EA201890394A1 - КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ИЛИ ПРЕДУПРЕЖДЕНИЯ АССОЦИИРОВАННЫХ С TTR ЗАБОЛЕВАНИЙ - Google Patents

КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ИЛИ ПРЕДУПРЕЖДЕНИЯ АССОЦИИРОВАННЫХ С TTR ЗАБОЛЕВАНИЙ

Info

Publication number
EA201890394A1
EA201890394A1 EA201890394A EA201890394A EA201890394A1 EA 201890394 A1 EA201890394 A1 EA 201890394A1 EA 201890394 A EA201890394 A EA 201890394A EA 201890394 A EA201890394 A EA 201890394A EA 201890394 A1 EA201890394 A1 EA 201890394A1
Authority
EA
Eurasian Patent Office
Prior art keywords
ttr
irna
methods
transtyretine
prevention
Prior art date
Application number
EA201890394A
Other languages
English (en)
Russian (ru)
Inventor
Трэйси Циммерманн
Эми Чань
Васант Джадхав
Мартин Майер
Каллантхоттатхил Г. Раджив
Original Assignee
Элнилэм Фармасьютикалз, Инк.
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 Элнилэм Фармасьютикалз, Инк. filed Critical Элнилэм Фармасьютикалз, Инк.
Publication of EA201890394A1 publication Critical patent/EA201890394A1/ru

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
    • 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
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/14Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • 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
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-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
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • 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
    • 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
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen
    • 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
    • C12N2320/30Special therapeutic applications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/158Expression markers
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/178Oligonucleotides characterized by their use miRNA, siRNA or ncRNA
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/435Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
    • G01N2333/46Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
    • G01N2333/47Assays involving proteins of known structure or function as defined in the subgroups
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/22Haematology
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/28Neurological disorders
    • G01N2800/2814Dementia; Cognitive disorders
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00Detection or diagnosis of diseases
    • G01N2800/32Cardiovascular disorders

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Cardiology (AREA)
EA201890394A 2015-07-31 2016-07-28 КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ИЛИ ПРЕДУПРЕЖДЕНИЯ АССОЦИИРОВАННЫХ С TTR ЗАБОЛЕВАНИЙ EA201890394A1 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562199563P 2015-07-31 2015-07-31
US201662287518P 2016-01-27 2016-01-27
PCT/US2016/044359 WO2017023660A1 (en) 2015-07-31 2016-07-28 TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES

Publications (1)

Publication Number Publication Date
EA201890394A1 true EA201890394A1 (ru) 2018-08-31

Family

ID=56682264

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201890394A EA201890394A1 (ru) 2015-07-31 2016-07-28 КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ИЛИ ПРЕДУПРЕЖДЕНИЯ АССОЦИИРОВАННЫХ С TTR ЗАБОЛЕВАНИЙ

Country Status (35)

