CL2017002028A1 - Mejora del suministro de partículas virales al cuerpo estriado y al córtex. - Google Patents

Mejora del suministro de partículas virales al cuerpo estriado y al córtex.

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Publication number
CL2017002028A1
CL2017002028A1 CL2017002028A CL2017002028A CL2017002028A1 CL 2017002028 A1 CL2017002028 A1 CL 2017002028A1 CL 2017002028 A CL2017002028 A CL 2017002028A CL 2017002028 A CL2017002028 A CL 2017002028A CL 2017002028 A1 CL2017002028 A1 CL 2017002028A1
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CL
Chile
Prior art keywords
supply
viral particles
striatum
cortex
improvement
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Application number
CL2017002028A
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English (en)
Inventor
Lamya Shihabuddin
Lisa M Stanek
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Genzyme Corp
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Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CL2017002028A1 publication Critical patent/CL2017002028A1/es

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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P25/16—Anti-Parkinson drugs
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N2750/00011—Details
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Neurosurgery (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

<p>LA INVENCIÓN PROPORCIONA NUEVOS MÉTODOS PARA SUMINISTRAR PARTÍCULAS VIRALES ADENO-ASOCIADAS RECOMBINANTES (rAAV) AL SISTEMA NERVIOSO CENTRAL DE UN MAMÍFERO (P. EJ. UN SER HUMANO). EN ASPECTOS, LOS MÉTODOS COMPRENDEN ADMINISTRAR PARTÍCULAS DE rAAV QUE CONTIENEN UN ÁCIDO NUCLÉICO HETERÓLOGO AL CUERPO ESTRIADO Y CAUSAR LA EXPRESIÓN DEL ÁCIDO NUCLÉICO HETERÓLOGO EN AL MENOS EL I CÓRTEX CEREBRAL Y EL CUERPO ESTRIADO DEL MAMÍFERO.</p>
CL2017002028A 2015-02-10 2017-08-08 Mejora del suministro de partículas virales al cuerpo estriado y al córtex. CL2017002028A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114544P 2015-02-10 2015-02-10
US201562220997P 2015-09-19 2015-09-19

Publications (1)

Publication Number Publication Date
CL2017002028A1 true CL2017002028A1 (es) 2018-04-13

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CL2017002028A CL2017002028A1 (es) 2015-02-10 2017-08-08 Mejora del suministro de partículas virales al cuerpo estriado y al córtex.

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US (3) US20190111157A1 (es)
EP (3) EP3256594B1 (es)
JP (4) JP6928558B2 (es)
KR (2) KR102625784B1 (es)
CN (2) CN116019934A (es)
AU (3) AU2016219398A1 (es)
BR (1) BR112017017037A2 (es)
CA (1) CA2976082A1 (es)
CL (1) CL2017002028A1 (es)
CR (1) CR20170407A (es)
DK (1) DK3256594T3 (es)
EA (1) EA201791803A1 (es)
ES (1) ES2904504T3 (es)
HU (1) HUE057272T2 (es)
IL (4) IL313840A (es)
LT (1) LT3256594T (es)
MX (2) MX388264B (es)
NZ (1) NZ772772A (es)
PH (1) PH12017501436A1 (es)
PL (1) PL3256594T3 (es)
PT (1) PT3256594T (es)
RS (1) RS62811B1 (es)
SG (3) SG11201706445SA (es)
SI (1) SI3256594T1 (es)
TN (1) TN2017000353A1 (es)
TW (1) TWI730952B (es)
WO (1) WO2016130591A2 (es)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
MX2017005834A (es) 2014-11-05 2017-11-17 Voyager Therapeutics Inc Polinucleotidos aad para el tratamiento de la enfermedad de parkinson.
CN107109407A (zh) 2014-11-14 2017-08-29 沃雅戈治疗公司 治疗肌萎缩性侧索硬化(als)的组合物和方法
SG10202001102XA (en) 2014-11-14 2020-03-30 Voyager Therapeutics Inc Modulatory polynucleotides
WO2016094783A1 (en) 2014-12-12 2016-06-16 Voyager Therapeutics, Inc. Compositions and methods for the production of scaav
CA2976082A1 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
WO2016172155A1 (en) 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
MA42934A (fr) 2015-09-24 2018-08-01 Biomarin Pharm Inc Vecteurs de facteur viii à virus adéno-associé, particules virales associées et formulations thérapeutiques comprenant ceux-ci
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3458588B1 (en) 2016-05-18 2025-10-29 Voyager Therapeutics, Inc. Modulatory polynucleotides
EP3458589A4 (en) 2016-05-18 2020-01-01 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING MORBUS HUNTINGTON
CA3035522A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
AU2017333336B2 (en) * 2016-09-30 2023-11-09 Esteve Pharmaceuticals, S.A. Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
SG11201909136PA (en) * 2017-03-31 2019-10-30 Staidson Beijing Biopharmaceuticals Co Ltd Shrna expression cassette, polynucleotide sequence carrying the same, and use thereof
CA3059891A1 (en) 2017-04-14 2018-10-18 National Taiwan University Gene therapy for aadc deficiency
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11752181B2 (en) * 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
CN111448308A (zh) 2017-08-03 2020-07-24 沃雅戈治疗公司 递送aav的组合物和方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3773748A4 (en) 2018-04-13 2022-02-23 University of Massachusetts BICISTRONIC AAV VECTORS ENCODING HEXOSAMINIDASE ALPHA AND BETA SUBUNITS AND THEIR USES
CN112424359A (zh) 2018-05-15 2021-02-26 沃雅戈治疗公司 用于治疗帕金森氏病的组合物和方法
CN113528510A (zh) * 2018-06-29 2021-10-22 武汉纽福斯生物科技有限公司 治疗遗传性视神经病变的组合物和方法
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
BR112021006344A2 (pt) 2018-10-05 2021-07-06 Univ Massachusetts vetores de raav para o tratamento de gangliosidose gm1 e gm2
JP2022513034A (ja) * 2018-11-14 2022-02-07 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
SG11202108504YA (en) * 2019-02-22 2021-09-29 Univ Pennsylvania Recombinant adeno-associated virus for treatment of grn-associated adult-onset neurodegeneration
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
WO2021007382A1 (en) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20220275367A1 (en) * 2019-07-24 2022-09-01 Voyager Therapeutics, Inc. Compositions and methods for treating huntington's disease
US12442017B2 (en) 2020-03-02 2025-10-14 National University Corporation Gunma University Microglial selective gene expression vector
JP2023517230A (ja) * 2020-03-11 2023-04-24 リモーター セラピューティクス インコーポレイテッド 中枢神経系全体にわたってタンパク質を散在させるための方法および材料
CN116171166A (zh) * 2020-06-30 2023-05-26 塞其里斯英国有限公司 水包油乳液佐剂的冷过滤
WO2022164860A1 (en) * 2021-01-27 2022-08-04 Research Institute At Nationwide Children's Hospital Materials and methods for the treatment of lysosomal acid lipase deficiency (lal-d)
WO2023113806A1 (en) 2021-12-16 2023-06-22 Affinia Therapeutics, Inc. Recombinant aav for treatment of neural disease
WO2022221193A1 (en) 2021-04-12 2022-10-20 Affinia Therapeutics Inc. Recombinant aav for treatment of neural disease
US20250297279A1 (en) * 2021-12-15 2025-09-25 Genans Biotechnology Co., Ltd Recombinant aav vectors and use thereof
US20250333758A1 (en) * 2021-12-15 2025-10-30 Genans Biotechnology Co., Ltd Microglia having car and use thereof
EP4605410A1 (en) 2022-10-19 2025-08-27 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity
WO2025151454A1 (en) * 2024-01-08 2025-07-17 AskBio Inc. Method or system of infusing and/or predicting an infusate volume
WO2025227063A1 (en) 2024-04-26 2025-10-30 Affinia Therapeutics Inc. Recombinant aavs with improved tropism and specificity

