CA3082907A1 - Compositions and methods for neurological diseases - Google Patents

Compositions and methods for neurological diseases Download PDF

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Publication number
CA3082907A1
CA3082907A1 CA3082907A CA3082907A CA3082907A1 CA 3082907 A1 CA3082907 A1 CA 3082907A1 CA 3082907 A CA3082907 A CA 3082907A CA 3082907 A CA3082907 A CA 3082907A CA 3082907 A1 CA3082907 A1 CA 3082907A1
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CA
Canada
Prior art keywords
receptor
engineered
vector
seq
pain
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3082907A
Other languages
English (en)
French (fr)
Inventor
Anthony Lau
Kenneth P. Greenberg
Orion KEIFER, Jr.
Stefanie MAKINSON
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Trames Bio Inc
Original Assignee
Coda Biotherapeutics Inc
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 Coda Biotherapeutics Inc filed Critical Coda Biotherapeutics Inc
Publication of CA3082907A1 publication Critical patent/CA3082907A1/en
Pending legal-status Critical Current

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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Immunology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3082907A 2017-11-27 2018-11-27 Compositions and methods for neurological diseases Pending CA3082907A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762590911P 2017-11-27 2017-11-27
US62/590,911 2017-11-27
US201862659911P 2018-04-19 2018-04-19
US62/659,911 2018-04-19
PCT/US2018/062575 WO2019104307A1 (en) 2017-11-27 2018-11-27 Compositions and methods for neurological diseases

Publications (1)

Publication Number Publication Date
CA3082907A1 true CA3082907A1 (en) 2019-05-31

Family

ID=66632192

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3082907A Pending CA3082907A1 (en) 2017-11-27 2018-11-27 Compositions and methods for neurological diseases

Country Status (12)

Country Link
US (2) US10538571B2 (https=)
EP (1) EP3716990A4 (https=)
JP (2) JP2021503960A (https=)
KR (2) KR20200112811A (https=)
CN (1) CN111741760A (https=)
AU (2) AU2018373279B2 (https=)
CA (1) CA3082907A1 (https=)
IL (2) IL274702B2 (https=)
MX (2) MX2020005470A (https=)
SG (1) SG11202004488TA (https=)
WO (1) WO2019104307A1 (https=)
ZA (1) ZA202408862B (https=)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12384797B2 (en) 2019-02-13 2025-08-12 Ptc Therapeutics, Inc. Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia
US12398140B2 (en) 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2020334147A1 (en) * 2019-08-21 2022-03-31 Trames Bio, Inc. Compositions and methods for neurological diseases
WO2021168110A1 (en) * 2020-02-20 2021-08-26 The Regents Of The University Of California Therapies for the treatment of diseases and disorders associated with abnormal expression of a neural-associated gene
IL300816A (en) * 2020-08-21 2023-04-01 Trames Bio Inc Preparations and methods for neurological diseases
WO2022067044A1 (en) * 2020-09-25 2022-03-31 The Regents Of The University Of California Compositions and methods for ameliorating medical conditions
MX2023008738A (es) * 2021-01-25 2023-09-12 Trames Bio Inc Cápsides de virus adenoasociados y canales iónicos controlados por ligandos diseñados para el tratamiento contra epilepsia focal y dolor neuropático.
US11969400B2 (en) * 2021-03-23 2024-04-30 Kumara V. Nibhanipudi Ibuprofen for symptomatic treatment of diarrheas in HIV patients
WO2023278295A1 (en) 2021-06-29 2023-01-05 The Broad Institute, Inc. Compositions and methods for ameliorating anterodorsal thalamus hyperexcitability
CN113730809B (zh) * 2021-10-09 2024-08-16 科悦医疗(苏州)有限公司 一种非侵入式迷走神经联合骶神经刺激装置
EP4473002A4 (en) * 2022-01-31 2026-04-08 Hughes Howard Med Inst Modified ion channels sensitive to a ligand and methods of use
CN119137144A (zh) * 2022-02-18 2024-12-13 特拉梅斯生物股份有限公司 用于神经系统疾病的组合物和方法
KR20240161957A (ko) * 2022-02-18 2024-11-13 트라메스 바이오 인코포레이티드 리간드 개폐 이온 통로 및 사용 방법
WO2025015092A1 (en) * 2023-07-10 2025-01-16 Kriya Therapeutics, Inc. Novel cam-kinase promoter and uses thereof for treating nervous system disorders
CN121866054A (zh) * 2023-08-16 2026-04-14 拜尔吉生物股份有限公司 用于治疗癫痫发作的方法
GB202401492D0 (en) 2024-02-05 2024-03-20 Ucl Business Ltd Chloride channels and uses thereof

