MX2024003412A - Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia. - Google Patents

Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia.

Info

Publication number
MX2024003412A
MX2024003412A MX2024003412A MX2024003412A MX2024003412A MX 2024003412 A MX2024003412 A MX 2024003412A MX 2024003412 A MX2024003412 A MX 2024003412A MX 2024003412 A MX2024003412 A MX 2024003412A MX 2024003412 A MX2024003412 A MX 2024003412A
Authority
MX
Mexico
Prior art keywords
mrna
haploinsufficiency
regulon
disorders
treatment
Prior art date
Application number
MX2024003412A
Other languages
English (en)
Spanish (es)
Inventor
Jeffery Coller
Original Assignee
Univ Johns Hopkins
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 Univ Johns Hopkins filed Critical Univ Johns Hopkins
Publication of MX2024003412A publication Critical patent/MX2024003412A/es

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
    • A61K48/005—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
    • 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
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/1025—Acyltransferases (2.3)
    • C12N9/1029—Acyltransferases (2.3) transferring groups other than amino-acyl groups (2.3.1)
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/095—Fusion polypeptide containing a localisation/targetting motif containing a nuclear export signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Marine Sciences & Fisheries (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
MX2024003412A 2021-09-21 2022-09-20 Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia. MX2024003412A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163246689P 2021-09-21 2021-09-21
US202263352530P 2022-06-15 2022-06-15
PCT/US2022/076723 WO2023049707A1 (en) 2021-09-21 2022-09-20 Mrna regulon therapy for the treatment of haploinsufficiency disorders

Publications (1)

Publication Number Publication Date
MX2024003412A true MX2024003412A (es) 2024-04-08

Family

ID=85719677

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024003412A MX2024003412A (es) 2021-09-21 2022-09-20 Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia.

Country Status (8)

Country Link
US (1) US20250135035A1 (https=)
EP (1) EP4404953A4 (https=)
JP (1) JP2024534496A (https=)
KR (1) KR20240099184A9 (https=)
AU (1) AU2022349620A1 (https=)
CA (1) CA3232214A1 (https=)
MX (1) MX2024003412A (https=)
WO (1) WO2023049707A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023212687A1 (en) * 2022-04-29 2023-11-02 The Johns Hopkins University Systems for enhancing mrna expression and uses thereof
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015221026A (ja) * 2014-05-23 2015-12-10 公立大学法人名古屋市立大学 人工合成ｍＲＮＡの翻訳効率化方法
EP3365454A1 (en) * 2015-10-20 2018-08-29 10X Genomics, Inc. Methods and systems for high throughput single cell genetic manipulation
US11730828B2 (en) * 2017-02-07 2023-08-22 The Regents Of The University Of California Gene therapy for haploinsufficiency
KR102318434B1 (ko) * 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CN115023499A (zh) * 2019-08-16 2022-09-06 麻省理工学院 使用CRISPR/Cas13的靶向反式剪接

Also Published As

Publication number Publication date
CA3232214A1 (en) 2023-03-30
WO2023049707A1 (en) 2023-03-30
US20250135035A1 (en) 2025-05-01
JP2024534496A (ja) 2024-09-20
EP4404953A1 (en) 2024-07-31
KR20240099184A (ko) 2024-06-28
EP4404953A4 (en) 2025-09-24
AU2022349620A1 (en) 2024-03-21
KR20240099184A9 (ko) 2025-12-10

Similar Documents

Publication Publication Date Title
MX2021014007A (es) Proteinas de union a epcam y metodos de uso.
BR112021005769A2 (pt) proteínas de ligação a dll3 e métodos de uso
AU2018338314A1 (en) Protein degraders and uses thereof
CL2024004048A1 (es) Proteínas diseñadas y métodos de uso de las mismas
BR112021025964A2 (pt) Formas de sal de ácido bempedoico e métodos para uso das mesmas
WO2020198509A3 (en) Modified oligonucleotides with increased stability
EP4442831A3 (en) Cationic lipids comprising a steroidal moiety
MX2024003412A (es) Terapia de regulon de arnm para el tratamiento de los trastornos por haploinsuficiencia.
EP4494706A3 (en) Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
MX2022004937A (es) Inhibidores de las cinasas raf.
BR112017021869A2 (pt) compostos quinazolina substituídos e métodos de uso dos mesmos
MX2025008039A (es) Uso de oligonucleótidos antisentido multisegmentados modificados
ECSP077315A (es) Proteínas de fusión de dominio de unión
BR0008188A (pt) Inibidores de fator de diferenciação de crescimento e usos para os mesmos
BR112021019109A2 (pt) Célula rpe manipulada, composições, molécula isolada de dna de fita dupla, dispositivo implantável, cápsula de hidrogel, e, método para aplicar uma terapia com fvii
CO2020000374A2 (es) Proceso mejorado para preparar derivados de aminopirimidina
WO2019217397A3 (en) Compositions and methods for improving strand biased
BR112022007158A2 (pt) Agentes imunomodulatórios de il-2 em combinação com inibidores do ponto de verificação imunológico
MX2022015769A (es) Tubulisinas y conjugados de proteina-tubulisina.
BR112022024501A2 (pt) Construtos de rnai para inibir a expressão de hsd17b13 e métodos de uso dos mesmos
CO2022006087A2 (es) Métodos para el tratamiento de la trombocitopenia inmunitaria mediante la administración de (r)-2-[3-[4-amino-3-(2-fluoro-4-fenoxi-fenil)pirazolo[3,4-d]pirimidin-1-il]piperidino-1-carbonil]-4-metil-4-[4-(oxetan-3-il)piperazin-1-il]pent-2-enonitrilo
BR112021024080A2 (pt) Construtos de rnai para inibir a expressão de scap e métodos de uso dos mesmos
EA202090700A1 (ru) Компетентные по репликации аденовирусные векторы
CL2019003248A1 (es) Disminución de homocisteína mediada por enzimas humanas para el tratamiento de pacientes con hiperhomocisteinemia y homocistinuria.
CL2025001963A1 (es) Proteína triespecífica dirigida a trop2 para el tratamiento del cáncer.