Country Link
US (5) US10208307B2 (enExample)
EP (1) EP3329002B1 (enExample)
JP (4) JP6896703B2 (enExample)
KR (2) KR20240074895A (enExample)
CN (2) CN108138182B (enExample)
BR (1) BR112018000542B1 (enExample)
CA (1) CA2994285A1 (enExample)
CL (1) CL2018000198A1 (enExample)
CY (1) CY1124545T1 (enExample)
DK (1) DK3329002T3 (enExample)
EA (1) EA201890394A1 (enExample)
ES (1) ES2842300T3 (enExample)
FI (1) FIC20230006I1 (enExample)
FR (1) FR23C1008I2 (enExample)
HK (1) HK1256683A1 (enExample)
HR (1) HRP20202082T1 (enExample)
HU (2) HUE051998T2 (enExample)
IL (3) IL313082A (enExample)
LT (2) LT3329002T (enExample)
LU (1) LUC00297I2 (enExample)
MA (1) MA43335B1 (enExample)
MX (2) MX2018000981A (enExample)
MY (1) MY195720A (enExample)
NL (1) NL301216I2 (enExample)
NO (1) NO2023007I1 (enExample)
PL (1) PL3329002T3 (enExample)
PT (1) PT3329002T (enExample)
RS (1) RS61297B1 (enExample)
SG (1) SG10201912341SA (enExample)
SI (1) SI3329002T1 (enExample)
SM (1) SMT202100020T1 (enExample)
TW (4) TWI883468B (enExample)
UA (1) UA126276C2 (enExample)
WO (1) WO2017023660A1 (enExample)
ZA (2) ZA201800571B (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2739895C (en) 2008-10-20 2018-09-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
WO2011005761A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc Novel nucleic acid prodrugs and methods use thereof
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
SI3366775T2 (sl) 2011-11-18 2025-12-31 Alnylam Pharmaceuticals, Inc. Modificirana sredstva RNAI
US9399775B2 (en) 2011-11-18 2016-07-26 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
HUE051998T2 (hu) 2015-07-31 2021-04-28 Alnylam Pharmaceuticals Inc Transztiretin (TTR) iRNS készítmények és eljárások azok alkalmazása TTR-asszociált betegségek kezelésére vagy megelõzésére
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
EP3555292A1 (en) * 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
WO2019060442A1 (en) 2017-09-19 2019-03-28 Alnylam Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN MEDIATED AMYLOSIS (TTR)
CN110997919B (zh) * 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
JP7470107B2 (ja) * 2018-09-28 2024-04-17 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 トランスサイレチン(TTR)iRNA組成物及びTTR関連眼疾患を治療又は予防するためのその使用方法
EP4022062A1 (en) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy
CA3174473A1 (en) 2020-03-06 2021-09-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin (ttr)
US20230391859A1 (en) * 2020-10-28 2023-12-07 Novo Nordisk A/S Anti-transthyretin antibodies and methods of use thereof
JP2024511473A (ja) * 2021-03-24 2024-03-13 アタランタ セラピューティクス,インコーポレーテッド パターン化化学修飾を有する二本鎖sirna
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
JP2024530018A (ja) 2021-08-03 2024-08-14 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 トランスサイレチン(TTR)iRNA組成物およびその使用方法
EP4634385A2 (en) * 2022-12-16 2025-10-22 Amgen Inc. Rnai constructs for inhibiting ttr expression and methods of use thereof
WO2025072748A2 (en) 2023-09-29 2025-04-03 Insitro, Inc. Compositions and methods for treating nonalcoholic fatty liver disease
WO2025077916A1 (zh) * 2023-10-13 2025-04-17 北京安龙生物医药有限公司 靶向转甲状腺素蛋白的寡核苷酸及其用途
WO2025098117A1 (zh) * 2023-11-06 2025-05-15 成都倍特药业股份有限公司 一种抑制甲状腺素转运蛋白(TTR)表达的RNAi试剂、组合物及应用
TW202521133A (zh) * 2023-11-27 2025-06-01 大陸商上海舶望製藥有限公司 抑制甲狀腺素運載蛋白(ttr)表達的組合物和方法
US20250333742A1 (en) 2024-04-25 2025-10-30 Insitro, Inc. Compositions and methods for treating metabolic dysfunction-associated steatotic liver disease

