IL288266A - Gene therapy for Alzheimer's disease - Google Patents

Gene therapy for Alzheimer's disease

Info

Publication number
IL288266A
IL288266A IL288266A IL28826621A IL288266A IL 288266 A IL288266 A IL 288266A IL 288266 A IL288266 A IL 288266A IL 28826621 A IL28826621 A IL 28826621A IL 288266 A IL288266 A IL 288266A
Authority
IL
Israel
Prior art keywords
alzheimer
disease
gene therapy
therapy
gene
Prior art date
Application number
IL288266A
Other languages
English (en)
Hebrew (he)
Original Assignee
Brigham & Womens Hospital Inc
Massachusetts Gen Hospital
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 Brigham & Womens Hospital Inc, Massachusetts Gen Hospital filed Critical Brigham & Womens Hospital Inc
Publication of IL288266A publication Critical patent/IL288266A/en

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
    • 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
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4711Alzheimer's disease; Amyloid plaque core protein
    • 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
    • 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
    • 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
    • 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
    • C12N2750/14171Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Psychiatry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
IL288266A 2019-05-24 2021-11-21 Gene therapy for Alzheimer's disease IL288266A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962852716P 2019-05-24 2019-05-24
PCT/US2020/034040 WO2020242892A1 (en) 2019-05-24 2020-05-21 Gene therapy for alzheimer's disease

Publications (1)

Publication Number Publication Date
IL288266A true IL288266A (en) 2022-01-01

Family

ID=73553511

Family Applications (1)

Application Number Title Priority Date Filing Date
IL288266A IL288266A (en) 2019-05-24 2021-11-21 Gene therapy for Alzheimer's disease

Country Status (8)

Country Link
US (1) US12540334B2 (https=)
EP (1) EP3976637A4 (https=)
JP (1) JP7686573B6 (https=)
CN (1) CN114144425B (https=)
AU (1) AU2020285638A1 (https=)
CA (1) CA3141712A1 (https=)
IL (1) IL288266A (https=)
WO (1) WO2020242892A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3100946A1 (en) 2018-05-22 2019-11-28 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
BR112022010373A2 (pt) 2019-11-29 2022-08-16 Paros Bio Inc Terapia genética para doenças neurodegenerativas
CN118660970A (zh) * 2022-01-29 2024-09-17 上海日馨医药科技股份有限公司 表达tpk的重组病毒及其治疗阿尔茨海默病的用途
AU2023312939A1 (en) * 2022-07-25 2025-03-06 Katholieke Universiteit Leuven Antisense oligonucleotides for treating a disease or condition associated with an abnormal processing of app

