PE20211749A1 - Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos - Google Patents
Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estosInfo
- 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
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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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- C07K14/435—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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61K31/00—Medicinal preparations containing organic active ingredients
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
- A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
- A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
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- A—HUMAN NECESSITIES
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- 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
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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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- 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
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- C07K—PEPTIDES
- C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
- C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/505—Medicinal preparations containing antigens or antibodies comprising antibodies
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- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/3341—5-Methylcytosine
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- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/3521—Methyl
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- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
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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).
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 |
Family
ID=65269098
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2020000954A PE20211749A1 (es) | 2018-01-12 | 2019-01-11 | Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos |
Country Status (15)
Country | Link |
---|---|
US (1) | US20200362347A1 (es) |
EP (1) | EP3737761A1 (es) |
JP (1) | JP2021510511A (es) |
KR (1) | KR20200140240A (es) |
CN (1) | CN111819283A (es) |
AU (1) | AU2019208011A1 (es) |
BR (1) | BR112020014011A2 (es) |
CA (1) | CA3087966A1 (es) |
CO (1) | CO2020009715A2 (es) |
EA (1) | EA202091693A1 (es) |
IL (1) | IL275903A (es) |
MX (1) | MX2020007439A (es) |
PE (1) | PE20211749A1 (es) |
SG (1) | SG11202006528XA (es) |
WO (1) | WO2019140236A1 (es) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP7136790B2 (ja) | 2017-02-17 | 2022-09-13 | ブリストル-マイヤーズ スクイブ カンパニー | アルファ-シヌクレインに対する抗体およびその使用 |
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 |
CA3088112A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
EP3752612A4 (en) | 2018-02-12 | 2021-11-10 | Ionis Pharmaceuticals, Inc. | MODIFIED COMPOUNDS AND USES THEREOF |
WO2021141917A1 (en) * | 2020-01-06 | 2021-07-15 | AUM LifeTech, Inc. | Antisense oligonucleotides for treatment of neurological disorders |
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JP2823959B2 (ja) | 1991-10-24 | 1998-11-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド |
JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
EP2341058A3 (en) | 1997-09-12 | 2011-11-23 | Exiqon A/S | Oligonucleotide Analogues |
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PT1178999E (pt) | 1999-05-04 | 2007-06-26 | Santaris Pharma As | Análogos de l-ribo-lna |
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DK2284269T3 (en) | 2002-11-18 | 2017-10-23 | Roche Innovation Ct Copenhagen As | Antisense design |
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WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
KR20080068019A (ko) * | 2005-09-15 | 2008-07-22 | 산타리스 팔마 에이/에스 | 아포지단백질-b100 발현 억제용 rna 길항제 화합물 |
JP5342881B2 (ja) | 2006-01-27 | 2013-11-13 | アイシス ファーマシューティカルズ, インコーポレーテッド | 6−修飾された二環式核酸類似体 |
CN103554205A (zh) | 2006-05-05 | 2014-02-05 | Isis制药公司 | 调节gccr的表达的化合物和方法 |
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CA3088112A1 (en) * | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
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2019
- 2019-01-11 AU AU2019208011A patent/AU2019208011A1/en active Pending
- 2019-01-11 CA CA3087966A patent/CA3087966A1/en active Pending
- 2019-01-11 BR BR112020014011-2A patent/BR112020014011A2/pt unknown
- 2019-01-11 CN CN201980018033.XA patent/CN111819283A/zh active Pending
- 2019-01-11 WO PCT/US2019/013255 patent/WO2019140236A1/en unknown
- 2019-01-11 KR KR1020207023162A patent/KR20200140240A/ko unknown
- 2019-01-11 EP EP19702766.7A patent/EP3737761A1/en active Pending
- 2019-01-11 EA EA202091693A patent/EA202091693A1/ru unknown
- 2019-01-11 PE PE2020000954A patent/PE20211749A1/es unknown
- 2019-01-11 US US16/961,578 patent/US20200362347A1/en active Pending
- 2019-01-11 JP JP2020538629A patent/JP2021510511A/ja active Pending
- 2019-01-11 SG SG11202006528XA patent/SG11202006528XA/en unknown
- 2019-01-11 MX MX2020007439A patent/MX2020007439A/es unknown
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2020
- 2020-07-07 IL IL275903A patent/IL275903A/en unknown
- 2020-08-05 CO CONC2020/0009715A patent/CO2020009715A2/es unknown
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CO2020009715A2 (es) | 2020-08-21 |
MX2020007439A (es) | 2020-09-14 |
WO2019140236A1 (en) | 2019-07-18 |
US20200362347A1 (en) | 2020-11-19 |
CA3087966A1 (en) | 2019-07-18 |
EP3737761A1 (en) | 2020-11-18 |
IL275903A (en) | 2020-08-31 |
SG11202006528XA (en) | 2020-08-28 |
JP2021510511A (ja) | 2021-04-30 |
EA202091693A1 (ru) | 2021-04-14 |
KR20200140240A (ko) | 2020-12-15 |
BR112020014011A2 (pt) | 2020-12-08 |
CN111819283A (zh) | 2020-10-23 |
AU2019208011A1 (en) | 2020-07-23 |
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