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030027216A1 (en) 2001-07-02 2003-02-06 Kiernan Urban A. Analysis of proteins from biological fluids using mass spectrometric immunoassay
AU2001247951A1 (en) 2000-02-07 2001-08-14 Roche Diagnostics Corporation Novel cationic amphiphiles
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US20030072794A1 (en) 2000-06-09 2003-04-17 Teni Boulikas Encapsulation of plasmid DNA (lipogenes™) and therapeutic agents with nuclear localization signal/fusogenic peptide conjugates into targeted liposome complexes
SI1407044T2 (en) 2000-12-01 2018-03-30 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Rna interference mediating small rna molecules
US20020160394A1 (en) 2001-01-24 2002-10-31 Bayer Corporation Regulation of transthyretin to treat obesity
US20030170891A1 (en) 2001-06-06 2003-09-11 Mcswiggen James A. RNA interference mediated inhibition of epidermal growth factor receptor gene expression using short interfering nucleic acid (siNA)
WO2003001182A2 (en) 2001-06-21 2003-01-03 The Cleveland Clinic Foundation Homocysteinylated transthyretin
US20030191056A1 (en) 2002-04-04 2003-10-09 Kenneth Walker Use of transthyretin peptide/protein fusions to increase the serum half-life of pharmacologically active peptides/proteins
KR101201664B1 (ko) 2002-08-05 2012-11-15 사일런스 테라퓨틱스 아게 신규한 형태의 간섭 rna 분자
US7956176B2 (en) 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
CA2502413A1 (en) 2002-11-01 2004-05-21 The Regents Of The University Of Colorado, A Body Corporate Quantitative analysis of protein isoforms using matrix-assisted laser desorption/ionization time of flight mass spectrometry
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン, インコーポレイテッド 機能的siRNAおよび超機能的siRNA
ATE479752T1 (de) 2003-03-07 2010-09-15 Alnylam Pharmaceuticals Inc Therapeutische zusammensetzungen
AU2004227414A1 (en) 2003-04-03 2004-10-21 Alnylam Pharmaceuticals iRNA conjugates
KR101168440B1 (ko) 2003-07-16 2012-07-27 프로티바 바이오쎄라퓨틱스, 인코포레이티드 지질 캡슐화된 간섭 rna
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP1765416A4 (en) 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc DOUBLE-STRONG COMPOSITIONS WITH DIFFERENTLY MODIFIED STRANDS FOR USE IN GENE MODULATION
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
EP1781593B1 (en) 2004-06-07 2011-12-14 Protiva Biotherapeutics Inc. Cationic lipids and methods of use
US7838561B2 (en) 2004-06-14 2010-11-23 Wisconsin Alumni Research Foundation Method for preventing or treating cardiac hypertrophy
BRPI0520207A2 (pt) 2004-12-08 2012-09-25 Revision Therapeutics Inc uso de uma quantidade eficaz de um primeiro composto, e, medicamento sistemicamente formulado
CA2597724A1 (en) 2005-02-14 2007-08-02 Sirna Therapeutics, Inc. Cationic lipids and formulated molecular compositions containing them
EP1866414B9 (en) 2005-03-31 2012-10-03 Calando Pharmaceuticals, Inc. Inhibitors of ribonucleotide reductase subunit 2 and uses thereof
US7915230B2 (en) 2005-05-17 2011-03-29 Molecular Transfer, Inc. Reagents for transfection of eukaryotic cells
CA2616877C (en) 2005-07-27 2014-01-28 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
CN101448849B (zh) 2006-03-31 2013-08-21 阿尔尼拉姆医药品有限公司 抑制Eg5基因表达的组合物和方法
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
EP2068886B1 (en) 2006-10-03 2013-09-18 Tekmira Pharmaceuticals Corporation Lipid containing formulations
GB0704764D0 (en) 2007-03-12 2007-04-18 Electrophoretics Ltd Isobarically labelled reagents and methods of their use
WO2009002944A1 (en) 2007-06-22 2008-12-31 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US20090247608A1 (en) 2007-12-04 2009-10-01 Alnylam Pharmaceuticals, Inc. Targeting Lipids
CA2710713C (en) 2007-12-27 2017-09-19 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
EP2224912B1 (en) 2008-01-02 2016-05-11 TEKMIRA Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
EP2245039A4 (en) 2008-01-31 2012-06-06 Alnylam Pharmaceuticals Inc OPTIMIZED METHODS FOR EXPORING DSRNA TO TARGET THE PCSK9 GEN
WO2009100390A2 (en) 2008-02-08 2009-08-13 Mayo Foundation For Medical Education And Research Classifying amyloidosis
JP5475753B2 (ja) 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
