FR23C1008I2 - TRANSTHYRETIN (TTR) RNAI COMPOSITIONS AND METHODS OF USING THEM FOR THE TREATMENT OR PREVENTION OF TTR-ASSOCIATED DISEASES - Google Patents
TRANSTHYRETIN (TTR) RNAI COMPOSITIONS AND METHODS OF USING THEM FOR THE TREATMENT OR PREVENTION OF TTR-ASSOCIATED DISEASESInfo
- Publication number
- FR23C1008I2 FR23C1008I2 FR23C1008C FR23C1008C FR23C1008I2 FR 23C1008 I2 FR23C1008 I2 FR 23C1008I2 FR 23C1008 C FR23C1008 C FR 23C1008C FR 23C1008 C FR23C1008 C FR 23C1008C FR 23C1008 I2 FR23C1008 I2 FR 23C1008I2
- Authority
- FR
- France
- Prior art keywords
- ttr
- transthyretin
- prevention
- treatment
- methods
- Prior art date
- Legal status (The legal status 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 status listed.)
- Active
Links
- 108010071690 Prealbumin Proteins 0.000 title 2
- 102000009190 Transthyretin Human genes 0.000 title 2
- 108091030071 RNAI Proteins 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 230000002265 prevention Effects 0.000 title 1
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- C—CHEMISTRY; METALLURGY
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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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
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- 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
- A61P25/28—Drugs 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
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- A—HUMAN NECESSITIES
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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- G—PHYSICS
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- G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
- G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
- G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
- G01N33/6893—Chemical 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
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C12N2310/351—Conjugate
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- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- 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
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- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
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- C12Q2600/00—Oligonucleotides characterized by their use
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- G01N2333/435—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans
- G01N2333/46—Assays involving biological materials from specific organisms or of a specific nature from animals; from humans from vertebrates
- G01N2333/47—Assays involving proteins of known structure or function as defined in the subgroups
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- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Epidemiology (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Hospice & Palliative Care (AREA)
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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 (2)
Publication Number | Publication Date |
---|---|
FR23C1008I1 FR23C1008I1 (en) | 2023-03-24 |
FR23C1008I2 true FR23C1008I2 (en) | 2024-04-05 |
Family
ID=56682264
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FR23C1008C Active FR23C1008I2 (en) | 2015-07-31 | 2023-02-08 | TRANSTHYRETIN (TTR) RNAI COMPOSITIONS AND METHODS OF USING THEM FOR THE TREATMENT OR PREVENTION OF TTR-ASSOCIATED DISEASES |
Country Status (33)
Country | Link |
---|---|
US (4) | US10208307B2 (en) |
EP (1) | EP3329002B1 (en) |
JP (3) | JP6896703B2 (en) |
KR (2) | KR20240074895A (en) |
CN (2) | CN108138182B (en) |
BR (1) | BR112018000542B1 (en) |
CA (1) | CA2994285A1 (en) |
CL (1) | CL2018000198A1 (en) |
CY (1) | CY1124545T1 (en) |
DK (1) | DK3329002T3 (en) |
EA (1) | EA201890394A1 (en) |
ES (1) | ES2842300T3 (en) |
FI (1) | FIC20230006I1 (en) |
FR (1) | FR23C1008I2 (en) |
HK (2) | HK1256494A1 (en) |
HR (1) | HRP20202082T1 (en) |
HU (2) | HUE051998T2 (en) |
IL (3) | IL296476B1 (en) |
LT (2) | LT3329002T (en) |
MA (1) | MA43335B1 (en) |
MX (2) | MX2018000981A (en) |
MY (1) | MY195720A (en) |
NL (1) | NL301216I2 (en) |
NO (1) | NO2023007I1 (en) |
PL (1) | PL3329002T3 (en) |
PT (1) | PT3329002T (en) |
RS (1) | RS61297B1 (en) |
SG (1) | SG10201912341SA (en) |
SI (1) | SI3329002T1 (en) |
TW (3) | TWI727963B (en) |
UA (1) | UA126276C2 (en) |
WO (1) | WO2017023660A1 (en) |
ZA (2) | ZA201800571B (en) |
Families Citing this family (24)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2616051T3 (en) | 2008-12-02 | 2017-06-09 | Wave Life Sciences Japan, Inc. | Method for the synthesis of modified nucleic acids in the phosphorus atom |
WO2011005761A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc | Novel nucleic acid prodrugs and methods use thereof |
MX343559B (en) | 2010-04-29 | 2016-11-10 | Ionis Pharmaceuticals Inc | Modulation of transthyretin expression. |
JP5868324B2 (en) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | Asymmetric auxiliary group |
WO2013012758A1 (en) | 2011-07-19 | 2013-01-24 | Ontorii, Inc. | Methods for the synthesis of functionalized nucleic acids |
KR102263352B1 (en) | 2011-11-18 | 2021-06-11 | 알닐람 파마슈티칼스 인코포레이티드 | RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES |
EA201490993A1 (en) | 2011-11-18 | 2014-09-30 | Элнилэм Фармасьютикалз, Инк. | MODIFIED MEANS OF RNA |
WO2014010250A1 (en) | 2012-07-13 | 2014-01-16 | Chiralgen, Ltd. | Asymmetric auxiliary group |
SG11201500232UA (en) | 2012-07-13 | 2015-04-29 | Wave Life Sciences Pte Ltd | Chiral control |
JPWO2015108048A1 (en) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | Chiral nucleic acid adjuvant and antitumor agent having antitumor activity |
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 |
KR20230152178A (en) | 2014-01-16 | 2023-11-02 | 웨이브 라이프 사이언시스 리미티드 | Chiral design |
MX2018000981A (en) | 2015-07-31 | 2018-06-11 | Alnylam Pharmaceuticals Inc | TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES. |
JOP20210043A1 (en) * | 2015-10-01 | 2017-06-16 | Arrowhead Pharmaceuticals Inc | Compositions and Methods for Inhibiting Gene Expression of LPA |
US20190350962A1 (en) * | 2016-12-16 | 2019-11-21 | Alnylam Pharmaceuticals, Inc. | METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS |
KR20200089656A (en) | 2017-09-19 | 2020-07-27 | 알닐람 파마슈티칼스 인코포레이티드 | Compositions and methods for treating transthyretin (TTR) mediated amyloidosis |
AU2018374219C1 (en) * | 2017-12-01 | 2023-05-11 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof |
EP3856907A1 (en) | 2018-09-28 | 2021-08-04 | Alnylam Pharmaceuticals, Inc. | Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases |
WO2021041884A1 (en) | 2019-08-30 | 2021-03-04 | Alnylam Pharmaceuticals, Inc. | Neurofilament light chain (nfl) as a biomarker for transthyretin amyloidosis polyneuropathy |
JP2023516748A (en) | 2020-03-06 | 2023-04-20 | 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 | Compositions and methods for inhibiting expression of transthyretin (TTR) |
WO2022094152A1 (en) * | 2020-10-28 | 2022-05-05 | Neotope Neuroscience Limited | Anti-transthyretin antibodies and methods of use thereof |
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 |
EP4381067A1 (en) | 2021-08-03 | 2024-06-12 | Alnylam Pharmaceuticals, Inc. | Transthyretin (ttr) irna compositions and methods of use thereof |
WO2024130142A2 (en) * | 2022-12-16 | 2024-06-20 | Amgen Inc. | Rnai constructs for inhibiting ttr expression and methods of use thereof |
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Publication number | Priority date | Publication date | Assignee | Title |
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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 |
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