BR112022019606A2 - Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas - Google Patents

Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas

Info

Publication number
BR112022019606A2
BR112022019606A2 BR112022019606A BR112022019606A BR112022019606A2 BR 112022019606 A2 BR112022019606 A2 BR 112022019606A2 BR 112022019606 A BR112022019606 A BR 112022019606A BR 112022019606 A BR112022019606 A BR 112022019606A BR 112022019606 A2 BR112022019606 A2 BR 112022019606A2
Authority
BR
Brazil
Prior art keywords
mapt
compositions
methods
associated protein
irna agents
Prior art date
Application number
BR112022019606A
Other languages
English (en)
Portuguese (pt)
Inventor
Meenakshi Soundarapandian Mangala
D Mcininch James
Fishilevich Elane
Castoreno Adam
Kaittanis Charalambos
K Schlegel Mark
Edward Farley Jonathan
Zuber Jeffrey
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 BR112022019606A2 publication Critical patent/BR112022019606A2/pt

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
    • 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
    • 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
    • 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
    • C12N2310/312—Phosphonates
    • 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
    • C12N2310/3183—Diol linkers, e.g. glycols or propanediols
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
BR112022019606A 2020-03-30 2021-03-30 Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas BR112022019606A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063002030P 2020-03-30 2020-03-30
US202163164467P 2021-03-22 2021-03-22
PCT/US2021/024858 WO2021202511A2 (en) 2020-03-30 2021-03-30 MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

Publications (1)

Publication Number Publication Date
BR112022019606A2 true BR112022019606A2 (pt) 2022-11-16

Family

ID=77929772

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022019606A BR112022019606A2 (pt) 2020-03-30 2021-03-30 Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas

Country Status (12)

Country Link
US (1) US20230203486A1 (https=)
EP (1) EP4126230A4 (https=)
JP (1) JP2023521604A (https=)
KR (1) KR20230005194A (https=)
CN (1) CN116234585A (https=)
AU (1) AU2021246024A1 (https=)
BR (1) BR112022019606A2 (https=)
CA (1) CA3178304A1 (https=)
IL (1) IL296851A (https=)
MX (1) MX2022012293A (https=)
TW (1) TW202143984A (https=)
WO (1) WO2021202511A2 (https=)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240067943A (ko) * 2021-09-24 2024-05-17 알닐람 파마슈티칼스 인코포레이티드 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법
US20250340872A1 (en) * 2021-10-07 2025-11-06 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing tau expression
EP4448765A1 (en) * 2021-12-13 2024-10-23 Eli Lilly and Company Mapt rna interference agents
JP2025507372A (ja) * 2022-02-11 2025-03-18 アルナイラム ファーマシューティカルズ， インコーポレイテッド 微小管関連タンパク質タウ（ＭＡＰＴ）ｉＲＮＡ剤組成物およびその使用方法
EP4493691A2 (en) * 2022-03-16 2025-01-22 JANSSEN Pharmaceutica NV Mapt sirna and uses thereof
KR20250129743A (ko) * 2022-12-29 2025-08-29 보이저 테라퓨틱스, 인크. Mapt를 조절하기 위한 조성물 및 방법
WO2024201423A2 (en) * 2023-03-30 2024-10-03 Biorchestra Co., Ltd. Microtubule-associated protein tau targeting sirnas and uses thereof
WO2024220930A2 (en) * 2023-04-20 2024-10-24 Adarx Pharmaceuticals, Inc. Mapt-modulating compositions and methods of use thereof
AU2024315297A1 (en) * 2023-08-01 2026-02-12 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna targeting mapt and pharmaceutical use thereof
WO2025027577A1 (en) * 2023-08-02 2025-02-06 Consejo Nacional De Investigaciones Científicas Y Técnicas (Conicet) Micrornas designed against the microtubule-associated protein tau for the treatment of tauopathies
TW202515584A (zh) * 2023-10-11 2025-04-16 大陸商上海舶望製藥有限公司 用於抑制微管相關蛋白ｔａｕ（ｍａｐｔ）表達的組合物和方法
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2025233849A2 (en) * 2024-05-07 2025-11-13 Janssen Pharmaceutica Nv MAPT siRNA AND USES THEREOF
WO2026010722A1 (en) * 2024-07-03 2026-01-08 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2026017176A1 (zh) * 2024-07-19 2026-01-22 北京安龙生物医药有限公司 靶向微管相关蛋白Tau基因的寡核苷酸及其用途
CN121780524A (zh) * 2024-08-02 2026-04-03 北京尧景基因技术有限公司 抑制MAPT基因表达的siRNA及其缀合物和应用
WO2026067655A1 (zh) * 2024-09-26 2026-04-02 大睿生物医药科技(上海)有限公司 调控TAU表达的dsRNA分子