TWI455944B (zh) 2008-07-01 2014-10-11 Daiichi Sankyo Co Ltd 雙股多核苷酸
US20110237646A1 (en) 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
CA2740000C (en) 2008-10-09 2017-12-12 Tekmira Pharmaceuticals Corporation Improved amino lipids and methods for the delivery of nucleic acids
CA2739895C (en) 2008-10-20 2018-09-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
MX353900B (es) 2008-11-07 2018-02-01 Massachusetts Inst Technology Lipidoides de aminoalcohol y usos de los mismos.
CA2743139C (en) 2008-11-10 2019-04-02 Alnylam Pharmaceuticals, Inc. Novel lipids and compositions for the delivery of therapeutics
US9493774B2 (en) 2009-01-05 2016-11-15 Rxi Pharmaceuticals Corporation Inhibition of PCSK9 through RNAi
EP3243504A1 (en) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Improved lipid formulation
KR102229618B1 (ko) 2009-05-05 2021-03-18 알닐람 파마슈티칼스 인코포레이티드 지질 조성물
NZ622843A (en) 2009-06-10 2015-10-30 Tekmira Pharmaceuticals Corp Improved lipid formulation
WO2010147992A1 (en) 2009-06-15 2010-12-23 Alnylam Pharmaceuticals, Inc. Methods for increasing efficacy of lipid formulated sirna
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
JP5723378B2 (ja) 2009-11-03 2015-05-27 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Alnylam Pharmaceuticals, Inc. トランスサイレチン(ttr)を阻害する脂質製剤化組成物及び方法
US20230340473A1 (en) 2010-03-29 2023-10-26 Alnylam Pharmaceuticals, Inc. Sirna therapy for transthyretin (ttr) related ocular amyloidosis
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
RS56011B1 (sr) 2010-04-29 2017-09-29 Ionis Pharmaceuticals Inc Modulacija ekspresije transtiretina
CA2812046A1 (en) 2010-09-15 2012-03-22 Alnylam Pharmaceuticals, Inc. Modified irna agents
DK2632472T3 (en) 2010-10-29 2018-03-19 Sirna Therapeutics Inc RNA INTERFERENCE-MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERRING NUCLEIC ACIDS (SINA)
US9339513B2 (en) 2010-11-09 2016-05-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
WO2012177906A1 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
US9399775B2 (en) 2011-11-18 2016-07-26 Alnylam Pharmaceuticals, Inc. RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
SI3366775T2 (sl) * 2011-11-18 2025-12-31 Alnylam Pharmaceuticals, Inc. Modificirana sredstva RNAI
WO2013166041A1 (en) 2012-05-01 2013-11-07 The Trustees Of Columbia University In The City Of New York Transthyretin ligands capable of inhibiting retinol-dependent rbp4-ttr interaction for treatment of age-related macular
US9708607B2 (en) 2012-08-03 2017-07-18 Alnylam Pharmaceuticals, Inc. Modified RNAi agents
WO2015042564A1 (en) * 2013-09-23 2015-03-26 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing transthyretin (ttr) associated diseases
AU2014360314B2 (en) 2013-12-06 2018-04-26 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA
IL314045A (en) * 2013-12-12 2024-09-01 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
IL316808A (en) 2014-08-20 2025-01-01 Alnylam Pharmaceuticals Inc Modified double-stranded RNA materials and their uses
PL3185957T3 (pl) 2014-08-29 2022-11-14 Alnylam Pharmaceuticals, Inc. Patisiran do zastosowania w leczeniu amyloidozy związanej z transtyretyną
US11117877B2 (en) 2014-11-21 2021-09-14 Bsim Therapeutics, S.A. 2-thioxothiazolidin-4-one derivatives active as transthyretin ligands and uses thereof
HUE051998T2 (hu) 2015-07-31 2021-04-28 Alnylam Pharmaceuticals Inc Transztiretin (TTR) iRNS készítmények és eljárások azok alkalmazása TTR-asszociált betegségek kezelésére vagy megelõzésére
EP3555292A1 (en) 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
WO2019060442A1 (en) 2017-09-19 2019-03-28 Alnylam Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN MEDIATED AMYLOSIS (TTR)
JP7470107B2 (ja) 2018-09-28 2024-04-17 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 トランスサイレチン(TTR)iRNA組成物及びTTR関連眼疾患を治療又は予防するためのその使用方法
EP4022062A1 (en) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
CA3174473A1 (en) 2020-03-06 2021-09-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin (ttr)
EP4334448A1 (en) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
JP2024530018A (ja) 2021-08-03 2024-08-14 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 トランスサイレチン(TTR)iRNA組成物およびその使用方法