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
EP0705344B8 (en) 1993-06-24 2006-05-10 Advec Inc. Adenovirus vectors for gene therapy
AU687117B2 (en) 1993-10-25 1998-02-19 Canji, Inc. Recombinant adenoviral vector and methods of use
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
ATE386131T1 (de) 1994-04-13 2008-03-15 Univ Rockefeller Aav-vermittelte überbringung von dna in zellen des nervensystems
US5986054A (en) 1995-04-28 1999-11-16 The Hospital For Sick Children, Hsc Research And Development Limited Partnership Genetic sequences and proteins related to alzheimer's disease
JP2000506375A (ja) 1996-01-26 2000-05-30 エイチエスシー リサーチ アンド デベロップメント リミテッド パートナーシップ アルツハイマー病に関連する核酸およびタンパク質、ならびにその使用
AR020107A1 (es) 1998-07-09 2002-04-10 Boehringer Ingelheim Pharma Metodo para identificar una sustancia capaz de reducir o eliminar la actividad de la presenilinasa, las sustancias identificables con dicho metodo, el usode estas ultimas en la elaboracion de un medicamento para el tratamiento de enfermedades neurodegenerativas y las composiciones farmaceuticas que
CA2333467A1 (en) 1998-07-16 2000-01-27 Incyte Pharmaceuticals, Inc. Human presenilin-associated protein
US6979537B2 (en) 2000-01-10 2005-12-27 Scios, Inc. Methods for identifying inhibitors of neuronal degeneration
US6686449B2 (en) 2000-06-30 2004-02-03 Pharmacia & Upjohn Company Mutant presenilin 1 polypeptides
AU2003243151A1 (en) 2002-08-16 2004-03-03 Agensys, Inc. Nucleic acid and corresponding protein entitled 251p5g2 useful in treatment and detection of cancer
US7271313B2 (en) 2003-04-09 2007-09-18 The Brigham And Women's Hospital, Inc. Presenilin-deficient mouse model of age-dependent neurodegeneration and cognitive loss
CA2528963A1 (en) 2003-06-27 2005-01-13 Sirna Therapeutics, Inc. Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina)
WO2005037226A2 (en) 2003-10-17 2005-04-28 Georgia Tech Research Corporation Genetically engineered enteroendocrine cells for treating glucose-related metabolic disorders
PT1675956E (pt) 2003-10-21 2010-11-04 Merck Serono Sa Sequência mínima de adn que actua como um isolador de cromatina e sua utilização na expressão de proteína
WO2005096781A2 (en) 2004-04-06 2005-10-20 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using rna interference
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロnasおよびその使用
WO2005116250A2 (en) 2004-05-26 2005-12-08 Rosetta Genomics Ltd. Viral and viral associated mirnas and uses thereof
US8129334B2 (en) 2006-03-31 2012-03-06 The Regents Of The University Of California Methods and compositions for treating neurodegenerative disorders and Alzheimer'S disease and improving normal memory
US9085778B2 (en) 2006-05-03 2015-07-21 VL27, Inc. Exosome transfer of nucleic acids to cells
WO2009042727A1 (en) 2007-09-24 2009-04-02 The Johns Hopkins University Immediate early gene arc interacts with endocytic machinery and regulates the trafficking and function of presenilin
KR20120089743A (ko) 2009-11-09 2012-08-13 제네포드 테라퓨틱스 에이비 생체 내에서 최적화된 뉴런 특이적 연속 도파 합성을 위한 신규한 바이러스 벡터 구성물
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
CN103816540B (zh) 2012-11-16 2018-01-02 中国科学院上海生命科学研究院 降低β‑抑制蛋白1与APH‑1蛋白的结合的物质在制备防治神经退行性疾病药物中的应用
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
HK1222673A1 (zh) 2013-07-12 2017-07-07 The Children's Hospital Of Philadelphia Aav载体和用於抗aav(腺相关病毒)中和抗体的检测
GB201404470D0 (en) * 2014-03-13 2014-04-30 Ucl Business Plc Therapeutic methods and materials
GB201413665D0 (en) 2014-07-03 2014-09-17 Transimmune Ag And Yale University Method for obtaining globally activated monocytes
WO2016118780A1 (en) * 2015-01-21 2016-07-28 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2016154055A1 (en) 2015-03-20 2016-09-29 Bluebird Bio, Inc. Vector formulations
WO2016209654A1 (en) 2015-06-22 2016-12-29 The Board Of Trustees Of The Leland Stanford Junior University Methods and devices for imaging and/or optogenetic control of light-responsive neurons
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
CN117018228A (zh) 2016-05-27 2023-11-10 创思瑞普泰克斯公司 用合成信使rna治疗原发性纤毛运动障碍
WO2018045022A1 (en) 2016-08-30 2018-03-08 University Of South Florida Adipose derived stem cell exosomes and uses thereof
EP3573627A4 (en) * 2017-01-24 2020-11-25 Fred Hutchinson Cancer Research Center SYSTEMS AND METHODS FOR EXPANSION OF HEMATOPOIETIC CELLS USING HYDROGELS
US11713470B2 (en) 2017-03-20 2023-08-01 University of Pittsburgh—of the Commonwealth System of Higher Education Targeted gene therapies for pain and other neuro-related disorders
ES3039647T3 (en) 2017-05-31 2025-10-23 Arcturus Therapeutics Inc Synthesis and structure of high potency rna therapeutics
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3100946A1 (en) 2018-05-22 2019-11-28 The Brigham And Women's Hospital, Inc. Gene therapy for alzheimer's disease
BR112022010373A2 (pt) 2019-11-29 2022-08-16 Paros Bio Inc Terapia genética para doenças neurodegenerativas
JP2023512079A (ja) 2020-01-31 2023-03-23 パロス バイオ インコーポレイテッド ポリヌクレオチドサイレンシング及び置換を使用する神経変性障害のための遺伝子療法

Also Published As

Publication number Publication date
JP7686573B2 (ja) 2025-06-02
EP3976637A1 (en) 2022-04-06
WO2020242892A1 (en) 2020-12-03
CN114144425A (zh) 2022-03-04
JP7686573B6 (ja) 2025-07-04
AU2020285638A1 (en) 2021-12-23
US20220235374A1 (en) 2022-07-28
EP3976637A4 (en) 2023-07-12
CN114144425B (zh) 2025-05-13
JP2022533451A (ja) 2022-07-22
CA3141712A1 (en) 2020-12-03
US12540334B2 (en) 2026-02-03

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