CL2021001605A1 - Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso. - Google Patents

Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso.

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Publication number
CL2021001605A1
CL2021001605A1 CL2021001605A CL2021001605A CL2021001605A1 CL 2021001605 A1 CL2021001605 A1 CL 2021001605A1 CL 2021001605 A CL2021001605 A CL 2021001605A CL 2021001605 A CL2021001605 A CL 2021001605A CL 2021001605 A1 CL2021001605 A1 CL 2021001605A1
Authority
CL
Chile
Prior art keywords
methods
app
rnai agent
precursor protein
amyloid precursor
Prior art date
Application number
CL2021001605A
Other languages
English (en)
Inventor
Vasant Jadhav
Martin Maier
Kevin Fitzgerald
Stuart Milstein
Kirk Brown
Jayaprakash Nair
Mark Keating
Adam Castoreno
Patrick Haslett
Mangala Meenakshi Soundarapandian
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.)
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Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of CL2021001605A1 publication Critical patent/CL2021001605A1/es

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    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • 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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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711—Alzheimer's disease; Amyloid plaque core protein
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
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  • Neurology (AREA)
  • Biotechnology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
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  • Neurosurgery (AREA)
  • Biophysics (AREA)
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  • Physics & Mathematics (AREA)
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  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Psychology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La divulgación se refiere a agentes de ácido ribonucleico bicatenario (iARNbc) y composiciones dirigidas al gen de PPA, así como también métodos para inhibir la expresión de un gen de PPA y métodos para tratar sujetos con una enfermedad o trastorno asociados a PPA, tales como angiopatía amiloide cerebral (AAC) y enfermedad de Alzheimer familiar de inicio precoz (EAFIP o EAFp), usando dichos agentes y composiciones de iARNdc.
CL2021001605A 2018-12-19 2021-06-17 Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso. CL2021001605A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862781774P 2018-12-19 2018-12-19
US201962862472P 2019-06-17 2019-06-17
US201962928795P 2019-10-31 2019-10-31

Publications (1)

Publication Number Publication Date
CL2021001605A1 true CL2021001605A1 (es) 2021-12-24

Family

ID=71101909

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021001605A CL2021001605A1 (es) 2018-12-19 2021-06-17 Composiciones de agentes de iarn de proteínas precursoras amiloides (ppa) y métodos para su uso.

Country Status (18)

Country Link
US (2) US11034957B2 (es)
EP (1) EP3898979A4 (es)
JP (1) JP2022515193A (es)
KR (1) KR20210111780A (es)
CN (1) CN113454222A (es)
AU (1) AU2019405783A1 (es)
BR (1) BR112021011895A2 (es)
CA (1) CA3124090A1 (es)
CL (1) CL2021001605A1 (es)
CO (1) CO2021009163A2 (es)
CR (1) CR20210393A (es)
DO (1) DOP2021000126A (es)
IL (1) IL283852A (es)
MX (1) MX2021007570A (es)
PE (1) PE20211420A1 (es)
SG (1) SG11202105886QA (es)
TW (1) TW202039844A (es)
WO (1) WO2020132227A2 (es)

Families Citing this family (10)

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WO2022165172A1 (en) * 2021-01-29 2022-08-04 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing amyloid precursor protein (app)
WO2023039503A2 (en) * 2021-09-10 2023-03-16 Alnylam Pharmaceuticals, Inc. App irna compositions and methods of use thereof for treating or preventing diseases characterized by enlarged endosomes
TW202333750A (zh) * 2021-11-08 2023-09-01 美商黛瑟納製藥公司 ＲＮＡｉ寡核苷酸共軛體
WO2023173061A2 (en) * 2022-03-11 2023-09-14 University Of Massachusetts Oligonucleotides for app modulation
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物
WO2023224979A1 (en) * 2022-05-16 2023-11-23 University Of Massachusetts Optimized sirna scaffolds
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Also Published As

Publication number Publication date
KR20210111780A (ko) 2021-09-13
JP2022515193A (ja) 2022-02-17
CR20210393A (es) 2021-10-27
EP3898979A2 (en) 2021-10-27
US20200339991A1 (en) 2020-10-29
DOP2021000126A (es) 2021-09-30
IL283852A (en) 2021-07-29
US11034957B2 (en) 2021-06-15
BR112021011895A2 (pt) 2021-09-08
EP3898979A4 (en) 2022-10-12
MX2021007570A (es) 2021-08-24
US20220002724A1 (en) 2022-01-06
WO2020132227A3 (en) 2020-07-30
PE20211420A1 (es) 2021-08-03
SG11202105886QA (en) 2021-07-29
CA3124090A1 (en) 2020-06-25
TW202039844A (zh) 2020-11-01
CN113454222A (zh) 2021-09-28
AU2019405783A1 (en) 2021-07-01
WO2020132227A2 (en) 2020-06-25
CO2021009163A2 (es) 2021-10-29

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