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PE20211749A1 - Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos - Google Patents

Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos

Info

Publication number
PE20211749A1
PE20211749A1 PE2020000954A PE2020000954A PE20211749A1 PE 20211749 A1 PE20211749 A1 PE 20211749A1 PE 2020000954 A PE2020000954 A PE 2020000954A PE 2020000954 A PE2020000954 A PE 2020000954A PE 20211749 A1 PE20211749 A1 PE 20211749A1
Authority
PE
Peru
Prior art keywords
alpha
nucleotides
snca
synuclein
sinuclein
Prior art date
Application number
PE2020000954A
Other languages
English (en)
Inventor
Richard E Olson
Angela M Cacace
Jr Jere E Meredith
Nino Devidze
James K Loy
Carl J Baldick
Annapurna Pendri
Ivar M Mcdonald
Peter Hagedorn
Marianne Lerbech Jensen
Original Assignee
Bristol Myers Squibb Co
Roche Innovation Ct Copenhagen As
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 Bristol Myers Squibb Co, Roche Innovation Ct Copenhagen As filed Critical Bristol Myers Squibb Co
Publication of PE20211749A1 publication Critical patent/PE20211749A1/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
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    • 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
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    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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Abstract

La presente invencion se refiere a un oligonucleotido antisentido (ASO) que comprende una secuencia de nucleotidos contiguos de 10 a 30 nucleotidos de longitud que son complementarios a una secuencia de acido nucleico dentro de un transcripto de alfa-sinucleina (SNCA). La secuencia de nucleotidos comprende los nucleotidos 7592-7637 que tiene la SEQ ID NO: 1. Tambien se refiere a un conjugado o composicion para la fabricacion y administracion de un medicamento. Dichos nucleotidos actuan sobre una union exon/intron del transcripto de alfa-sinucleina (SNCA) en una celula, lo que reduce la expresion de mRNA de la proteina alfasinucleina (SNCA) en al menos alrededor de 10% hasta al menos alrededor de 90%, util en el tratamiento de trastornos medicos neurologicos tales como atrofia multisistemica, enfermedad de Parkinson y la demencia asociada a la enfermedad de Parkinson (PDD).
PE2020000954A 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos PE20211749A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862616994P 2018-01-12 2018-01-12
PCT/US2019/013255 WO2019140236A1 (en) 2018-01-12 2019-01-11 Antisense oligonucleotides targeting alpha-synuclein and uses thereof

Publications (1)

Publication Number Publication Date
PE20211749A1 true PE20211749A1 (es) 2021-09-06

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PE2020000954A PE20211749A1 (es) 2018-01-12 2019-01-11 Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos

Country Status (15)

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US (2) US12241065B2 (es)
EP (1) EP3737761A1 (es)
JP (2) JP7547201B2 (es)
KR (1) KR102839166B1 (es)
CN (1) CN111819283A (es)
AU (1) AU2019208011B2 (es)
BR (1) BR112020014011A2 (es)
CA (1) CA3087966A1 (es)
CO (1) CO2020009715A2 (es)
EA (1) EA202091693A1 (es)
IL (1) IL275903B1 (es)
MX (1) MX2020007439A (es)
PE (1) PE20211749A1 (es)
SG (1) SG11202006528XA (es)
WO (1) WO2019140236A1 (es)

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WO2018151821A1 (en) 2017-02-17 2018-08-23 Bristol-Myers Squibb Company Antibodies to alpha-synuclein and uses thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
JP7547201B2 (ja) 2018-01-12 2024-09-09 ブリストル－マイヤーズ スクイブ カンパニー アルファ－シヌクレインを標的とするアンチセンスオリゴヌクレオチドおよびその使用
MY203356A (en) * 2018-01-12 2024-06-25 Bristol Myers Squibb Co Antisense oligonucleotides targeting alpha-synuclein and uses thereof
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
EP4096680A4 (en) * 2020-01-06 2024-07-10 AUM LifeTech, Inc. Antisense oligonucleotides for treatment of neurological disorders
MX2023010535A (es) * 2021-03-08 2023-09-19 Servier Lab Oligonucleotidos antisentido para inhibir la expresion de la alfa-sinucleina.
KR102680316B1 (ko) * 2021-11-01 2024-07-01 오토텔릭바이오 주식회사 안티센스 올리고뉴클레오타이드
EP4665357A2 (en) * 2023-02-17 2025-12-24 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating alpha-synuclein expression
WO2024196752A2 (en) * 2023-03-17 2024-09-26 Duke University Compositions for cell specific snca-targeted gene therapy and methods of making and using same

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