PL3444350T3 - Sposoby i kompozycje do specyficznego hamowania alfa-1 antytrypsyny przez dwuniciowy rna - Google Patents

Sposoby i kompozycje do specyficznego hamowania alfa-1 antytrypsyny przez dwuniciowy rna

Info

Publication number
PL3444350T3
PL3444350T3 PL18196106T PL18196106T PL3444350T3 PL 3444350 T3 PL3444350 T3 PL 3444350T3 PL 18196106 T PL18196106 T PL 18196106T PL 18196106 T PL18196106 T PL 18196106T PL 3444350 T3 PL3444350 T3 PL 3444350T3
Authority
PL
Poland
Prior art keywords
antitrypsin
alpha
compositions
double
methods
Prior art date
Application number
PL18196106T
Other languages
English (en)
Polish (pl)
Inventor
Bob D. Brown
Henryk T. Dudek
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 PL3444350T3 publication Critical patent/PL3444350T3/pl

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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/32—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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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. ==--==--==--

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Electric Double-Layer Capacitors Or The Like (AREA)
PL18196106T 2013-07-03 2014-07-03 Sposoby i kompozycje do specyficznego hamowania alfa-1 antytrypsyny przez dwuniciowy rna PL3444350T3 (pl)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361842551P 2013-07-03 2013-07-03
US201361891548P 2013-10-16 2013-10-16
EP18196106.1A EP3444350B1 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
EP14820312.8A EP3017047A4 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna
PCT/US2014/045365 WO2015003113A2 (en) 2013-07-03 2014-07-03 Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded rna

Publications (1)

Publication Number Publication Date
PL3444350T3 true PL3444350T3 (pl) 2022-03-21

Family

ID=52133228

Family Applications (1)

Application Number Title Priority Date Filing Date
PL18196106T PL3444350T3 (pl) 2013-07-03 2014-07-03 Sposoby i kompozycje do specyficznego hamowania alfa-1 antytrypsyny przez dwuniciowy rna

Country Status (11)

Country Link
US (11) US9458457B2 (https=)
EP (3) EP3017047A4 (https=)
JP (4) JP2016528887A (https=)
AU (2) AU2014284298B2 (https=)
CA (1) CA2916499A1 (https=)
DK (1) DK3444350T3 (https=)
ES (1) ES2905257T3 (https=)
HU (1) HUE058794T2 (https=)
PL (1) PL3444350T3 (https=)
PT (1) PT3444350T (https=)
WO (1) WO2015003113A2 (https=)

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TW201620526A (zh) * 2014-06-17 2016-06-16 愛羅海德研究公司 用於抑制α－１抗胰蛋白酶基因表現之組合物及方法
EA201891338A1 (ru) * 2015-12-04 2018-12-28 Новартис Аг Композиции и способы для иммуноонкологии
WO2017139616A1 (en) * 2016-02-10 2017-08-17 Arrowhead Pharmaceuticals, Inc. Methods of treatment for alpha-1 antitrypsin deficiency
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AU2017379073B2 (en) * 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
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MX2021015003A (es) 2019-06-06 2022-01-24 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de la deficiencia de alfa-1 antitripsina (aatd).
AU2021345026A1 (en) * 2020-09-15 2023-05-11 Arrowhead Pharmaceuticals, Inc. Methods for the reduction of z-aat protein levels
JP2024532019A (ja) 2021-08-25 2024-09-05 ノボ ノルディスク エー／エス アルファ－１アンチトリプシン発現を阻害するための組成物及び方法
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WO2024229229A2 (en) * 2023-05-02 2024-11-07 Sirnaomics, Inc. Products and compositions

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Also Published As

Publication number Publication date
PT3444350T (pt) 2022-02-04
DK3444350T3 (da) 2022-02-07
EP3444350A1 (en) 2019-02-20
EP3017047A2 (en) 2016-05-11
CA2916499A1 (en) 2015-01-08
US11408002B1 (en) 2022-08-09
US11851658B2 (en) 2023-12-26
US11208658B2 (en) 2021-12-28
JP2022078069A (ja) 2022-05-24
ES2905257T3 (es) 2022-04-07
US10370655B2 (en) 2019-08-06
US10844381B2 (en) 2020-11-24
US11312961B1 (en) 2022-04-26
AU2014284298A1 (en) 2016-01-21
US11312960B1 (en) 2022-04-26
JP2016528887A (ja) 2016-09-23
AU2020260479B2 (en) 2023-06-15
EP4012031A1 (en) 2022-06-15
US20210180061A1 (en) 2021-06-17
EP3444350B1 (en) 2021-12-01
US9458457B2 (en) 2016-10-04
US20220228149A1 (en) 2022-07-21
US20220396793A1 (en) 2022-12-15
AU2020260479A1 (en) 2020-11-26
AU2014284298B2 (en) 2020-12-10
WO2015003113A3 (en) 2015-02-26
US11912996B2 (en) 2024-02-27
JP2024075702A (ja) 2024-06-04
US20170130221A1 (en) 2017-05-11
EP3017047A4 (en) 2017-06-14
US11459566B1 (en) 2022-10-04
US20150011607A1 (en) 2015-01-08
US20190390198A1 (en) 2019-12-26
HUE058794T2 (hu) 2022-09-28
WO2015003113A2 (en) 2015-01-08
JP2020188771A (ja) 2020-11-26
US20230069235A1 (en) 2023-03-02

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