CA3111836A1 - Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders - Google Patents
Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders Download PDFInfo
- Publication number
- CA3111836A1 CA3111836A1 CA3111836A CA3111836A CA3111836A1 CA 3111836 A1 CA3111836 A1 CA 3111836A1 CA 3111836 A CA3111836 A CA 3111836A CA 3111836 A CA3111836 A CA 3111836A CA 3111836 A1 CA3111836 A1 CA 3111836A1
- Authority
- CA
- Canada
- Prior art keywords
- abcb4
- mir
- sequence
- utr
- polynucleotide
- 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
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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
-
- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- 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
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- 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
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
- C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
-
- 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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Gastroenterology & Hepatology (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biophysics (AREA)
- Optics & Photonics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Toxicology (AREA)
- Immunology (AREA)
- Biotechnology (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Nanotechnology (AREA)
- Inorganic Chemistry (AREA)
- Dermatology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862731055P | 2018-09-13 | 2018-09-13 | |
| US62/731,055 | 2018-09-13 | ||
| US201962840369P | 2019-04-29 | 2019-04-29 | |
| US62/840,369 | 2019-04-29 | ||
| PCT/US2019/051172 WO2020056370A1 (en) | 2018-09-13 | 2019-09-13 | Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3111836A1 true CA3111836A1 (en) | 2020-03-19 |
Family
ID=69778403
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3111836A Pending CA3111836A1 (en) | 2018-09-13 | 2019-09-13 | Modified mrna for the treatment of progressive familial intrahepatic cholestasis disorders |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20220054653A1 (enExample) |
| EP (1) | EP3863645A4 (enExample) |
| JP (1) | JP2022500443A (enExample) |
| AU (1) | AU2019338557A1 (enExample) |
| CA (1) | CA3111836A1 (enExample) |
| WO (1) | WO2020056370A1 (enExample) |
Families Citing this family (53)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL4023249T3 (pl) | 2014-04-23 | 2025-03-10 | Modernatx, Inc. | Szczepionki z kwasem nukleinowym |
| MA42502A (fr) | 2015-07-21 | 2018-05-30 | Modernatx Inc | Vaccins contre une maladie infectieuse |
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| MA42543A (fr) | 2015-07-30 | 2018-06-06 | Modernatx Inc | Arn épitope peptidiques concatémériques |
| US11564893B2 (en) | 2015-08-17 | 2023-01-31 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| WO2017070624A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Tropical disease vaccines |
| EP4011451A1 (en) | 2015-10-22 | 2022-06-15 | ModernaTX, Inc. | Metapneumovirus mrna vaccines |
| AU2016342045A1 (en) | 2015-10-22 | 2018-06-07 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| WO2017070601A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Nucleic acid vaccines for varicella zoster virus (vzv) |
| AU2016341309A1 (en) | 2015-10-22 | 2018-06-07 | Modernatx, Inc. | Cancer vaccines |
| PL3386484T3 (pl) | 2015-12-10 | 2022-07-25 | Modernatx, Inc. | Kompozycje i sposoby dostarczania środków terapeutycznych |
| WO2017201342A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding jagged1 for the treatment of alagille syndrome |
| CA3036831A1 (en) | 2016-09-14 | 2018-03-22 | Modernatx, Inc. | High purity rna compositions and methods for preparation thereof |
| EP3528821A4 (en) | 2016-10-21 | 2020-07-01 | ModernaTX, Inc. | HUMAN CYTOMEGALOVIRUS VACCINE |
| MA46766A (fr) | 2016-11-11 | 2019-09-18 | Modernatx Inc | Vaccin antigrippal |
| WO2018107088A2 (en) | 2016-12-08 | 2018-06-14 | Modernatx, Inc. | Respiratory virus nucleic acid vaccines |
| US11384352B2 (en) | 2016-12-13 | 2022-07-12 | Modernatx, Inc. | RNA affinity purification |
| WO2018170256A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Herpes simplex virus vaccine |
| WO2018170270A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Varicella zoster virus (vzv) vaccine |
| EP3595713A4 (en) | 2017-03-15 | 2021-01-13 | ModernaTX, Inc. | RESPIRATORY SYNCYTIAL VIRUS Vaccine |
| WO2018170245A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
| EP3595676A4 (en) | 2017-03-17 | 2021-05-05 | Modernatx, Inc. | RNA VACCINES AGAINST ZOONOSES |
| US11905525B2 (en) | 2017-04-05 | 2024-02-20 | Modernatx, Inc. | Reduction of elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins |
| MA49421A (fr) | 2017-06-15 | 2020-04-22 | Modernatx Inc | Formulations d'arn |
| MA49914A (fr) | 2017-08-18 | 2021-04-21 | Modernatx Inc | Procédés analytiques par hplc |
| WO2019036682A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | RNA VARIANTS POLYMERASE |
| EP3668979A4 (en) | 2017-08-18 | 2021-06-02 | Modernatx, Inc. | METHOD OF HPLC ANALYSIS |
