BR112021001878A2 - métodos de terapia genética para o controle da função do órgão - Google Patents

métodos de terapia genética para o controle da função do órgão Download PDF

Info

Publication number
BR112021001878A2
BR112021001878A2 BR112021001878-6A BR112021001878A BR112021001878A2 BR 112021001878 A2 BR112021001878 A2 BR 112021001878A2 BR 112021001878 A BR112021001878 A BR 112021001878A BR 112021001878 A2 BR112021001878 A2 BR 112021001878A2
Authority
BR
Brazil
Prior art keywords
aav
mammal
vector
viral vector
nerve
Prior art date
Application number
BR112021001878-6A
Other languages
English (en)
Portuguese (pt)
Inventor
Roberta Marongiu
Michael G. Kaplitt
Original Assignee
Cornell University
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 Cornell University filed Critical Cornell University
Publication of BR112021001878A2 publication Critical patent/BR112021001878A2/pt

Links

Classifications

    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1787Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/007Pulmonary tract; Aromatherapy
    • A61K9/0073Sprays or powders for inhalation; Aerolised or nebulised preparations generated by other means than thermal energy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • A61P11/14Antitussive agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pulmonology (AREA)
  • Neurosurgery (AREA)
  • Otolaryngology (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
BR112021001878-6A 2018-07-31 2019-07-31 métodos de terapia genética para o controle da função do órgão BR112021001878A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862712669P 2018-07-31 2018-07-31
US62/712,669 2018-07-31
PCT/US2019/044290 WO2020028466A1 (en) 2018-07-31 2019-07-31 Gene therapy methods to control organ function

Publications (1)

Publication Number Publication Date
BR112021001878A2 true BR112021001878A2 (pt) 2021-04-27

Family

ID=69230802

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021001878-6A BR112021001878A2 (pt) 2018-07-31 2019-07-31 métodos de terapia genética para o controle da função do órgão

Country Status (14)

Country Link
US (1) US20210301306A1 (https=)
EP (1) EP3829718A4 (https=)
JP (2) JP2021533126A (https=)
KR (1) KR20210052450A (https=)
CN (1) CN113301956B (https=)
AU (1) AU2019315445A1 (https=)
BR (1) BR112021001878A2 (https=)
CA (1) CA3108324A1 (https=)
EA (1) EA202190358A1 (https=)
IL (1) IL280531A (https=)
MX (1) MX2021001336A (https=)
PH (1) PH12021550243A1 (https=)
SG (1) SG11202101042SA (https=)
WO (1) WO2020028466A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150111918A1 (en) 2012-03-08 2015-04-23 Medtronic Ardian Luxembourg S.a.r.l Immune system neuromodulation and associated systems and methods
EP3226795B1 (en) 2014-12-03 2020-08-26 Metavention, Inc. Systems for modulating nerves or other tissue
US10524859B2 (en) 2016-06-07 2020-01-07 Metavention, Inc. Therapeutic tissue modulation devices and methods
CN115707773A (zh) * 2021-08-20 2023-02-21 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
WO2023019565A1 (zh) * 2021-08-20 2023-02-23 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
CN116019909B (zh) * 2022-09-16 2025-07-29 上海市第十人民医院 一种CRISPR/Cas9纳米系统
CN116115758B (zh) * 2022-11-22 2024-12-03 上海市内分泌代谢病研究所 Gabaa受体作为靶点在制备或筛选降血脂、治疗肥胖和/或改善代谢的药物中的应用
CN121313878A (zh) * 2024-12-04 2026-01-13 良渚实验室 治疗帕金森病的化学遗传学基因治疗新方案