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DE69434067T2 (de) * 1993-03-08 2006-03-02 Merck & Co., Inc. Menschliche neuronale nikotin-acetylcholin-rezeptor-verbindungen und methoden ihres einsatzes
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
EP0893950A4 (en) 1996-03-26 2002-02-27 Univ California SELECTIVE TARGET CELL ACTIVATION BY EXPRESSION OF A RECEPTOR SPECIFICALLY ACTIVATED BY SYNTHETIC LIGANDS
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
AU8672198A (en) 1997-07-31 1999-02-22 Chiron Corporation Method enabling readministration of aav vector via immunosuppression of host
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
AU4980200A (en) 1999-05-27 2000-12-18 Pharmacia & Upjohn Company Methods and compositions for measuring ion channel conductance
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
JP2003523320A (ja) 1999-09-29 2003-08-05 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ウイルスベクターの迅速なｐｅｇ改変方法、高められた遺伝子形質導入のための組成物、高められた物理的安定性を伴う組成物、およびそのための用途
EP1280902A2 (en) 2000-04-29 2003-02-05 Millennium Pharmaceuticals, Inc. 23949 and 32391, human ion channels and uses thereof
EP1532452A4 (en) 2002-05-31 2006-11-29 Sloan Kettering Inst Cancer HETEROLOGY STIMULUS-CONTROLLED ION CHANNELS AND METHOD FOR USE THEREOF
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
WO2008143875A1 (en) 2007-05-14 2008-11-27 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Targeted delivery of glycine receptors to excitable cells
EP2344636B1 (en) * 2008-10-09 2017-12-06 Howard Hughes Medical Institute Novel chimeric ligand-gated ion channels and methods of use thereof
WO2014093251A1 (en) * 2012-12-10 2014-06-19 University Of Pittsburgh Of The Commonwealth System Of Higher Education Engineered receptors and their use
GB201404470D0 (en) 2014-03-13 2014-04-30 Ucl Business Plc Therapeutic methods and materials
US10682424B2 (en) 2015-03-31 2020-06-16 New York University Compositions and method for reducing seizures
AU2016324317A1 (en) 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
IL304040B2 (en) 2016-07-07 2024-09-01 Hughes Howard Med Inst Modified ligand-gated ion channels and methods of use
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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12384797B2 (en) 2019-02-13 2025-08-12 Ptc Therapeutics, Inc. Thioeno[3,2-b] pyridin-7-amine compounds for treating familial dysautonomia
US12398140B2 (en) 2019-02-13 2025-08-26 Ptc Therapeutics, Inc. Substituted pyrrolo [2,3-d]pyrimidines for treating familial dysautonomia

Also Published As

Publication number Publication date
SG11202004488TA (en) 2020-06-29
IL274702B2 (en) 2026-04-01
WO2019104307A1 (en) 2019-05-31
EP3716990A1 (en) 2020-10-07
KR20260030128A (ko) 2026-03-05
JP2024160218A (ja) 2024-11-13
AU2018373279B2 (en) 2024-07-25
AU2024227560A1 (en) 2024-11-14
IL324810A (en) 2026-01-01
US20210061873A1 (en) 2021-03-04
JP2021503960A (ja) 2021-02-15
IL274702B1 (en) 2025-12-01
EP3716990A4 (en) 2021-12-08
US20190161529A1 (en) 2019-05-30
MX2020005470A (es) 2020-11-09
KR20200112811A (ko) 2020-10-05
IL274702A (en) 2020-06-30
AU2018373279A1 (en) 2020-06-04
ZA202408862B (en) 2026-01-28
US10538571B2 (en) 2020-01-21
MX2024014448A (es) 2024-12-06
CN111741760A (zh) 2020-10-02

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