CN113423418A (zh) 2021-09-21
EP4223306A2 (en) 2023-08-09
EP3923971B1 (en) 2023-12-20
US20220211807A1 (en) 2022-07-07
CN118949075A (zh) 2024-11-15
ZA202106769B (en) 2024-01-31
ES2974368T3 (es) 2024-06-27
WO2020165352A1 (en) 2020-08-20
EP3923971A1 (en) 2021-12-22
JP2024096766A (ja) 2024-07-17
AU2020220636A1 (en) 2021-07-29
HUE065017T2 (hu) 2024-04-28

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