IL293599A - Retromer stabilization for the treatment of Alzheimer's disease and other neurodegenerative disorders - Google Patents

Retromer stabilization for the treatment of Alzheimer's disease and other neurodegenerative disorders

Info

Publication number
IL293599A
IL293599A IL293599A IL29359922A IL293599A IL 293599 A IL293599 A IL 293599A IL 293599 A IL293599 A IL 293599A IL 29359922 A IL29359922 A IL 29359922A IL 293599 A IL293599 A IL 293599A
Authority
IL
Israel
Prior art keywords
retromer
transgene
core protein
vps26b
vps26a
Prior art date
Application number
IL293599A
Other languages
English (en)
Hebrew (he)
Original Assignee
Univ Columbia
Univ Cornell
Meiragtx Uk Ii Ltd
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 Univ Columbia, Univ Cornell, Meiragtx Uk Ii Ltd filed Critical Univ Columbia
Publication of IL293599A publication Critical patent/IL293599A/en

Links

Classifications

    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
IL293599A 2019-12-05 2020-12-07 Retromer stabilization for the treatment of Alzheimer's disease and other neurodegenerative disorders IL293599A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962943999P 2019-12-05 2019-12-05
US202063074578P 2020-09-04 2020-09-04
PCT/US2020/063627 WO2021113824A1 (en) 2019-12-05 2020-12-07 Stabilization of retromer for the treatment of alzheimer's disease and other neurodegenerative disorders

Publications (1)

Publication Number Publication Date
IL293599A true IL293599A (en) 2022-08-01

Family

ID=76222705

Family Applications (1)

Application Number Title Priority Date Filing Date
IL293599A IL293599A (en) 2019-12-05 2020-12-07 Retromer stabilization for the treatment of Alzheimer's disease and other neurodegenerative disorders

Country Status (12)

Country Link
US (1) US20230021959A1 (https=)
EP (1) EP4069315A4 (https=)
JP (2) JP2023505271A (https=)
KR (1) KR20220152192A (https=)
CN (2) CN115297891A (https=)
AU (1) AU2020395327A1 (https=)
BR (1) BR112022011073A2 (https=)
CA (1) CA3160785A1 (https=)
IL (1) IL293599A (https=)
MX (1) MX2022006895A (https=)
PH (1) PH12022551373A1 (https=)
WO (1) WO2021113824A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023159238A1 (en) * 2022-02-21 2023-08-24 The Scripps Research Institute Mef2 transcriptional activators to treat neurologic conditions

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1938104A2 (en) * 2005-10-17 2008-07-02 Institute for Systems Biology Tissue-and serum-derived glycoproteins and methods of their use
US20080214482A1 (en) * 2005-11-15 2008-09-04 Scott Small Retromer-based assays and methods for treating alzheimer's disease
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP2607900A1 (de) * 2011-12-22 2013-06-26 Protagen AG Markersequenzen für Brustkrebs und deren Verwendung
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
WO2014110481A2 (en) * 2013-01-11 2014-07-17 The Trustees Of Columbia University In The City Of New York Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson' s disease risk
US20160250182A1 (en) * 2013-01-11 2016-09-01 The Trustees Of Columbia University In The City Of Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk
GB201409519D0 (en) * 2014-05-29 2014-07-16 Univ Leicester Senescent cell biomarkers
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
WO2018112446A2 (en) * 2016-12-18 2018-06-21 Selonterra, Inc. Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease

Also Published As

Publication number Publication date
CA3160785A1 (en) 2021-06-10
AU2020395327A1 (en) 2022-07-21
BR112022011073A2 (pt) 2022-09-20
MX2022006895A (es) 2022-11-09
CN120114618A (zh) 2025-06-10
WO2021113824A1 (en) 2021-06-10
EP4069315A1 (en) 2022-10-12
KR20220152192A (ko) 2022-11-15
US20230021959A1 (en) 2023-01-26
JP2025149978A (ja) 2025-10-08
PH12022551373A1 (en) 2023-05-08
EP4069315A4 (en) 2024-03-20
CN115297891A (zh) 2022-11-04
JP2023505271A (ja) 2023-02-08

Similar Documents

Publication Publication Date Title
ES2768763T3 (es) Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
CA2939950C (en) Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
US20230104357A1 (en) Combination of Retromer Pharmacological Chaperones and Exogenous Retromer for the Treatment of Alzheimer's Disease and Other Neurodegenerative Diseases and Disorders
AU2021236481A1 (en) Evasion of neutralizing antibodies by a recombinant adeno-associated virus
US11739346B2 (en) Minigene therapy
US20230174994A1 (en) Engineered parkin and uses thereof
US20210371480A1 (en) Compositions and methods for treating age-related macular degeneration and other diseases
JP2025149978A (ja) アルツハイマー病および他の神経変性障害の処置のためのレトロマーの安定化
JP7253274B2 (ja) Ａａｖ適合性ラミニン－リンカー重合タンパク質
US20230151390A1 (en) Vectors for the treatment of acid ceramidase deficiency
US20250186621A1 (en) Materials and methods for the treatment of eif2b5 mutations and diseases resulting therefrom
US20260022402A1 (en) Recombinant adeno-associated viral vector for gene delivery
WO2024259064A1 (en) Materials and methods for the treatment of neurofibromin 1 mutations and diseases resulting therefrom
HK40077196A (en) Evasion of neutralizing antibodies by a recombinant adeno-associated virus
CN121195072A (zh) 用于帕金森病的基因治疗载体
EP4565705A2 (en) Compositions and methods for modifying muller glial cells