MX2022007144A - Composiciones de acido ribonucleico de interferencia (arni) que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus metodos de uso. - Google Patents

Composiciones de acido ribonucleico de interferencia (arni) que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus metodos de uso.

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Publication number
MX2022007144A
MX2022007144A MX2022007144A MX2022007144A MX2022007144A MX 2022007144 A MX2022007144 A MX 2022007144A MX 2022007144 A MX2022007144 A MX 2022007144A MX 2022007144 A MX2022007144 A MX 2022007144A MX 2022007144 A MX2022007144 A MX 2022007144A
Authority
MX
Mexico
Prior art keywords
pnpla3
methods
patatin
domain containing
phospholipase domain
Prior art date
Application number
MX2022007144A
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English (en)
Inventor
Mark K Schlegel
James D Mcininch
Frederic Tremblay
Adam Castoreno
Charalambos Kaittanis
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
Priority claimed from PCT/US2020/064776 external-priority patent/WO2021126734A1/en
Publication of MX2022007144A publication Critical patent/MX2022007144A/es

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    • 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
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    • 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
    • A—HUMAN NECESSITIES
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    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02—Inorganic compounds
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
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    • 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
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    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Wood Science & Technology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Inorganic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (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)
  • Enzymes And Modification Thereof (AREA)

Abstract

Agentes de ARNi, por ejemplo, agentes de ARN de hebra doble (ARNhd), dirigidos al gen que contiene un dominio fosfolipasa tipo patatina 3 (PNPLA3). La invención también se relaciona con métodos de uso de los agentes de ARNi para inhibir la expresión de un gen PNPLA3 y con métodos para prevenir y tratar un tastorno asociado a PNPLA3, por ejemplo, la enfermedad de hígado graso no alcohólico (NAFLD).
MX2022007144A 2019-12-16 2020-12-14 Composiciones de acido ribonucleico de interferencia (arni) que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus metodos de uso. MX2022007144A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962948445P 2019-12-16 2019-12-16
US202063040602P 2020-06-18 2020-06-18
PCT/US2020/064776 WO2021126734A1 (en) 2019-12-16 2020-12-14 Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
MX2022007144A true MX2022007144A (es) 2022-07-11

Family

ID=76437193

Family Applications (1)

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MX2022007144A MX2022007144A (es) 2019-12-16 2020-12-14 Composiciones de acido ribonucleico de interferencia (arni) que contienen un dominio fosfolipasa tipo patatina 3 (pnpla3) y sus metodos de uso.

Country Status (11)

Country Link
US (2) US11149276B2 (es)
EP (1) EP4077666A1 (es)
JP (1) JP2023506258A (es)
KR (1) KR20220116520A (es)
CN (1) CN115176007A (es)
AU (1) AU2020404905A1 (es)
BR (1) BR112022011412A2 (es)
CA (1) CA3164628A1 (es)
CO (1) CO2022008105A2 (es)
IL (1) IL293860A (es)
MX (1) MX2022007144A (es)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023519246A (ja) * 2020-03-26 2023-05-10 アローヘッド ファーマシューティカルズ インコーポレイテッド ＰＮＰＬＡ３の発現を阻害するためのＲＮＡｉ薬、その医薬組成物、及び使用方法
WO2024120412A1 (en) * 2022-12-07 2024-06-13 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of patatin-like phospholipase domain-containing 3 (pnpla3)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
KR102689177B1 (ko) 2011-11-18 2024-07-30 알닐람 파마슈티칼스 인코포레이티드 변형된 ＲＮＡｉ 제제
PE20142362A1 (es) 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2017048620A1 (en) 2015-09-14 2017-03-23 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting patatin-like phospholipase domain containing 3 (pnpla3) and methods of use thereof

Also Published As

Publication number Publication date
EP4077666A1 (en) 2022-10-26
AU2020404905A1 (en) 2022-06-23
US20220348926A1 (en) 2022-11-03
US20210189400A1 (en) 2021-06-24
CA3164628A1 (en) 2021-06-24
BR112022011412A2 (pt) 2022-08-30
US11149276B2 (en) 2021-10-19
CO2022008105A2 (es) 2022-07-08
CN115176007A (zh) 2022-10-11
IL293860A (en) 2022-08-01
KR20220116520A (ko) 2022-08-23
JP2023506258A (ja) 2023-02-15

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