MX2022016550A - Composiciones de lnp que comprenden agentes terapeuticos de mrna con semivida extendida. - Google Patents

Composiciones de lnp que comprenden agentes terapeuticos de mrna con semivida extendida.

Info

Publication number
MX2022016550A
MX2022016550A MX2022016550A MX2022016550A MX2022016550A MX 2022016550 A MX2022016550 A MX 2022016550A MX 2022016550 A MX2022016550 A MX 2022016550A MX 2022016550 A MX2022016550 A MX 2022016550A MX 2022016550 A MX2022016550 A MX 2022016550A
Authority
MX
Mexico
Prior art keywords
lnp compositions
life
payload
extended half
mrna therapeutics
Prior art date
Application number
MX2022016550A
Other languages
English (en)
Inventor
David Reid
Ruchi Jain
Alicia Bicknell
Caroline Kohrer
Original Assignee
Modernatx 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 Modernatx Inc filed Critical Modernatx Inc
Publication of MX2022016550A publication Critical patent/MX2022016550A/es

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

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

Abstract

La divulgación presenta un polinucleótido que codifica un polipéptido, dicho polinucleótido comprende: una 5' UTR descrita en el presente documento, una región codificante que comprende una carga activa y un elemento de parada descrita en este documento, y una 3' UTR descrita en el presente documento, y composiciones de LNP que lo comprenden. Los polinucleótidos y/o las composiciones de LNP de la presente divulgación pueden: aumentar el nivel y/o la actividad de la carga activa mediante el aumento de la semivida y/o la duración de la expresión del polinucleótido que codifica la carga activa o del polipéptido de carga activa. También se describen en este documento métodos para tratar una enfermedad o trastorno en un sujeto mediante el uso de las composiciones de LNP de la presente divulgación.
MX2022016550A 2020-06-23 2021-06-23 Composiciones de lnp que comprenden agentes terapeuticos de mrna con semivida extendida. MX2022016550A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202063042822P 2020-06-23 2020-06-23
US202163165094P 2021-03-23 2021-03-23
US202163165469P 2021-03-24 2021-03-24
PCT/US2021/038780 WO2021262909A2 (en) 2020-06-23 2021-06-23 Lnp compositions comprising mrna therapeutics with extended half-life

Publications (1)

Publication Number Publication Date
MX2022016550A true MX2022016550A (es) 2023-04-10

Family

ID=77155849

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022016550A MX2022016550A (es) 2020-06-23 2021-06-23 Composiciones de lnp que comprenden agentes terapeuticos de mrna con semivida extendida.

Country Status (11)

Country Link
US (1) US20230242908A1 (es)
EP (1) EP4168556A2 (es)
JP (1) JP2023531511A (es)
KR (1) KR20230042005A (es)
CN (1) CN116194151A (es)
AU (1) AU2021297248A1 (es)
BR (1) BR112022025991A2 (es)
CA (1) CA3187261A1 (es)
IL (1) IL299196A (es)
MX (1) MX2022016550A (es)
WO (1) WO2021262909A2 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023183550A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Messenger ribonucleic acids with extended half-life
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia
WO2023196399A1 (en) * 2022-04-06 2023-10-12 Modernatx, Inc. Lipid nanoparticles and polynucleotides encoding argininosuccinate lyase for the treatment of argininosuccinic aciduria
WO2023215498A2 (en) * 2022-05-05 2023-11-09 Modernatx, Inc. Compositions and methods for cd28 antagonism

