CL2023003984A1 - Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina - Google Patents

Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina

Info

Publication number
CL2023003984A1
CL2023003984A1 CL2023003984A CL2023003984A CL2023003984A1 CL 2023003984 A1 CL2023003984 A1 CL 2023003984A1 CL 2023003984 A CL2023003984 A CL 2023003984A CL 2023003984 A CL2023003984 A CL 2023003984A CL 2023003984 A1 CL2023003984 A1 CL 2023003984A1
Authority
CL
Chile
Prior art keywords
leucine
rich repeat
arni
repeat kinase
agent compositions
Prior art date
Application number
CL2023003984A
Other languages
English (en)
Spanish (es)
Inventor
Castoreno Adam
K Schlegel Mark
D Mcininch James
Thi Hoang Dang Lan
M Nguyen Tuyen
Barry Joseph
Stricos Matthew
Leblanc Sarah
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 CL2023003984A1 publication Critical patent/CL2023003984A1/es

Links

Classifications

    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N15/1137—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 against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/53—Immunoassay; Biospecific binding assay; Materials therefor
    • G01N33/573—Immunoassay; Biospecific binding assay; Materials therefor for enzymes or isoenzymes
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527—Other alkyl chain
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/91—Transferases (2.)
    • G01N2333/912—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
    • G01N2800/2835—Movement 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)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Food Science & Technology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Ophthalmology & Optometry (AREA)
  • General Physics & Mathematics (AREA)
CL2023003984A 2021-06-29 2023-12-29 Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina CL2023003984A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163216119P 2021-06-29 2021-06-29
US202263353953P 2022-06-21 2022-06-21

Publications (1)

Publication Number Publication Date
CL2023003984A1 true CL2023003984A1 (es) 2024-08-16

Family

ID=84692073

Family Applications (3)

Application Number Title Priority Date Filing Date
CL2023003984A CL2023003984A1 (es) 2021-06-29 2023-12-29 Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina
CL2025000168A CL2025000168A1 (es) 2021-06-29 2025-01-20 Composiciones de agentes arni de lrrk2 y sus usos
CL2025003326A CL2025003326A1 (es) 2021-06-29 2025-10-28 Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina

Family Applications After (2)

Application Number Title Priority Date Filing Date
CL2025000168A CL2025000168A1 (es) 2021-06-29 2025-01-20 Composiciones de agentes arni de lrrk2 y sus usos
CL2025003326A CL2025003326A1 (es) 2021-06-29 2025-10-28 Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina

Country Status (13)

Country Link
US (1) US20240301426A1 (enExample)
EP (1) EP4363588A4 (enExample)
JP (1) JP2024528503A (enExample)
KR (1) KR20240033237A (enExample)
AU (1) AU2022303358A1 (enExample)
BR (1) BR112023027446A2 (enExample)
CA (1) CA3225740A1 (enExample)
CL (3) CL2023003984A1 (enExample)
CO (1) CO2023018579A2 (enExample)
IL (1) IL309548A (enExample)
MX (1) MX2024000065A (enExample)
TW (1) TW202317149A (enExample)
WO (1) WO2023278607A1 (enExample)

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
CN122074089A (zh) 2023-09-27 2026-05-22 三晟医药公司 新型siRNA构建体、治疗剂和修饰
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 (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007124096A2 (en) * 2006-04-21 2007-11-01 The Trustees Of Columbia University In The City Of New York Lrrk2 regulaton of neuronal process morphology
GB201105137D0 (en) * 2011-03-28 2011-05-11 Isis Innovation Therapeutic molecules for use in the suppression of Parkinson's disease
WO2014110205A1 (en) * 2013-01-11 2014-07-17 Children's Medical Center Corporation Methods and compositions for the production of sirnas
EP3400300A4 (en) * 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. PROCESS FOR REDUCING LRRK2 EXPRESSION
CA3098623A1 (en) * 2018-05-07 2019-11-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
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
EP3769768A1 (en) * 2019-07-23 2021-01-27 Consejo Superior de Investigaciones Cientificas (CSIC) Compounds useful for the treatment of parkinson s disease

Also Published As

Publication number Publication date
EP4363588A4 (en) 2026-05-13
TW202317149A (zh) 2023-05-01
AU2022303358A1 (en) 2024-01-18
IL309548A (en) 2024-02-01
KR20240033237A (ko) 2024-03-12
US20240301426A1 (en) 2024-09-12
JP2024528503A (ja) 2024-07-30
CL2025000168A1 (es) 2025-05-30
CO2023018579A2 (es) 2024-01-15
CA3225740A1 (en) 2023-01-05
MX2024000065A (es) 2024-02-23
EP4363588A1 (en) 2024-05-08
BR112023027446A2 (pt) 2024-03-12
CL2025003326A1 (es) 2026-02-20
WO2023278607A1 (en) 2023-01-05

Similar Documents

Publication Publication Date Title
CL2025003326A1 (es) Composiciones del agente de arni de la cinasa de repetición 2 rica en leucina
CL2021002326A1 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso (divisional de solicitud n° 202002865)
CL2021001490A1 (es) Constructos de iarn modificados químicamente y usos de estos
MX2025007610A (es) Composiciones de arni de 17beta-hidroxiesteroide deshidrogenasa tipo 13 (hsd17b13) y metodos de uso de las mismas
CR20210393A (es) COMPOSICIONES DE AGENTE DE ARNi DE PROTEÍNA PRECURSORA DE AMILOIDE (APP) Y MÉTODO DE USO DE LAS MISMAS
MX2023008469A (es) Oligonucleotidos de doble hebra modificados.
CO2018006590A2 (es) Métodos y composiciones para el tratamiento de un trastorno asociado a serpinc1
CL2021002998A1 (es) Composiciones de arni contra tmprss6 y métodos para su uso (divisional de solicitud n° 03440-2015)
BR112022009216A2 (pt) Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
MX382225B (es) Composiciones y metodos para inhibir la expresion del gen de hidroxiacido-oxidasa 1 (glicolato-oxidasa) (hao1).
CL2019000107A1 (es) Composiciones de arni contra el componente c5 del complemento y métodos para su uso.(divisional solicitud 201702014)
CL2024001319A1 (es) Composiciones de arni del factor b del complemento (cfb) y métodos de uso
CO2021002691A2 (es) Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1)
AR120341A1 (es) COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
CL2024000858A1 (es) Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt)
MX2022009026A (es) Composiciones de agentes de arni de cinasa de repeticion rica en leucina 2 (lrrk2) y metodos de uso de las mismas.
UY36671A (es) COMPOSICIONES DE ARNi CONTRA Factor XII (FACTOR DE Hageman) (F12),CALICREÍNA B, PLASMÁTICA (FACTOR DE FLETCHER) 1 (KLKB1), Y QUININÓGENO 1 (KNG1) Y MÉTODOS DE USO DE LOS MISMO
BR112014011896A2 (pt) agentes de rnai, composições e métodos de uso destes para o tratamento de doenças associadas à transtirretina (ttr)
AR102777A1 (es) COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO
CL2021001488A1 (es) Transposasa de piggybac mutada
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.
BR112019008810A2 (pt) micrornas modificadas com 5-halouracila e seu uso no tratamento de câncer
AR121138A1 (es) COMPOSICIONES DE AGENTE DE iRNA DE CINASAS 2 RICAS EN REPETICIONES DE LEUCINAS (LRRK2) Y MÉTODOS DE SU USO
CL2022000539A1 (es) Composiciones y métodos para inhibir la expresión del gen lect2
CL2025003306A1 (es) Composiciones de mivelsiran y métodos de uso de estas