Also Published As

Publication number Publication date
CN116064515A (zh) 2023-05-05
HRP20202082T1 (hr) 2021-02-19
JP2025165939A (ja) 2025-11-05
TWI883468B (zh) 2025-05-11
MA43335B1 (fr) 2021-01-29
US20200377889A1 (en) 2020-12-03
LUC00297I2 (enExample) 2025-09-22
IL296476B1 (en) 2024-06-01
HUE051998T2 (hu) 2021-04-28
IL296476B2 (en) 2024-10-01
MA43335A (fr) 2018-06-06
TW201718855A (zh) 2017-06-01
NZ739169A (en) 2025-05-30
US10683501B2 (en) 2020-06-16
PT3329002T (pt) 2021-01-12
TW202344688A (zh) 2023-11-16
US20170029817A1 (en) 2017-02-02
ZA201902447B (en) 2021-10-27
US12049628B2 (en) 2024-07-30
CY1124545T1 (el) 2022-07-22
KR20180028537A (ko) 2018-03-16
SG10201912341SA (en) 2020-02-27
HK1256683A1 (zh) 2019-10-04
SI3329002T1 (sl) 2021-02-26
JP7711150B2 (ja) 2025-07-22
LTPA2023505I1 (enExample) 2023-02-27
HK1256494A1 (en) 2019-09-27
CN108138182B (zh) 2022-08-19
FR23C1008I1 (fr) 2023-03-24
MX2023012080A (es) 2023-10-25
LT3329002T (lt) 2021-01-11
KR102667020B1 (ko) 2024-05-21
WO2017023660A1 (en) 2017-02-09
EP3329002B1 (en) 2020-10-07
FIC20230006I1 (fi) 2023-02-06
NZ778442A (en) 2025-05-30
HUS2300007I1 (hu) 2023-02-28
IL313082A (en) 2024-07-01
JP2024028749A (ja) 2024-03-05
BR112018000542A2 (pt) 2018-09-18
US10208307B2 (en) 2019-02-19
TWI807302B (zh) 2023-07-01
JP2021167313A (ja) 2021-10-21
DK3329002T3 (da) 2021-01-11
ZA201800571B (en) 2019-07-31
IL256614B (en) 2022-10-01
US11286486B2 (en) 2022-03-29
TWI727963B (zh) 2021-05-21
CN108138182A (zh) 2018-06-08
JP2018523655A (ja) 2018-08-23
LTC3329002I2 (enExample) 2024-11-11
ES2842300T3 (es) 2021-07-13
US20230022185A1 (en) 2023-01-26
JP7417559B2 (ja) 2024-01-18
NL301216I2 (nl) 2023-05-24
EP3329002A1 (en) 2018-06-06
NO2023007I1 (no) 2023-02-07
CA2994285A1 (en) 2017-02-09
IL256614A (en) 2018-02-28
PL3329002T3 (pl) 2021-04-19
JP6896703B2 (ja) 2021-06-30
US20190345492A1 (en) 2019-11-14
IL256614B2 (en) 2023-02-01
BR112018000542B1 (pt) 2023-01-24
KR20240074895A (ko) 2024-05-28
MY195720A (en) 2023-02-07
NL301216I1 (enExample) 2023-02-08
SMT202100020T1 (it) 2021-03-15
UA126276C2 (uk) 2022-09-14
TW202130810A (zh) 2021-08-16
RS61297B1 (sr) 2021-02-26
MX2018000981A (es) 2018-06-11
IL296476A (en) 2022-11-01
TW202532647A (zh) 2025-08-16
CL2018000198A1 (es) 2018-07-20
FR23C1008I2 (fr) 2024-04-05
US20250075211A1 (en) 2025-03-06

Similar Documents

Publication Publication Date Title
EA201890394A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ТРАНСТИРЕТИНА (TTR) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ ДЛЯ ЛЕЧЕНИЯ ИЛИ ПРЕДУПРЕЖДЕНИЯ АССОЦИИРОВАННЫХ С TTR ЗАБОЛЕВАНИЙ
ZA202306507B (en) Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
MX2023015185A (es) Degradadores de proteinas y usos de los mismos.
ZA201901924B (en) Anti¿lag¿3 antibodies and compositions
GEP20247593B (en) Compositions and methods of inhibiting masp-3 for the treatment of various diseases and disorders
EA201791874A1 (ru) Фторированные ингибиторы лизилоксидазоподобного фермента-2 и их применение
EA201891199A1 (ru) Композиции, содержащие бактериальные штаммы
EA201890047A1 (ru) Композиции, содержащие бактериальные штаммы
EA201890048A1 (ru) Композиции, содержащие бактериальные штаммы
MX376201B (es) Compuestos inhibidores de metaloenzima.
EA201691134A1 (ru) Новые ингибиторы глутаминазы
EA201891200A1 (ru) Композиции, содержащие бактериальные штаммы
PH12014501106A1 (en) Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
EA201692201A1 (ru) Соединения и композиции в качестве агонистов toll-подобного рецептора 7
EA201890325A1 (ru) Пептидные ингибиторы рецептора интерлейкина-23 и их применение для лечения воспалительных заболеваний
BR112019002331A2 (pt) anticorpos anti-siglec-7 para o tratamento do câncer e métodos para obtenção dos mesmos
MX392098B (es) Anticuerpos neutralizantes de poliomavirus.
EA201890051A1 (ru) Композиции, содержащие бактериальные штаммы
MX2017012738A (es) Inhibidores de indolamina 2.3-dioxigenasa (ido) para el tratamiento de cancer.
MX2018009325A (es) Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
EA201890185A1 (ru) Способы лечения опосредованных гепсидином нарушений
JOP20210158A1 (ar) مُعَدِّلات التعبير الجيني عن hsd17b13
EA201792604A1 (ru) Ингибиторы аутотаксина и их применения
EA201990400A1 (ru) Соединения и композиции и их применение
MX389724B (es) Compuestos de fenil-2-hidroxi-acetilamino-2-metil-fenilo