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MX2022011536A - Oligonucleotidos para la modulacion de snca. - Google Patents

Oligonucleotidos para la modulacion de snca.

Info

Publication number
MX2022011536A
MX2022011536A MX2022011536A MX2022011536A MX2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A
Authority
MX
Mexico
Prior art keywords
snca
oligonucleotides
modulation
novel
targeting
Prior art date
Application number
MX2022011536A
Other languages
English (en)
Inventor
Anastasia Khvorova
Chantal Ferguson
Sarah Davis
Kathryn Monopoli
Original Assignee
Univ Massachusetts
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 Univ Massachusetts filed Critical Univ Massachusetts
Publication of MX2022011536A publication Critical patent/MX2022011536A/es

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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
    • 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
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
    • A61K47/551—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • 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
    • 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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    • C12N2310/11—Antisense
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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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/30—Chemical structure
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  • Physics & Mathematics (AREA)
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  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)

Abstract

Esta descripción se refiere a secuencias dirigidas a SNCA nuevas. También se proporcionan oligonucleótidos dirigidos a SNCA nuevos para el tratamiento de enfermedades neurodegenerativas.
MX2022011536A 2020-03-18 2021-03-17 Oligonucleotidos para la modulacion de snca. MX2022011536A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062991406P 2020-03-18 2020-03-18
US202063071115P 2020-08-27 2020-08-27
PCT/US2021/022748 WO2021188661A1 (en) 2020-03-18 2021-03-17 Oligonucleotides for snca modulation

Publications (1)

Publication Number Publication Date
MX2022011536A true MX2022011536A (es) 2023-01-04

Family

ID=77772170

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022011536A MX2022011536A (es) 2020-03-18 2021-03-17 Oligonucleotidos para la modulacion de snca.

Country Status (10)

Country Link
US (2) US20210363524A1 (es)
EP (1) EP4121538A4 (es)
JP (1) JP2023518268A (es)
KR (1) KR20230002327A (es)
CN (1) CN115777021A (es)
AU (1) AU2021238326A1 (es)
BR (1) BR112022018475A2 (es)
CA (1) CA3171757A1 (es)
MX (1) MX2022011536A (es)
WO (1) WO2021188661A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230416735A1 (en) * 2022-04-14 2023-12-28 University Of Massachusetts Oligonucleotides for atn1 modulation
WO2023215390A2 (en) * 2022-05-04 2023-11-09 University Of Massachusetts Oligonucleotides for pms1 modulation
AU2023354015A1 (en) * 2022-09-29 2025-04-17 Atalanta Therapeutics, Inc. Sirna compositions and methods targeting alpha-synuclein nucleic acids
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof
WO2025085713A1 (en) * 2023-10-20 2025-04-24 Spark Therapeutics, Inc. Inhibitory nucleic acid targeting alpha synuclein expression
WO2025128134A1 (en) * 2023-12-14 2025-06-19 National Taiwan University Hospital Crispri targeting alpha-synuclein
WO2025162214A1 (en) * 2024-01-30 2025-08-07 Shanghai Argo Biopharmaceutical Co., Ltd. Compositions and methods for inhibiting expression of synuclein alpha (snca) gene

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005045034A2 (en) * 2003-10-23 2005-05-19 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA)
WO2009079399A2 (en) * 2007-12-14 2009-06-25 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
JP5875976B2 (ja) * 2009-06-01 2016-03-02 ヘイロー−バイオ アールエヌエーアイ セラピューティクス， インコーポレイテッド 多価ｒｎａ干渉のためのポリヌクレオチド、組成物およびそれらの使用方法
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
AU2011329777B2 (en) * 2010-11-17 2016-06-09 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
WO2016161388A1 (en) * 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
WO2016161374A1 (en) * 2015-04-03 2016-10-06 University Of Massachusetts Oligonucleotide compounds for targeting huntingtin mrna
EP3408391A4 (en) * 2016-01-31 2019-08-28 University of Massachusetts BRANCHED OLIGONUCLEOTIDES
BR112021001980A2 (pt) * 2018-08-03 2021-05-04 Genzyme Corporation rnai variante contra alfa-sinucleína
CA3109133A1 (en) * 2018-08-10 2020-02-13 University Of Massachusetts Modified oligonucleotides targeting snps

Also Published As

Publication number Publication date
JP2023518268A (ja) 2023-04-28
WO2021188661A1 (en) 2021-09-23
EP4121538A4 (en) 2024-07-31
US20210363524A1 (en) 2021-11-25
CA3171757A1 (en) 2021-09-23
KR20230002327A (ko) 2023-01-05
EP4121538A1 (en) 2023-01-25
US20250043284A1 (en) 2025-02-06
CN115777021A (zh) 2023-03-10
AU2021238326A1 (en) 2022-10-13
BR112022018475A2 (pt) 2022-11-29

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