MX2021009754A - Metodos y composiciones para inhibir la expresion de cyp27a1. - Google Patents

Metodos y composiciones para inhibir la expresion de cyp27a1.

Info

Publication number
MX2021009754A
MX2021009754A MX2021009754A MX2021009754A MX2021009754A MX 2021009754 A MX2021009754 A MX 2021009754A MX 2021009754 A MX2021009754 A MX 2021009754A MX 2021009754 A MX2021009754 A MX 2021009754A MX 2021009754 A MX2021009754 A MX 2021009754A
Authority
MX
Mexico
Prior art keywords
cyp27a1
compositions
methods
oligonucleotides
inhibiting expression
Prior art date
Application number
MX2021009754A
Other languages
English (en)
Spanish (es)
Inventor
Utsav Saxena
Original Assignee
Dicerna 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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of MX2021009754A publication Critical patent/MX2021009754A/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
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/15Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced iron-sulfur protein as one donor, and incorporation of one atom of oxygen (1.14.15)
    • 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
    • C12N2310/11Antisense
    • 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
    • C12N2310/32Chemical 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
    • C12N2310/35Nature 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; 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)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
MX2021009754A 2019-02-12 2020-02-07 Metodos y composiciones para inhibir la expresion de cyp27a1. MX2021009754A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962804410P 2019-02-12 2019-02-12
PCT/US2020/017129 WO2020167593A1 (en) 2019-02-12 2020-02-07 Methods and compositions for inhibiting expression of cyp27a1

Publications (1)

Publication Number Publication Date
MX2021009754A true MX2021009754A (es) 2021-09-08

Family

ID=69941447

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021009754A MX2021009754A (es) 2019-02-12 2020-02-07 Metodos y composiciones para inhibir la expresion de cyp27a1.

Country Status (13)

Country Link
US (1) US20220186229A1 (https=)
EP (1) EP3908661A1 (https=)
JP (1) JP2022520653A (https=)
KR (1) KR20210132661A (https=)
CN (1) CN113692444A (https=)
AU (1) AU2020221892A1 (https=)
BR (1) BR112021015651A2 (https=)
CA (1) CA3128059A1 (https=)
CL (2) CL2021002120A1 (https=)
IL (1) IL285367A (https=)
MX (1) MX2021009754A (https=)
SG (1) SG11202108532RA (https=)
WO (1) WO2020167593A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2024269205A1 (en) * 2023-05-11 2025-12-11 Keymed Biosciences (Us) Inc. Dsrna molecules for regulating masp2 gene activity

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT1309726E (pt) 2000-03-30 2010-03-08 Whitehead Biomedical Inst Mediadores de interferência por rna específicos de sequência de rna
AU2002213489A1 (en) * 2000-10-13 2002-04-22 Genaissance Pharmaceuticals, Inc. Haplotypes of the cyp27a1 gene
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
JP2005508196A (ja) 2001-11-07 2005-03-31 アプレラ コーポレイション 核酸分析の汎用ヌクレオチド
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
WO2007030167A1 (en) 2005-09-02 2007-03-15 Nastech Pharmaceutical Company Inc. Modification of double-stranded ribonucleic acid molecules
JP2012504389A (ja) * 2008-09-22 2012-02-23 ダイセルナ ファーマシューティカルズ, インコーポレイテッド 修飾を有するdsRNAによる遺伝子発現の特異的な阻害のための組成物および方法
US8691971B2 (en) 2008-09-23 2014-04-08 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating RNA interference
US20100249214A1 (en) 2009-02-11 2010-09-30 Dicerna Pharmaceuticals Multiplex dicer substrate rna interference molecules having joining sequences
AU2009336191B2 (en) 2008-12-18 2017-08-24 Novo Nordisk A/S Extended dicer substrate agents and methods for the specific inhibition of gene expression
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
JP2015501155A (ja) 2011-10-25 2015-01-15 アイシス ファーマシューティカルズ, インコーポレーテッド Gccr発現のアンチセンス調整
EP2928877B1 (en) 2012-12-06 2020-01-22 Merck Sharp & Dohme Corp. Disulfide masked prodrug compositions and methods
JP2017522046A (ja) 2014-06-06 2017-08-10 ソルスティス バイオロジクス,リミティッド 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物
JP7105065B2 (ja) * 2014-12-15 2022-07-22 ダイセルナ ファーマシューティカルズ, インコーポレイテッド リガンド修飾二本鎖核酸
KR101916583B1 (ko) * 2017-04-20 2018-11-07 가천대학교 산학협력단 칼피르조밉을 유효성분으로 함유하는 담석증의 예방 또는 치료용 약학적 조성물

Also Published As

Publication number Publication date
IL285367A (en) 2021-09-30
US20220186229A1 (en) 2022-06-16
AU2020221892A1 (en) 2021-08-19
CL2021002120A1 (es) 2022-04-01
SG11202108532RA (en) 2021-09-29
BR112021015651A2 (pt) 2021-10-05
WO2020167593A1 (en) 2020-08-20
CA3128059A1 (en) 2020-08-20
JP2022520653A (ja) 2022-03-31
KR20210132661A (ko) 2021-11-04
CN113692444A (zh) 2021-11-23
CL2023003914A1 (es) 2024-07-12
EP3908661A1 (en) 2021-11-17

Similar Documents

Publication Publication Date Title
MX2020011006A (es) Pcsk9 dirigido a oligonucleótidos para el tratamiento de la hipercolesterolemia y afecciones relacionadas.
MX2020000154A (es) Composiciones y métodos para inhibir la expresión de hmgb1.
MX2020007582A (es) Composiciones y metodos para inhibir la expresion de aldehido deshidrogenasa mitocondrial 2 (aldh2).
WO2018027106A3 (en) RNAi AGENTS FOR HEPATITIS B VIRUS INFECTION
TN2020000038A1 (en) Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
MX2021007855A (es) Composiciones y metodos para inhibir la expresion de hmgb1.
MY202025A (en) Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
WO2008131419A3 (en) Glycoconjugates of rna interference agents
WO2006078414A3 (en) METHODS AND COMPOSITIONS CONCERNING siRNA'S AS MEDIATORS OF RNA INTERFERENCE
ZA201902226B (en) Aav treatment of huntington's disease
FI3604532T3 (fi) Uusia crispr-entsyymejä ja järjestelmiä
WO2017059223A3 (en) Compositions and methods for inhibiting gene expression of lpa
HK1219955A1 (zh) 含4'-硫代修饰的核苷酸的核糖核酸及相关方法
WO2011153323A8 (en) Compositions and methods directed to treating liver fibrosis
WO2007095316A3 (en) Compositions and methods for oligonucleotide formulations
WO2003080807A3 (en) Compositions and methods for suppressing eukaryotic gene expression
MY162210A (en) Pharmaceutical composition
MX2020009072A (es) Composiciones y metodos para inhibir la expresion de gys2.
WO2012177949A3 (en) Compositions and methods for inhibition of expression of protein c (proc) genes
MX2021009754A (es) Metodos y composiciones para inhibir la expresion de cyp27a1.
MX2020009074A (es) Metodos y composiciones para el tratamiento de condiciones asociadas a la deficiencia del conducto biliar.
WO2008098181A8 (en) In vivo genome-wide mutagenesis
WO2013040572A3 (en) Compositions and methods for the production and use of human cholinesterases
TW202615067A (zh) 治療非酒精性脂肪肝疾病的組合物與方法
PH12019500737A1 (en) Improvements in or relating to gene silencing