CY1120272T1 - ΠΑΡΟΧΗ mRNA ΓΙΑ ΤΗΝ ΑΥΞΗΣΗ ΠΡΩΤΕΪΝΩΝ ΚΑΙ ΕΝΖΥΜΩΝ ΣΕ ΑΝΘΡΩΠΙΝΕΣ ΓΕΝΕΤΙΚΕΣ ΑΣΘΕΝΕΙΕΣ - Google Patents

ΠΑΡΟΧΗ mRNA ΓΙΑ ΤΗΝ ΑΥΞΗΣΗ ΠΡΩΤΕΪΝΩΝ ΚΑΙ ΕΝΖΥΜΩΝ ΣΕ ΑΝΘΡΩΠΙΝΕΣ ΓΕΝΕΤΙΚΕΣ ΑΣΘΕΝΕΙΕΣ

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Publication number
CY1120272T1
CY1120272T1 CY20181100491T CY181100491T CY1120272T1 CY 1120272 T1 CY1120272 T1 CY 1120272T1 CY 20181100491 T CY20181100491 T CY 20181100491T CY 181100491 T CY181100491 T CY 181100491T CY 1120272 T1 CY1120272 T1 CY 1120272T1
Authority
CY
Cyprus
Prior art keywords
mrna
enzymes
provision
genetic diseases
human genetic
Prior art date
Application number
CY20181100491T
Other languages
English (en)
Inventor
Braydon Charles Guild
Frank Derosa
Michael Heartlein
Michael Concino
Original Assignee
Translate Bio, 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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=44115246&utm_source=***_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1120272(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Translate Bio, Inc. filed Critical Translate Bio, Inc.
Publication of CY1120272T1 publication Critical patent/CY1120272T1/el

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    • 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
    • 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
    • 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
    • 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
    • A61K9/0019—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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • 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
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • 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
    • C07J—STEROIDS
    • C07J43/00—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton
    • C07J43/003—Normal steroids having a nitrogen-containing hetero ring spiro-condensed or not condensed with the cyclopenta(a)hydrophenanthrene skeleton not condensed
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Dispersion Chemistry (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Nanotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Steroid Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

Στο παρόν περιγράφονται συνθέσεις και μέθοδοι τροποποίησης της έκφρασης γονιδίου ή της παραγωγής μιας πρωτεΐνης με διαμόλυνση κυττάρων στόχων με νουκλεϊκά οξέα. Οι περιγραφόμενες στο παρόν συνθέσεις εμφανίζουν υψηλή αποτελεσματικότητα διαμόλυνσης και μπορούν να βελτιώνουν ασθένειες συνδεόμενες με ανεπάρκεια πρωτεϊνών ή ενζύμων.
CY20181100491T 2009-12-01 2018-05-14 ΠΑΡΟΧΗ mRNA ΓΙΑ ΤΗΝ ΑΥΞΗΣΗ ΠΡΩΤΕΪΝΩΝ ΚΑΙ ΕΝΖΥΜΩΝ ΣΕ ΑΝΘΡΩΠΙΝΕΣ ΓΕΝΕΤΙΚΕΣ ΑΣΘΕΝΕΙΕΣ CY1120272T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US26565309P 2009-12-01 2009-12-01
PCT/US2010/058457 WO2011068810A1 (en) 2009-12-01 2010-11-30 Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases

Publications (1)

Publication Number Publication Date
CY1120272T1 true CY1120272T1 (el) 2019-07-10

Family

ID=44115246

Family Applications (2)

Application Number Title Priority Date Filing Date
CY20181100491T CY1120272T1 (el) 2009-12-01 2018-05-14 ΠΑΡΟΧΗ mRNA ΓΙΑ ΤΗΝ ΑΥΞΗΣΗ ΠΡΩΤΕΪΝΩΝ ΚΑΙ ΕΝΖΥΜΩΝ ΣΕ ΑΝΘΡΩΠΙΝΕΣ ΓΕΝΕΤΙΚΕΣ ΑΣΘΕΝΕΙΕΣ
CY20191100274T CY1121516T1 (el) 2009-12-01 2019-03-07 ΣΤΕΡΟΕΙΔΕΣ ΠΑΡΑΓΩΓΟ ΓΙΑ ΠΑΡΟΧΗ mRNA ΣΕ ΠΕΡΙΠΤΩΣΕΙΣ ΑΝΘΡΩΠΙΝΩΝ ΓΕΝΕΤΙΚΩΝ ΝΟΣΩΝ

Family Applications After (1)

Application Number Title Priority Date Filing Date
CY20191100274T CY1121516T1 (el) 2009-12-01 2019-03-07 ΣΤΕΡΟΕΙΔΕΣ ΠΑΡΑΓΩΓΟ ΓΙΑ ΠΑΡΟΧΗ mRNA ΣΕ ΠΕΡΙΠΤΩΣΕΙΣ ΑΝΘΡΩΠΙΝΩΝ ΓΕΝΕΤΙΚΩΝ ΝΟΣΩΝ

Country Status (19)

Country Link
US (9) US20110244026A1 (el)
EP (5) EP3403647A1 (el)
AU (1) AU2010326132B9 (el)
CA (2) CA2782676C (el)
CY (2) CY1120272T1 (el)
DK (2) DK3338765T3 (el)
ES (3) ES2713852T3 (el)
HR (2) HRP20180696T1 (el)
HU (2) HUE042177T2 (el)
LT (2) LT3338765T (el)
ME (2) ME03327B (el)
NO (1) NO2506857T3 (el)
NZ (5) NZ733634A (el)
PL (2) PL3338765T3 (el)
PT (2) PT2506857T (el)
RS (2) RS57314B1 (el)
SI (2) SI2506857T1 (el)
TR (1) TR201901311T4 (el)
WO (1) WO2011068810A1 (el)

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