MX2024000065A - Composiciones del agente de arni de la cinasa de repeticion 2 rica en leucina (lrrk2) y metodos de uso de estas. - Google Patents

Composiciones del agente de arni de la cinasa de repeticion 2 rica en leucina (lrrk2) y metodos de uso de estas.

Info

Publication number
MX2024000065A
MX2024000065A MX2024000065A MX2024000065A MX2024000065A MX 2024000065 A MX2024000065 A MX 2024000065A MX 2024000065 A MX2024000065 A MX 2024000065A MX 2024000065 A MX2024000065 A MX 2024000065A MX 2024000065 A MX2024000065 A MX 2024000065A
Authority
MX
Mexico
Prior art keywords
lrrk2
methods
leucine
rich repeat
repeat kinase
Prior art date
Application number
MX2024000065A
Other languages
English (en)
Spanish (es)
Inventor
Mark K Schlegel
James D Mcininch
Adam Castoreno
Lan Thi Hoang Dang
Sarah Leblanc
Tuyen M Nguyen
Joseph Barry
Matthew Stricos
Original Assignee
Alnylam Pharmaceuticals 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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of MX2024000065A publication Critical patent/MX2024000065A/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/318Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525MOE, methoxyethoxy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527Other alkyl chain
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/91Transferases (2.)
    • G01N2333/912Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2835Movement disorders, e.g. Parkinson, Huntington, Tourette

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Analytical Chemistry (AREA)
  • Cell Biology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Food Science & Technology (AREA)
MX2024000065A 2021-06-29 2022-06-29 Composiciones del agente de arni de la cinasa de repeticion 2 rica en leucina (lrrk2) y metodos de uso de estas. MX2024000065A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163216119P 2021-06-29 2021-06-29
US202263353953P 2022-06-21 2022-06-21
PCT/US2022/035561 WO2023278607A1 (en) 2021-06-29 2022-06-29 Leucine-rich repeat kinase 2 (lrrk2) irna agent compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2024000065A true MX2024000065A (es) 2024-02-23

Family

ID=84692073

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024000065A MX2024000065A (es) 2021-06-29 2022-06-29 Composiciones del agente de arni de la cinasa de repeticion 2 rica en leucina (lrrk2) y metodos de uso de estas.

Country Status (13)

Country Link
US (1) US20240301426A1 (https=)
EP (1) EP4363588A1 (https=)
JP (1) JP2024528503A (https=)
KR (1) KR20240033237A (https=)
AU (1) AU2022303358A1 (https=)
BR (1) BR112023027446A2 (https=)
CA (1) CA3225740A1 (https=)
CL (3) CL2023003984A1 (https=)
CO (1) CO2023018579A2 (https=)
IL (1) IL309548A (https=)
MX (1) MX2024000065A (https=)
TW (1) TW202317149A (https=)
WO (1) WO2023278607A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024238385A2 (en) * 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN121712891A (zh) * 2023-06-20 2026-03-20 阿达尔克斯制药有限公司 Lrrk2调节组合物及其使用方法
AU2024353921A1 (en) 2023-09-27 2026-04-16 Neuro3 Therapeutics (Suzhou) Ltd. Novel sirna constructs, therapeutics, and modifications
CN120082552A (zh) * 2023-12-01 2025-06-03 艾码生物科技(南京)有限公司 初级-miRNA及其应用

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014110205A1 (en) * 2013-01-11 2014-07-17 Children's Medical Center Corporation Methods and compositions for the production of sirnas
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法

Also Published As

Publication number Publication date
CL2025000168A1 (es) 2025-05-30
KR20240033237A (ko) 2024-03-12
JP2024528503A (ja) 2024-07-30
WO2023278607A1 (en) 2023-01-05
CL2025003326A1 (es) 2026-02-20
IL309548A (en) 2024-02-01
TW202317149A (zh) 2023-05-01
AU2022303358A1 (en) 2024-01-18
BR112023027446A2 (pt) 2024-03-12
CL2023003984A1 (es) 2024-08-16
EP4363588A1 (en) 2024-05-08
CA3225740A1 (en) 2023-01-05
US20240301426A1 (en) 2024-09-12
CO2023018579A2 (es) 2024-01-15

Similar Documents

Publication Publication Date Title
MX2024000065A (es) Composiciones del agente de arni de la cinasa de repeticion 2 rica en leucina (lrrk2) y metodos de uso de estas.
WO2021150969A8 (en) LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
MX2023011466A (es) Composiciones de agentes de ácido ribonucleico de interferencia (arni) de huntingtina (htt) y métodos de uso de estas.
WO2021222065A8 (en) Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
MX2020009812A (es) COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS.
MX2024000981A (es) Composiciones de acido ribonucleico de interferencia (arni) de gen diana asociado con trastorno metabolico y sus metodos de uso.
WO2021096763A8 (en) Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
MX2024003519A (es) Composiciones de agentes de acido ribonucleico de interferencia (arni) de proteina tau asociada a microtubulos (mapt) y sus metodos de uso.
WO2016201301A8 (en) Complement component c5 irna compositions and methods of use thereof
ZA202405869B (en) Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof
MX2024002440A (es) Composiciones de acido ribonucleico de interferencia (arni) del efector b similar al factor de fragmentacion de adn subunidad alfa (dffa) que induce la muerte celular (cideb) y metodos de uso de estas.
MX2025001351A (es) Arn bicatenario con acción selectiva al angiotensinógeno (agt), y métodos para el uso del mismo
MY203259A (en) 5-halouracil-modified micrornas and their use in the treatment of cancer
WO2021099838A9 (en) Adenosine receptor antagonist compounds
WO2023076450A3 (en) HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2025096970A3 (en) Modified oligonucleotides and methods of use thereof
MX2023014168A (es) Marco de lectura abierto 72 del cromosoma humano 9 (c9orf72) composiciones de agente de arni y metodos de uso de este.
WO2021262919A3 (en) 5-halouracil-modified micrornas and their use in the treatment of cancer
MY181555A (en) 5?-dibenzyl phosphates of 5-azacytidine or 2?-deoxy-5-azacytidine
MX2025012729A (es) Composiciones de mivelsiran y metodos de uso de las mismas
AR121138A1 (es) COMPOSICIONES DE AGENTE DE iRNA DE CINASAS 2 RICAS EN REPETICIONES DE LEUCINAS (LRRK2) Y MÉTODOS DE SU USO
WO2021252523A3 (en) TUMOR NECROSIS FACTOR RECEPTOR ASSOCIATED FACTOR 6 (TRAF6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2025259747A3 (en) Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof
EA201070055A1 (ru) N-оксиды венлафаксина и о-десметил венлафаксина в качестве пролекарств
WO2023056478A8 (en) iRNA COMPOSITIONS AND METHODS FOR TARGETING ANGPTL7