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MA43335A - Compositions d'arni de transthyrétine (ttr) et procédés pour les utiliser pour le traitement ou la prévention de maladies associées à ttr - Google Patents

Compositions d'arni de transthyrétine (ttr) et procédés pour les utiliser pour le traitement ou la prévention de maladies associées à ttr

Info

Publication number
MA43335A
MA43335A MA043335A MA43335A MA43335A MA 43335 A MA43335 A MA 43335A MA 043335 A MA043335 A MA 043335A MA 43335 A MA43335 A MA 43335A MA 43335 A MA43335 A MA 43335A
Authority
MA
Morocco
Prior art keywords
ttr
methods
diseases associated
transthyretine
prevention
Prior art date
Application number
MA043335A
Other languages
English (en)
Other versions
MA43335B1 (fr
Inventor
Amy Chan
Vasant Jadhav
Martin Maier
Kallanthottathil G Rajeev
Tracy Zimmermann
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
Publication of MA43335A publication Critical patent/MA43335A/fr
Publication of MA43335B1 publication Critical patent/MA43335B1/fr

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    • 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
    • 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
    • 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
    • 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • 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 nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12Q2600/00—Oligonucleotides characterized by their use
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA
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    • G01—MEASURING; TESTING
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    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • 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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    • G01N2800/28—Neurological disorders
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    • G01N2800/00—Detection or diagnosis of diseases
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    • G01N2800/2814—Dementia; Cognitive disorders
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/32—Cardiovascular disorders

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  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • Physics & Mathematics (AREA)
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  • Plant Pathology (AREA)
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  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)

Abstract

La présente invention concerne des agents arni, par exemple des agents arni double brin, qui ciblent le gène de la transthyrétine (ttr) et des procédés d'utilisation de tels agents arni pour traiter ou prévenir les maladies associées à la ttr.
MA43335A 2015-07-31 2016-07-28 Compositions d'arni de transthyrétine (ttr) et procédés pour les utiliser pour le traitement ou la prévention de maladies associées à ttr MA43335B1 (fr)

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 (fr) 2015-07-31 2016-07-28 Compositions d'arni de transthyrétine (ttr) et procédés pour les utiliser pour le traitement ou la prévention de maladies associées à ttr

Publications (2)

Publication Number Publication Date
MA43335A true MA43335A (fr) 2018-06-06
MA43335B1 MA43335B1 (fr) 2021-01-29

Family

ID=56682264

Family Applications (1)

Application Number Title Priority Date Filing Date
MA43335A MA43335B1 (fr) 2015-07-31 2016-07-28 Compositions d'arni de transthyrétine (ttr) et procédés pour les utiliser pour le traitement ou la prévention de maladies associées à ttr

Country Status (35)

Country Link
US (5) US10208307B2 (fr)
EP (1) EP3329002B1 (fr)
JP (4) JP6896703B2 (fr)
KR (2) KR20240074895A (fr)
CN (2) CN116064515A (fr)
BR (1) BR112018000542B1 (fr)
CA (1) CA2994285A1 (fr)
CL (1) CL2018000198A1 (fr)
CY (1) CY1124545T1 (fr)
DK (1) DK3329002T3 (fr)
EA (1) EA201890394A1 (fr)
ES (1) ES2842300T3 (fr)
FI (1) FIC20230006I1 (fr)
FR (1) FR23C1008I2 (fr)
HK (1) HK1256683A1 (fr)
HR (1) HRP20202082T1 (fr)
HU (2) HUE051998T2 (fr)
IL (3) IL296476B2 (fr)
LT (2) LT3329002T (fr)
LU (1) LUC00297I2 (fr)
MA (1) MA43335B1 (fr)
MX (2) MX2018000981A (fr)
MY (1) MY195720A (fr)
NL (1) NL301216I2 (fr)
NO (1) NO2023007I1 (fr)
PL (1) PL3329002T3 (fr)
PT (1) PT3329002T (fr)
RS (1) RS61297B1 (fr)
SG (1) SG10201912341SA (fr)
SI (1) SI3329002T1 (fr)
SM (1) SMT202100020T1 (fr)
TW (4) TWI727963B (fr)
UA (1) UA126276C2 (fr)
WO (1) WO2017023660A1 (fr)
ZA (2) ZA201800571B (fr)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE026604T2 (hu) 2008-10-20 2016-06-28 Alnylam Pharmaceuticals Inc Transztiretin-expresszió gátlására szolgáló készítmények és eljárások
CA2744987C (fr) 2008-12-02 2018-01-16 Chiralgen, Ltd. Procede pour la synthese d'acides nucleiques modifies par des atomes de phosphore
CA2767253A1 (fr) 2009-07-06 2011-01-13 Ontorii, Inc. Nouveaux precurseurs d'acide nucleique et leurs methodes d'utilisation
WO2011139917A1 (fr) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Modulation de l'expression de la transthyrétine
WO2012039448A1 (fr) 2010-09-24 2012-03-29 株式会社キラルジェン Groupe auxiliaire asymétrique
WO2013012758A1 (fr) 2011-07-19 2013-01-24 Ontorii, Inc. Procédés pour la synthèse d'acides nucléiques fonctionnalisés
MX391815B (es) * 2011-11-18 2025-03-21 Alnylam Pharmaceuticals Inc AGENTES DE iARN, COMPOSICIONES Y METODOS DE LOS MISMOS PARA USARSE EN EL TRATAMIENTO DE ENFERMEDADES ASOCIADAS CON TRANSTITERINA (TTR).
KR102853694B1 (ko) 2011-11-18 2025-09-03 알닐람 파마슈티칼스 인코포레이티드 변형된 ＲＮＡｉ 제제
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
KR102712879B1 (ko) 2012-07-13 2024-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
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
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP4137572A1 (fr) 2014-01-16 2023-02-22 Wave Life Sciences Ltd. Conception chirale
KR20240074895A (ko) 2015-07-31 2024-05-28 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물 및 TTR-관련 질병을 치료하거나, 예방하기 위한 그의 사용 방법
JOP20210043A1 (ar) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
TWI865997B (zh) * 2016-12-16 2024-12-11 美商阿尼拉製藥公司 使用甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物於治療或預防ＴＴＲ相關疾病之方法
AU2018336806B2 (en) 2017-09-19 2025-04-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
HRP20250277T1 (hr) * 2017-12-01 2025-05-09 Suzhou Ribo Life Science Co., Ltd. Dvolančani oligonukleotid, sastav i konjugat koji sadrži dvolančani oligonukleotid, postupak njihove pripreme i upotreba
CA3114396A1 (fr) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Compositions d'arni de la transthyretine (ttr) et leurs procedes d'utilisation pour traiter ou prevenir des maladies oculaires associees a la ttr
EP4022062A1 (fr) 2019-08-30 2022-07-06 Alnylam Pharmaceuticals, Inc. Chaîne légère neurofilamentaire (nfl) en tant que biomarqueur pour la polyneuropathie liée une amylose à transthyrétine
CN115461460A (zh) 2020-03-06 2022-12-09 阿尔尼拉姆医药品有限公司 用于抑制转甲状腺素蛋白(ttr)的表达的组合物和方法
WO2022094152A1 (fr) * 2020-10-28 2022-05-05 Neotope Neuroscience Limited Anticorps anti-transthyrétine et méthodes d'utilisation associées
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