HK1214179A1 - 通過選擇性遞送寡核苷酸分子至特定神經元類型治療帕金森病的組合物和方法 - Google Patents

通過選擇性遞送寡核苷酸分子至特定神經元類型治療帕金森病的組合物和方法

Info

Publication number
HK1214179A1
HK1214179A1 HK16102420.8A HK16102420A HK1214179A1 HK 1214179 A1 HK1214179 A1 HK 1214179A1 HK 16102420 A HK16102420 A HK 16102420A HK 1214179 A1 HK1214179 A1 HK 1214179A1
Authority
HK
Hong Kong
Prior art keywords
compositions
treatment
methods
parkinson disease
oligonucleotide molecules
Prior art date
Application number
HK16102420.8A
Other languages
English (en)
Inventor
.卡爾莫納奧羅斯科
.蒙泰費爾特羅
.阿爾瓦拉多
.維拉博韋爾
.博爾托洛齊
.阿蒂加斯佩雷斯
Original Assignee
Nlife Therapeutics S L
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 Nlife Therapeutics S L filed Critical Nlife Therapeutics S L
Publication of HK1214179A1 publication Critical patent/HK1214179A1/zh

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/54Medicinal 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
    • A61K47/55Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
HK16102420.8A 2012-10-26 2016-03-02 通過選擇性遞送寡核苷酸分子至特定神經元類型治療帕金森病的組合物和方法 HK1214179A1 (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261719284P 2012-10-26 2012-10-26
EP12382414 2012-10-26
PCT/EP2013/072410 WO2014064257A1 (en) 2012-10-26 2013-10-25 Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types

Publications (1)

Publication Number Publication Date
HK1214179A1 true HK1214179A1 (zh) 2016-07-22

Family

ID=47148690

Family Applications (1)

Application Number Title Priority Date Filing Date
HK16102420.8A HK1214179A1 (zh) 2012-10-26 2016-03-02 通過選擇性遞送寡核苷酸分子至特定神經元類型治療帕金森病的組合物和方法

Country Status (9)

Country Link
US (2) US9084825B2 (zh)
EP (1) EP2911695B1 (zh)
JP (1) JP6386461B2 (zh)
CN (1) CN104884094A (zh)
AU (1) AU2013336581A1 (zh)
CA (1) CA2890112A1 (zh)
HK (1) HK1214179A1 (zh)
MX (1) MX2015005352A (zh)
WO (1) WO2014064257A1 (zh)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20130103662A (ko) 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
CA2817960C (en) 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN104039960B (zh) 2011-08-04 2017-05-10 耶达研究及发展有限公司 用于治疗和诊断与五羟色胺‑、肾上腺素‑、去甲肾上腺素‑、谷氨酸‑和促肾上腺皮质素释放激素相关的病症的微rna和包含微rna的组合物
EP2911695B1 (en) 2012-10-26 2023-11-29 Palomo Limited Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
JP2017506228A (ja) 2014-02-05 2017-03-02 イェダ リサーチ アンド ディベロップメント カンパニー リミテッドYeda Research And Development Co.Ltd. セロトニン放出ホルモン、アドレナリン放出ホルモン、ノルアドレナリン放出ホルモン、グルタミン酸放出ホルモンおよび副腎皮質刺激ホルモン放出ホルモン関連の医学的状態の処置および診断のためのマイクロrnaおよび該マイクロrnaを含む組成物
WO2016186968A1 (en) 2015-05-15 2016-11-24 The Arizona Board Of Regents On Behalf Of The University Of Arizona Compositions and methods for treating motor disorders
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US10733023B1 (en) * 2015-08-06 2020-08-04 D2Iq, Inc. Oversubscription scheduling
EP3415524A4 (en) * 2016-01-07 2019-09-04 Osaka University INHIBITOR OF EXPRESSION OF α-SYNUCLEIN
WO2019009299A1 (ja) * 2017-07-05 2019-01-10 国立大学法人大阪大学 α-シヌクレイン発現抑制するENAアンチセンスオリゴヌクレオチド
WO2019009298A1 (ja) * 2017-07-05 2019-01-10 国立大学法人大阪大学 α-シヌクレイン発現抑制剤
KR101890925B1 (ko) 2017-08-08 2018-08-24 주식회사 이레산업 캠핑용 그릴 세트
KR20200060496A (ko) * 2017-10-04 2020-05-29 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 그의 용도
WO2019079887A1 (en) * 2017-10-23 2019-05-02 Carleton University APTAMERS AS A THERAPEUTIC TO PREVENT AGGREGATION OF PROTEINS IN NEURODEGENERATIVE DISEASE
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
EP3737759A1 (en) 2018-01-12 2020-11-18 Roche Innovation Center Copenhagen A/S Alpha-synuclein antisense oligonucleotides and uses thereof
EP3883612A4 (en) * 2018-11-21 2021-09-29 The University Of Georgia Research Foundation, Inc. BIMODAL NANOPARTICLE CONJUGATES FOR NON-INVASIVE TISSUE IMAGING OF THE CENTRAL NERVOUS SYSTEM