| EP3675817A1 (en) | 2017-08-31 | 2020-07-08 | Modernatx, Inc. | Methods of making lipid nanoparticles |
| WO2019055807A1 (en) | 2017-09-14 | 2019-03-21 | Modernatx, Inc. | RNA VACCINES AGAINST ZIKA VIRUS |
| EP3746090A4 (en) | 2018-01-29 | 2021-11-17 | ModernaTX, Inc. | RSV RNA Vaccines |
| CA3113025A1 (en) | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | Peg lipids and uses thereof |
| WO2020061295A1 (en) | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| WO2020061457A1 (en) | 2018-09-20 | 2020-03-26 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
| US11351242B1 (en) | 2019-02-12 | 2022-06-07 | Modernatx, Inc. | HMPV/hPIV3 mRNA vaccine composition |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| AU2020224103A1 (en) | 2019-02-20 | 2021-09-16 | Modernatx, Inc. | Rna polymerase variants for co-transcriptional capping |
| CN113874502A (zh) | 2019-03-11 | 2021-12-31 | 摩登纳特斯有限公司 | 补料分批体外转录方法 |
| US12070495B2 (en) | 2019-03-15 | 2024-08-27 | Modernatx, Inc. | HIV RNA vaccines |
| CN111041025B (zh) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法 |
| IL297419B2 (en) | 2020-04-22 | 2025-02-01 | BioNTech SE | Coronavirus vaccine |
| WO2021231503A2 (en) * | 2020-05-11 | 2021-11-18 | The Board Of Trustees Of The Leland Stanford Junior University | Systems and methods for enhancing gene expression |
| CN111744019B (zh) * | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | 基于甘露糖的mRNA靶向递送系统及其应用 |
| EP3936946A1 (fr) | 2020-07-10 | 2022-01-12 | Patek Philippe SA Genève | Oscillateur horloger a pivot flexible |
| US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| EP4274607A1 (en) | 2021-01-11 | 2023-11-15 | ModernaTX, Inc. | Seasonal rna influenza virus vaccines |
| US20220363937A1 (en) | 2021-05-14 | 2022-11-17 | Armstrong World Industries, Inc. | Stabilization of antimicrobial coatings |
| US12186387B2 (en) | 2021-11-29 | 2025-01-07 | BioNTech SE | Coronavirus vaccine |
| US12529047B1 (en) | 2021-12-21 | 2026-01-20 | Modernatx, Inc. | mRNA quantification methods |
| WO2024002985A1 (en) | 2022-06-26 | 2024-01-04 | BioNTech SE | Coronavirus vaccine |
| CN115404273B (zh) * | 2022-06-30 | 2023-06-23 | 湖南家辉生物技术有限公司 | 导致进行性家族性肝内胆汁淤积症ⅰ型的atp8b1基因突变体、蛋白和应用 |
| WO2025024442A1 (en) * | 2023-07-26 | 2025-01-30 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating conditions associated with bsep deficiency |
| WO2025212858A1 (en) | 2024-04-03 | 2025-10-09 | Children's Hospital Medical Center | Ivt messenger rna and methods of treating or preventing pfic type iv |
| CN119587504B (zh) * | 2024-12-10 | 2025-11-14 | 华中科技大学同济医学院附属协和医院 | 一种靶向骨组织巨噬细胞的纳米颗粒及其制备方法与应用 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9012219B2 (en) * | 2005-08-23 | 2015-04-21 | The Trustees Of The University Of Pennsylvania | RNA preparations comprising purified modified RNA for reprogramming cells |
| PL2578685T3 (pl) * | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| WO2011025862A2 (en) * | 2009-08-28 | 2011-03-03 | Curna, Inc. | Treatment of atp-binding cassette sub-family b member 1 (abcb1) related diseases by inhibition of natural antisense transcript to abcb1 |
| US20140275229A1 (en) * | 2012-04-02 | 2014-09-18 | Moderna Therapeutics, Inc. | Modified polynucleotides encoding udp glucuronosyltransferase 1 family, polypeptide a1 |
| EP2906697A4 (en) * | 2012-10-15 | 2016-06-22 | Ionis Pharmaceuticals Inc | METHOD FOR MONITORING THE C9ORF72 EXPRESSION |
| US20160032316A1 (en) * | 2013-03-14 | 2016-02-04 | The Trustees Of The University Of Pennsylvania | Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides |
| GB2560250A (en) * | 2015-06-30 | 2018-09-05 | Ethris Gmbh | ATP-Binding cassette family coding polyribonucleotides and formulations thereof |
| EP3368089B1 (en) * | 2015-10-26 | 2025-11-05 | Translate Bio Ma, Inc. | Nanoparticle formulations for delivery of nucleic acid complexes |
| PL3386484T3 (pl) * | 2015-12-10 | 2022-07-25 | Modernatx, Inc. | Kompozycje i sposoby dostarczania środków terapeutycznych |
| EP3426775A1 (en) * | 2016-03-10 | 2019-01-16 | Novartis AG | Chemically modified messenger rna's |
| US12123030B2 (en) * | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| BR112019015797A2 (pt) * | 2017-02-01 | 2020-03-17 | Modernatx, Inc. | Composições de mrna terapêuticas imunomoduladoras que codificam peptídeos de mutação de oncogene de ativação |
-
2019
- 2019-09-13 CA CA3111836A patent/CA3111836A1/en active Pending
- 2019-09-13 EP EP19861226.9A patent/EP3863645A4/en active Pending
- 2019-09-13 JP JP2021514132A patent/JP2022500443A/ja active Pending
- 2019-09-13 WO PCT/US2019/051172 patent/WO2020056370A1/en not_active Ceased
- 2019-09-13 AU AU2019338557A patent/AU2019338557A1/en not_active Abandoned
- 2019-09-13 US US17/276,112 patent/US20220054653A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| US20220054653A1 (en) | 2022-02-24 |
| WO2020056370A9 (en) | 2020-05-07 |
| EP3863645A1 (en) | 2021-08-18 |
| EP3863645A4 (en) | 2022-11-16 |
| AU2019338557A1 (en) | 2021-03-25 |
| JP2022500443A (ja) | 2022-01-04 |
| WO2020056370A1 (en) | 2020-03-19 |
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