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8101741B2 (en) * 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
GB0605337D0 (en) * 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
AU2013203395A1 (en) * 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
US9133461B2 (en) * 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
ES2807379T3 (es) * 2013-03-14 2021-02-22 Ionis Pharmaceuticals Inc Composiciones y métodos para regular la expresión de Tau
JP2016523980A (ja) * 2013-07-11 2016-08-12 ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク タウ発現を抑制するマイクロｒｎａ
TWI772856B (zh) * 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
EP3043826A4 (en) * 2013-09-13 2017-05-24 Moderna Therapeutics, Inc. Polynucleotide compositions containing amino acids
IL316808A (en) * 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
AU2016215155A1 (en) * 2015-02-04 2017-08-17 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
EP3274456B1 (en) * 2015-03-25 2020-06-24 Università Degli Studi Di Trento Rna interference mediated therapy for neurodegenerative diseases
CN108135921B (zh) * 2015-07-22 2023-10-17 波涛生命科学有限公司 寡核苷酸组合物及其方法
PH12018501207B1 (en) * 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression
CA3086485A1 (en) * 2017-12-21 2019-06-27 Alnylam Pharmaceuticals, Inc. Chirally-enriched double-stranded rna agents
SG11202012759XA (en) * 2018-07-03 2021-01-28 Hoffmann La Roche Oligonucleotides for modulating tau expression
WO2021156833A1 (en) * 2020-02-07 2021-08-12 Biorchestra Co., Ltd. Use of mirna-485 inhibitors for treating tauopathy
AU2021238319A1 (en) * 2020-03-18 2022-10-06 University Of Massachusetts Oligonucleotides for MAPT modulation

Also Published As

Publication number Publication date
AU2021246024A1 (en) 2022-10-27
EP4126230A2 (en) 2023-02-08
WO2021202511A2 (en) 2021-10-07
IL296851A (en) 2022-11-01
US20230203486A1 (en) 2023-06-29
MX2022012293A (es) 2022-10-27
TW202143984A (zh) 2021-12-01
WO2021202511A3 (en) 2021-11-25
KR20230005194A (ko) 2023-01-09
CA3178304A1 (en) 2021-10-07
JP2023521604A (ja) 2023-05-25
EP4126230A4 (en) 2025-07-30
CN116234585A (zh) 2023-06-06

Similar Documents

Publication Publication Date Title
BR112022019606A2 (pt) Composições de agentes de irna para a proteína associada a microtúbulos tau (mapt) e métodos de uso das mesmas
BR112022021813A2 (pt) Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
CO2024003968A2 (es) Composiciones de agentes de arni de proteína tau asociada a microtúbulos (mapt) y sus métodos de uso
BR112021005769A2 (pt) proteínas de ligação a dll3 e métodos de uso
Goubau et al. Antiviral immunity via RIG-I-mediated recognition of RNA bearing 5′-diphosphates
BR112023016299A2 (pt) Composições e métodos para inibir kras
BR112019010342A2 (pt) composições do irna de serpina1 e métodos de uso destas
BR112022011417A2 (pt) Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas
MX2023001538A (es) Composiciones y metodos para inhibir la expresion de lpa.
PE20210518A1 (es) Compuestos y metodos para la reduccion de la expresion de lrrk2
BRPI0415622A (pt) linhas de células aviárias importalizadas para produção de vìrus
BR112023027446A2 (pt) Composições de agente irna de quinase 2 de repetição rica em leucina (lrrk2) e métodos de uso das mesmas
EP4219713A3 (en) Products and compositions
BR112023019981A2 (pt) Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas
BR112019008810A2 (pt) micrornas modificadas com 5-halouracila e seu uso no tratamento de câncer
BR112022001324A2 (pt) Composto, composição farmacêutica e método para tratamento de uma doença mediada por p38 map quinase em um indivíduo necessitando do mesmo
BR112018077358A2 (pt) tagatose-6-fosfato fosfatase termoestável inovadora e um método para produzir tagatose com o uso da mesma
BR112021025781A2 (pt) Composto, composição farmacêutica, método de tratamento de uma doença ou transtorno associado com um defeito no metabolismo de glioxilato, método de tratamento de uma doença ou transtorno associado com a enzima glicolato oxidase (go) ou alterações no metabolismo de oxalato e método para inibição da enzima go
BR112018011059A2 (pt) synp161, um promotor para a expressão específica de genes em fotoreceptores de haste
BR112023001456A2 (pt) Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
Folsom et al. Protein expression of targets of the FMRP regulon is altered in brains of subjects with schizophrenia and mood disorders
BR112022012230A2 (pt) Variantes de progranulina
MX2023007319A (es) Vacunas de acido nucleico.
BR112021020727A2 (pt) Materiais e métodos para produção de proteína
BR112017014433A2 (pt) proteína dnase i modificada, processo para preparar proteína dnase i modificada e composição farmacêutica