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL66831A0 (en) 1981-10-05 1982-12-31 Kefalas As Indane derivatives
US4902505A (en) 1986-07-30 1990-02-20 Alkermes Chimeric peptides for neuropeptide delivery through the blood-brain barrier
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5256775A (en) 1989-06-05 1993-10-26 Gilead Sciences, Inc. Exonuclease-resistant oligonucleotides
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US6455736B1 (en) 1994-12-16 2002-09-24 Uop Llc Process for preparation of pharmaceutically desired sertraline and sertraline analogs
EP1108724B1 (en) 1996-01-16 2007-09-19 Sirna Therpeutics, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
WO2006006948A2 (en) * 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease
WO2005067391A2 (en) * 2004-01-19 2005-07-28 Technion Research & Development Foundation Ltd. Diagnostic test for parkinson's disease
WO2007050789A2 (en) * 2005-10-25 2007-05-03 Nëdken Corporation Glycosyl-oligonucleotide conjugates and methods
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US20090176729A1 (en) * 2007-12-14 2009-07-09 Alnylam Pharmaceuticals, Inc. Method of treating neurodegenerative disease
MX2010006925A (es) 2007-12-20 2011-05-02 Angiochem Inc Conjugados de polipeptido-acido nucleico y sus usos.
US9255266B2 (en) * 2009-05-06 2016-02-09 Rutgers, The State University Of New Jersey RNA targeting in alpha-synucleinopathies
WO2011087804A2 (en) 2009-12-21 2011-07-21 Medtronic, Inc. Peptide-polynucleotide compositions, and methods for transfecting a cell with dna and treatment of neurodegenerative disease
KR20130103662A (ko) 2010-04-19 2013-09-24 엔라이프 떼라퓨틱스, 에스.엘. 올리고뉴클레오티드 분자들의 특정 뉴런 유형들로의 선택적인 전달을 위한 조성물들 및 방법
WO2012027713A2 (en) * 2010-08-26 2012-03-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of snca
CA2817960C (en) * 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP2911695B1 (en) 2012-10-26 2023-11-29 Palomo Limited Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types
US9938525B2 (en) 2012-10-26 2018-04-10 Nlife Therapeutics, S.L. Compositions and methods for selective delivery of oligonucleotide molecules to cell types

Also Published As

Publication number Publication date
US20160083726A1 (en) 2016-03-24
AU2013336581A1 (en) 2015-06-11
EP2911695B1 (en) 2023-11-29
US10125363B2 (en) 2018-11-13
JP6386461B2 (ja) 2018-09-05
US20140120158A1 (en) 2014-05-01
JP2015536927A (ja) 2015-12-24
WO2014064257A1 (en) 2014-05-01
US9084825B2 (en) 2015-07-21
EP2911695C0 (en) 2023-11-29
EP2911695A1 (en) 2015-09-02
MX2015005352A (es) 2015-10-29
CN104884094A (zh) 2015-09-02
CA2890112A1 (en) 2014-05-01

Similar Documents

Publication Publication Date Title
HK1214179A1 (zh) 通過選擇性遞送寡核苷酸分子至特定神經元類型治療帕金森病的組合物和方法
IL274469A (en) Compositions, uses and methods for the treatment of metabolic disorders and diseases
HRP20171254T1 (hr) Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
LT2560687T (lt) Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams
HK1214832A1 (zh) 用於代謝病症和疾病治療的組合物和方法
HK1210216A1 (zh) 靶向 的雙鏈寡核苷酸分子及其使用方法
EP2872120A4 (en) TARGETED THERAPEUTIC NANOPARTICLES
LT2819663T (lt) Nauji terapiniai parkinsono ligos gydymo būdai
HK1215173A1 (zh) 治療金黃色葡萄球菌相關疾病的方法
HUS2000020I1 (hu) Ciklezonid a légúti betegségek kezelésére lovaknál
IL233544B (en) A compound containing a modified oligonucleotide consisting of 10 to 30 linked nucleotides for use in the treatment of familial dysautonomia
IL232710A0 (en) l-serine preparations, methods and uses for the treatment of neurodegenerative diseases and disorders
IL234606A0 (en) Innovative methods and compounds for the treatment of diseases
HK1191233A1 (zh) 用於處理與缺乏血液供給、休克和神經損傷相關的疾病的方法和組合物
PL2892556T3 (pl) Kompozycje i sposoby odnoszące się do leczenia chorób
ES2627541T8 (es) Métodos para tratar la enfermedad de Parkinson
EP2822600A4 (en) METHOD OF DISTRIBUTING NUCLEIC ACID ANOPARTICLES TO THE CENTRAL NERVOUS SYSTEM FOR THE TREATMENT OF DISEASES OF THE CENTRAL NERVOUS SYSTEM
EP2882451A4 (en) METHOD AND COMPOSITIONS FOR THE TREATMENT OF SUFFERING IN CONNECTION WITH LACK OF BLOOD SUPPLIES, SHOCKS AND INJURIES
HK1243733A1 (zh) 用於治療神經肌肉障礙的具有改善特性的rna調節寡核苷酸