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US5464758A (en) 1993-06-14 1995-11-07 Gossen; Manfred Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
FR2814642B1 (fr) 2000-10-03 2005-07-01 Ass Pour Le Dev De La Rech En Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee
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
JP2009523006A (ja) * 2004-05-07 2009-06-18 アプレラ コーポレイション 脈管疾患に関連する遺伝的多型、その検出方法および使用
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
EP2561073B1 (en) * 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
KR20150020250A (ko) * 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
CA3011939A1 (en) * 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
US20170218842A1 (en) * 2016-02-02 2017-08-03 General Electric Company Adjusting Airflow Distortion in Gas Turbine Engine
CA3014732A1 (en) * 2016-03-09 2017-09-14 Fadi ASSAF Using dreadd for neuronal modulation in treating neuronal
MA44546B1 (fr) * 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
WO2018008770A1 (ja) * 2016-07-06 2018-01-11 国立研究開発法人国立循環器病研究センター 末梢神経を操作する方法、脳および脊髄を除く臓器などにおいて神経細胞機能を変化させる機能を発生させる方法、および新規疾病の予防または治療方法、ならびに末梢神経投与用医薬
MX2022004812A (es) * 2019-10-22 2023-02-23 Applied Genetic Tech Corporation Sistemas de virus adeno-asociados (aav) para el tratamiento de enfermedades o trastornos neurodegenerativos asociados a la progranulina.

Also Published As

Publication number Publication date
PH12021550243A1 (en) 2021-11-22
CN113301956B (zh) 2025-06-24
EP3829718A4 (en) 2022-06-22
CN113301956A (zh) 2021-08-24
MX2021001336A (es) 2021-09-10
AU2019315445A1 (en) 2021-03-18
US20210301306A1 (en) 2021-09-30
SG11202101042SA (en) 2021-02-25
EP3829718A1 (en) 2021-06-09
WO2020028466A1 (en) 2020-02-06
KR20210052450A (ko) 2021-05-10
IL280531A (en) 2021-03-25
EA202190358A1 (ru) 2021-05-04
CA3108324A1 (en) 2020-02-06
JP2024041871A (ja) 2024-03-27
JP2021533126A (ja) 2021-12-02

Similar Documents

Publication Publication Date Title
BR112021001878A2 (pt) métodos de terapia genética para o controle da função do órgão
JP6805174B2 (ja) 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
ES2704677T3 (es) Suministro eficaz de genes grandes por vectores de AAV duales
US20210395776A1 (en) Frataxin expression constructs having engineered promoters and methods of use thereof
ES2741502T3 (es) Cápside de AAV8 modificada para la transferencia génica para terapias de retina
Zheng et al. Lentiviral vectors and adeno‐associated virus vectors: useful tools for gene transfer in pain research
ES2919880T3 (es) Sistemas de vectores múltiples y usos de los mismos
JP2017531652A (ja) Aavに基づく遺伝子治療
US20250057899A1 (en) Gene therapy for ciliopathies
WO2021247995A2 (en) Compositions and methods of treating neuropathic pain
ES2862164T3 (es) Genoterapia de la enfermedad de Wilson
JP2021520811A (ja) ヘキソサミニダーゼアルファおよびベータサブユニットをコードしているバイシストロニックaavベクターならびにその使用
CN115697487A (zh) 神经障碍的活性依赖性基因疗法
WO2016073900A1 (en) Compositions and methods of treating usher syndrome iii
ES2819973T3 (es) Terapia génica para la diabetes
ES2883358T3 (es) Gen Dgkk recombinante para terapia génica del síndrome de X frágil
ES2624659T3 (es) Composiciones terapéuticas de SERCA2 y métodos de uso
JP2025523953A (ja) 肢帯型筋ジストロフィーのためのauf1遺伝子療法
EA049462B1 (ru) Способы генной терапии для контроля функции органов
US20220280657A1 (en) Genetic Construct
KR102933125B1 (ko) 평활근 세포 특이적 유전자 전달이 가능한 아데노연관바이러스 벡터
US20260041791A1 (en) Compositions and methods for treatment of achromotopsia
BR102020023425A2 (pt) Vetor biológico para tratamento de doenças neurodegenerativas com base em terapia gênica
KR20250090302A (ko) Bdnf 유전자 요법

Legal Events

Date Code Title Description
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]