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8613481D0 (en) 1986-06-04 1986-07-09 Diatech Ltd Translation of mrna
WO1995014775A1 (en) 1993-11-26 1995-06-01 British Technology Group Limited Translational enhancer dna
US5824497A (en) 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
EP1430128B1 (en) 2001-09-28 2018-04-25 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Micro-rna molecules
US20050222064A1 (en) 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
EP2530157B1 (en) 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP2205618B1 (en) 2007-09-26 2016-11-09 Intrexon Corporation Synthetic 5'utrs, expression vectors, and methods for increasing transgene expression
CN102036652B (zh) 2008-04-25 2016-04-13 西北大学 适于螯合胆甾醇的纳米结构
PL215513B1 (pl) 2008-06-06 2013-12-31 Univ Warszawski Nowe boranofosforanowe analogi dinukleotydów, ich zastosowanie, czasteczka RNA, sposób otrzymywania RNA oraz sposób otrzymywania peptydów lub bialka
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
WO2012099755A1 (en) 2011-01-11 2012-07-26 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
WO2014113089A2 (en) 2013-01-17 2014-07-24 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
WO2014164253A1 (en) 2013-03-09 2014-10-09 Moderna Therapeutics, Inc. Heterologous untranslated regions for mrna
WO2015130584A2 (en) 2014-02-25 2015-09-03 Merck Sharp & Dohme Corp. Lipid nanoparticle vaccine adjuvants and antigen delivery systems
US11866754B2 (en) 2015-10-16 2024-01-09 Modernatx, Inc. Trinucleotide mRNA cap analogs
HUE059955T2 (hu) 2016-05-18 2023-01-28 Modernatx Inc Immunmoduláló polipeptideket kódoló MRNS-ek kombinációi és azok felhasználása
JP2022500436A (ja) * 2018-09-13 2022-01-04 モダーナティエックス・インコーポレイテッドModernaTX, Inc. 糖原病を処置するためのグルコース−６−ホスファターゼをコードするポリヌクレオチド
US20220401584A1 (en) * 2018-09-14 2022-12-22 Modernatx, Inc. Polynucleotides encoding uridine diphosphate glycosyltransferase 1 family, polypeptide a1 for the treatment of crigler-najjar syndrome
AU2021205337A1 (en) * 2020-01-10 2022-07-21 Modernatx, Inc. Methods of making tolerogenic dendritic cells
CA3168945A1 (en) * 2020-01-30 2021-08-05 Modernatx, Inc. Mrnas encoding metabolic reprogramming polypeptides and uses thereof

Also Published As

Publication number Publication date
JP2023531511A (ja) 2023-07-24
WO2021262909A3 (en) 2022-04-21
AU2021297248A1 (en) 2023-02-02
CN116194151A (zh) 2023-05-30
EP4168556A2 (en) 2023-04-26
KR20230042005A (ko) 2023-03-27
IL299196A (en) 2023-02-01
CA3187261A1 (en) 2021-12-30
WO2021262909A2 (en) 2021-12-30
US20230242908A1 (en) 2023-08-03
BR112022025991A2 (pt) 2023-03-14

Similar Documents

Publication Publication Date Title
MX2022016550A (es) Composiciones de lnp que comprenden agentes terapeuticos de mrna con semivida extendida.
FR21C1008I1 (fr) Arnm stabilise avec un contenu augmente en g/c, codant pour un antigene virale
MX2019013752A (es) Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
Slikker Jr et al. Behavioral and neurochemical effects of orally administered MDMA in the rodent and nonhuman primate.
MX2022006365A (es) Receptores de antigeno quimerico cd19 y cd22 y usos de los mismos.
IN2012DN03824A (es)
HK1143078A1 (en) Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
MX339277B (es) Agente inductor de inmunidad.
WO2008063382A3 (en) Antagonists of pcsk9
WO2008057457A3 (en) Antagonists of pcsk9
MY176331A (en) Carbohydrate degrading polypeptide and uses thereof
MX2021008131A (es) Metodos y composiciones para el tratamiento de la enfermedad de fabry.
MX2019009551A (es) Enzima lipolitica para usarse en horneado.
MX2020005785A (es) Formulaciones inyectables de toxina botulínica y métodos de uso de las mismas que tienen una alta tasa de respuesta y una larga duración del efecto.
AR049215A1 (es) Homologos de galactinol sintetasa en plantas
CY1111941T1 (el) Χρηση ενος μοριου νουκλεϊνικου οξεως το οποιο κωδικοποιει τον τομεα δισιντεγρινης μιας αδαμαλυσινης ως αντι-αγγειογονου, αντι-διεισδυτικου και αντι-μεταστατικου παραγοντα
MX2021010301A (es) Composiciones y métodos para el tratamiento de laminopatías.
IT1244634B (it) Composizione indurente per colle urea-formaldeide, procedimento per lasua produzione e kit che la comprende.
BR0209551A (pt) Promotor de gene especìfico de folha de café
EP1688498A4 (en) RECOMBINANT PROTEIN WITH CANCER SUPPRESSIVE EFFECT, ITS CODING GENE AND ITS USE
WO2019118727A3 (en) Rescue of the pathology of lrrk2 on lysosmes with snx25 or snx27
WO2023183550A3 (en) Messenger ribonucleic acids with extended half-life
MX2021012739A (es) Retrasar el efecto pico y/o prolongar la duracion de la respuesta.
MX2021000582A (es) Terapia genica de hemofilia a mediante el uso de vectores virales que codifican variantes del factor viii (fviii) recombinantes con mayor expresion.
MX2020008905A (es) Usos terapeuticos de activadores de glucoquinasa en combinacion con insulina o analogos de insulinas.