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4692147A (en) 1980-04-02 1987-09-08 Medtronic, Inc. Drug administration device
US5735815A (en) 1993-07-26 1998-04-07 Sentinel Medical, Inc. Method of using fluid jet surgical cutting tool
IL128780A0 (en) 1996-09-06 2000-01-31 Univ Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US6042579A (en) 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE316576T1 (de) 1998-05-27 2006-02-15 Avigen Inc Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
WO2001011034A2 (en) 1999-08-09 2001-02-15 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
CA2452980A1 (en) 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
GB2389791B (en) 2002-04-30 2006-12-13 Steven Gill Implantable drug delivery pump
PT2277996E (pt) 2003-05-21 2014-12-16 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
MX2007003850A (es) * 2004-10-05 2007-11-21 Genzyme Corp Canula escalonada.
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
PL1879624T3 (pl) * 2005-05-02 2012-02-29 Genzyme Corp Terapia genowa dla zaburzeń neurometabolicznych
JP5238503B2 (ja) * 2005-08-23 2013-07-17 ザ・リージェンツ・オブ・ザ・ユニバーシティ・オブ・カリフォルニア 対流増加送達を用いた治療薬の慢性的な送達のための逆流防止カニューレ及びシステム
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
HUE030903T2 (en) 2006-04-25 2017-06-28 Univ California Administration of growth factors for the treatment of CNS disorders
JP2009535360A (ja) 2006-04-26 2009-10-01 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 高分子量神経治療薬の対流増加送達のための組成物および方法
RU2442617C2 (ru) 2007-05-17 2012-02-20 Медженезиз Терапьютикс Инк. Конвекционный доставляющий катетер со съемным элементом жесткости и способ его применения
BRPI1007155A2 (pt) 2009-01-29 2017-05-30 Univ Of California San Francisco métodos para tratar um distúrbio neurológico cortical, e para dispensar um agente terapêutico para o córtex em um primata
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purification of recombinant aav vectors
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
WO2012109667A1 (en) * 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
CN103502458B (zh) 2011-02-17 2016-11-16 宾夕法尼亚大学托管会 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法
KR102863726B1 (ko) * 2014-05-02 2025-09-25 젠자임 코포레이션 망막 및 cns 유전자 요법을 위한 aav 벡터
CA2976082A1 (en) * 2015-02-10 2016-08-18 Genzyme Corporation Enhanced delivery of viral particles to the striatum and cortex
CN111417231A (zh) 2019-01-04 2020-07-14 青岛海尔股份有限公司 电磁波发生系统及具有该电磁波发生系统的加热装置

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