CA3127808A1 - Compositions and methods for treating neurocognitive disorders - Google Patents

Compositions and methods for treating neurocognitive disorders Download PDF

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Publication number
CA3127808A1
CA3127808A1 CA3127808A CA3127808A CA3127808A1 CA 3127808 A1 CA3127808 A1 CA 3127808A1 CA 3127808 A CA3127808 A CA 3127808A CA 3127808 A CA3127808 A CA 3127808A CA 3127808 A1 CA3127808 A1 CA 3127808A1
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Canada
Prior art keywords
proteins
hla
cells
patient
pharmaceutical composition
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Pending
Application number
CA3127808A
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English (en)
French (fr)
Inventor
Chris Mason
Oliver Cooper
Robert Plasschaert
Nico Peter Van Til
Mark Deandrade
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Tectonic Therapeutic Inc
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Avrobio Inc
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Publication of CA3127808A1 publication Critical patent/CA3127808A1/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/414—Nervous system antigens
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/31—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterized by the route of administration
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—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
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Reproductive Health (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
CA3127808A 2019-02-01 2020-01-31 Compositions and methods for treating neurocognitive disorders Pending CA3127808A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962800177P 2019-02-01 2019-02-01
US62/800,177 2019-02-01
PCT/US2020/016206 WO2020160468A1 (en) 2019-02-01 2020-01-31 Compositions and methods for treating neurocognitive disorders

Publications (1)

Publication Number Publication Date
CA3127808A1 true CA3127808A1 (en) 2020-08-06

Family

ID=71840202

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3127808A Pending CA3127808A1 (en) 2019-02-01 2020-01-31 Compositions and methods for treating neurocognitive disorders

Country Status (7)

Country Link
US (1) US20220133808A1 (https=)
EP (1) EP3917620A4 (https=)
JP (2) JP2022523514A (https=)
CA (1) CA3127808A1 (https=)
IL (1) IL285270A (https=)
MA (1) MA54880A (https=)
WO (1) WO2020160468A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JOP20190248A1 (ar) 2017-04-21 2019-10-20 Amgen Inc بروتينات ربط مولد ضد trem2 واستخداماته
CN112204050B (zh) 2018-01-31 2025-03-14 艾莱克特有限责任公司 抗ms4a6a抗体及其使用方法
CN111971301B (zh) 2018-01-31 2025-01-07 艾莱克特有限责任公司 抗ms4a4a抗体及其使用方法
EP4037696A4 (en) * 2019-10-01 2024-02-07 Children's Medical Center Corporation Compositions and methods for treating alzheimer's disease
EP4146797A1 (en) * 2020-05-06 2023-03-15 Orchard Therapeutics (Europe) Limited Treatment for neurodegenerative diseases
US20230322897A1 (en) * 2020-07-02 2023-10-12 Avrobio, Inc. Compositions and methods for treating neurocognitive disorders
US20230265456A1 (en) * 2020-08-10 2023-08-24 Fundacion Para La Investigacion Medica Aplicada Gene therapy vector expressing cyp27a1 for the treatment of cerebrotendinous xanthomatosis
US20220127347A1 (en) * 2020-10-26 2022-04-28 The Regents Of The University Of California Inhibition of Tau Propagation
JP2024516548A (ja) 2021-04-08 2024-04-16 ジョスリン ダイアビーティス センター インコーポレイテッド 腎機能低下の診断及び予測の方法
AU2022441199A1 (en) * 2021-09-27 2024-05-16 Curelab Oncology, Inc. Prevention and treatment of diseases by modulating activity of mesenchymal stem cells with p62 (sqstm1)-encoding vector and pharmaceutical formulations containing p62 (sqstm1) proteins
US20250051391A1 (en) * 2021-12-15 2025-02-13 The Governing Council Of The University Of Toronto Peptide inhibitors and methods for inhibiting protein aggregation in neurons and neurodegenerative diseases
WO2024178386A1 (en) * 2023-02-24 2024-08-29 Aarhus Universitet Methods of treating endosomal trafficking diseases
WO2025064926A1 (en) * 2023-09-22 2025-03-27 The Board Of Regents Of The University Of Texas System Novel secretory signal peptides and uses thereof
CN117736321A (zh) * 2023-12-20 2024-03-22 中国人民解放军空军军医大学 一种乙酰化修饰的特异性抗体及其应用
WO2025186293A1 (en) * 2024-03-06 2025-09-12 Institut National de la Santé et de la Recherche Médicale Chmp2b mutants for use for treating infection
KR20260001543A (ko) * 2024-06-25 2026-01-06 연세대학교 산학협력단 바이러스 벡터를 포함한 난청 치료용 조성물

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU4869399A (en) * 1998-07-10 2000-02-01 Curagen Corporation Interaction of human beta amyloid precursor protein (beta-app) with human lon-protease like protein (hslon)
US20050163760A1 (en) * 2001-12-06 2005-07-28 Nathalie Cartier-Lacave Use of cd34+ hematopoietic progenitor cells for the treatment of cns disorders
US20130102538A1 (en) * 2010-05-05 2013-04-25 Amicus Therapeutics, Inc. Method of treating alzheimer's disease using pharmacological chaperones to increase presenilin function and gamma-secretase activity
JP2016537995A (ja) * 2013-11-05 2016-12-08 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル 新しいアルツハイマー病動物モデル
US11066456B2 (en) * 2016-02-25 2021-07-20 Washington University Compositions comprising TREM2 and methods of use thereof
JOP20190248A1 (ar) * 2017-04-21 2019-10-20 Amgen Inc بروتينات ربط مولد ضد trem2 واستخداماته
KR102040364B1 (ko) * 2017-05-26 2019-11-05 가천대학교 산학협력단 알츠하이머병 관련 ３０개 유전자를 이용한 유전적 진단방법
KR20200032722A (ko) * 2017-07-27 2020-03-26 노파르티스 아게 부분절단효소 내성 trem2 변이체

Also Published As

Publication number Publication date
WO2020160468A1 (en) 2020-08-06
MA54880A (fr) 2021-12-08
EP3917620A1 (en) 2021-12-08
IL285270A (en) 2021-09-30
JP2025062601A (ja) 2025-04-14
JP2022523514A (ja) 2022-04-25
EP3917620A4 (en) 2023-02-08
US20220133808A1 (en) 2022-05-05

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