DK3338765T3 - Steroidderivat til leveringen af mrna i humangenetiske sygdomme - Google Patents

Steroidderivat til leveringen af mrna i humangenetiske sygdomme Download PDF

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Publication number
DK3338765T3
DK3338765T3 DK18156531.8T DK18156531T DK3338765T3 DK 3338765 T3 DK3338765 T3 DK 3338765T3 DK 18156531 T DK18156531 T DK 18156531T DK 3338765 T3 DK3338765 T3 DK 3338765T3
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mrna
transfer vehicle
protein
nucleic acid
cells
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DK18156531.8T
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English (en)
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Braydon Charles Guild
Frank Derosa
Michael Heartlein
Michael Concino
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Translate Bio Inc
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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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Dermatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Optics & Photonics (AREA)
  • Nanotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (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)

Claims (16)

1. Forbindelse med følgende struktur:
2. Transfervehikel omfattende forbindelsen ifølge krav 1 og en terapeutisk mængde af: (a) nukleinsyre, eller (b) et protein.
3. Transfervehikel ifølge krav 2, yderligere omfattende én eller flere kationiske lipider, ikke-kationiske lipider, og/eller PEG-modificerede lipider.
4. Transfervehikel ifølge krav 2 eller krav 3, hvor nukleinsyren er RNA.
5. Transfervehikel ifølge krav 4, hvor RNA'et er mRNA.
6. Transfervehikel ifølge krav 2, hvor proteinet er et enzym.
7. Transfervehikel ifølge et hvilket som helst af kravene 2 til 6, hvor transfervehiklet er en lipidnanopartikel eller et liposom.
8. Transfervehikel ifølge et hvilket som helst af kravene 2 til 7, hvor transfervehiklet indkapsler nukleinsyre eller protein.
9. Transfervehikel ifølge et hvilket som helst af kravene 2 til 8, hvor transfervehiklet yderligere omfatter ét eller flere terapeutiske eller diagnostiske midler.
10. Farmaceutisk sammensætning omfattende transfervehiklet ifølge et hvilket som helst af kravene 2 til 9.
11. Transfervehikel eller farmaceutisk sammensætning ifølge et hvilket som helst af kravene 2 til 10, til anvendelse i en fremgangsmåde til behandling af en sygdom i et individ forårsaget af proteinmangel, hvor nukleinsyren koder for det protein som individet er mangelfuldt i, eller proteinet er proteinet som individet er mangelfuldt i.
12. Transfervehikel eller farmaceutisk sammensætning til anvendelse ifølge krav 11, hvor sygdommen i et individ forårsaget af proteinmangel er valgt fra: SMN1-relateret spinal muskulær atrofi (SMA); amyotrof lateral sclerose (ALS); GALT-relateret galactosæmi; cystisk fibrose (CF); SLC3Al-relaterede sygdomme herunder cystinuria; COL4A5-relaterede sygdomme herunder Alport syndrom; galactocerebrosidase-mangel; X-forbundet adrenoleukodystrofi og adrenomyeloneuropati; Friedreich's ataxi; Pelizaeus-Merzbacher sygdom; TSC1 og TSC2-relateret tuberøs sclerose; Sanfilippo B syndrom (MPS HIB); CTNS-relateret cystinose; FMRl-relaterede sygdomme der omfatter Fragile X syndrom, Fragile X-Associated Tremor/Ataxia syndrom og Fragil-X omfattende præmatur ovarieinsufficienssyndrom; Prader-Willi syndrom; arvelig hæmorragisk telangiectasi (AT); Niemann-Pick sygdom Type Cl; neuronal ceroid lipofuscinose-relaterede sygdomme herunder juvenil neuronal Ceroid Lipofuscinose (JNCL), juvenil Batten sygdom, Santavuori-Haltia sygdom, Jansky-Bielschowsky sygdom, og PTT-1- og TPPl-mangler; EIF2B1, EIF2B2, EIF2B3, EIF2B4 og EIF2B5-relateret barndoms-ataxi med hypomyelination/forsvinden af hvid substans i centralnervesystemet; CACNA1A og CACNB4-relateret episodisk ataxi Type 2; MECP2-relaterede sygdomme herunder klassisk Rett-Syndrom, MECP2-relateret svær neonatal encephalopathi og PPM-X Syndrom; CD KL5-relate ret atypisk Rett-Syndrom; Kennedy's sygdom (SBMA); Notch-3 relateret cerebral autosomal dominant arteriopati med subcorticale infarkter og leukoencephalopathi (CADASIL); SCNIAog SCNIB-relaterede epileptiske lidelser; polymerase G-relaterede sygdomme der omfatter Alpers-Huttenlocher-syndrom, POLG-relate ret sensorisk ataxisk neuropati, dysarthri, og ophthalmoparese, og autosomal dominant og recessiv progressiv extern ophthalmoplegi med mitochondrielle DNA- deletioner; X-forbundet adrenal hypoplasi; X-forbundet agammaglobulinæmi; og Wilson's sygdom.
13. Fremgangsmåde til fremstilling af forbindelsen ifølge krav 1, idet fremgangsmåden omfatter trin (a), at danne forbindelse 3 fra forbindelserne 1 og 2: Trin (a)
14. Fremgangsmåden ifølge krav 13, hvor fremgangsmåden yderligere omfatter trin (b) at behandle forbindelse 3 med en stærk syre for at få forbindelsen ifølge krav 1: Trin (b)
15. Fremgangsmåden ifølge krav 13 eller krav 14, hvor trin (a) yderligere omfatter dicyclohexylcarbodiimid (DCC) og dimethylaminopyridin (DMAP).
16. Fremgangsmåden ifølge krav 14 eller krav 15, hvor den stærke syre anvendt i trin (b) er trifluoreddikesyre (TFA).
DK18156531.8T 2009-12-01 2010-11-30 Steroidderivat til leveringen af mrna i humangenetiske sygdomme DK3338765T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US26565309P 2009-12-01 2009-12-01
EP10835016.6A EP2506857B1 (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
DK3338765T3 true DK3338765T3 (da) 2019-03-04

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ID=44115246

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Application Number Title Priority Date Filing Date
DK18156531.8T DK3338765T3 (da) 2009-12-01 2010-11-30 Steroidderivat til leveringen af mrna i humangenetiske sygdomme
DK10835016.6T DK2506857T3 (da) 2009-12-01 2010-11-30 Levering af mrna til forstærkning af proteiner og enzymer i human-genetiske sygdomme

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DK10835016.6T DK2506857T3 (da) 2009-12-01 2010-11-30 Levering af mrna til forstærkning af proteiner og enzymer i human-genetiske sygdomme

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US (9) US20110244026A1 (da)
EP (5) EP3403647A1 (da)
AU (1) AU2010326132B9 (da)
CA (2) CA3077990A1 (da)
CY (2) CY1120272T1 (da)
DK (2) DK3338765T3 (da)
ES (3) ES2713852T3 (da)
HR (2) HRP20180696T1 (da)
HU (2) HUE042177T2 (da)
LT (2) LT3338765T (da)
ME (2) ME03091B (da)
NO (1) NO2506857T3 (da)
NZ (6) NZ600616A (da)
PL (2) PL3338765T3 (da)
PT (2) PT2506857T (da)
RS (2) RS57314B1 (da)
SI (2) SI2506857T1 (da)
TR (1) TR201901311T4 (da)
WO (1) WO2011068810A1 (da)

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