LT4004022T - Medžiagos ir metodai, skirti fridraicho ataksijos gydymui - Google Patents

Medžiagos ir metodai, skirti fridraicho ataksijos gydymui

Info

Publication number
LT4004022T
LT4004022T LTEPPCT/US2020/044069T LTUS2020044069T LT4004022T LT 4004022 T LT4004022 T LT 4004022T LT US2020044069 T LTUS2020044069 T LT US2020044069T LT 4004022 T LT4004022 T LT 4004022T
Authority
LT
Lithuania
Prior art keywords
ataxia
materials
methods
treating friedreich
friedreich
Prior art date
Application number
LTEPPCT/US2020/044069T
Other languages
English (en)
Inventor
Ronald Mark PAYNE
Original Assignee
The Trustees Of Indiana 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 The Trustees Of Indiana University filed Critical The Trustees Of Indiana University
Publication of LT4004022T publication Critical patent/LT4004022T/lt

Links

Classifications

    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/02—Non-specific cardiovascular stimulants, e.g. drugs for syncope, antihypotensives
    • 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
    • C12N15/70—Vectors or expression systems specially adapted for E. coli
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/07—Fusion polypeptide containing a localisation/targetting motif containing a mitochondrial localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Plant Pathology (AREA)
  • Pulmonology (AREA)
LTEPPCT/US2020/044069T 2019-07-29 2020-07-29 Medžiagos ir metodai, skirti fridraicho ataksijos gydymui LT4004022T (lt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962880073P 2019-07-29 2019-07-29
US201962891029P 2019-08-23 2019-08-23
PCT/US2020/044069 WO2021021931A1 (en) 2019-07-29 2020-07-29 Materials and methods for treating friedreich's ataxia

Publications (1)

Publication Number Publication Date
LT4004022T true LT4004022T (lt) 2026-04-27

Family

ID=72087246

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEPPCT/US2020/044069T LT4004022T (lt) 2019-07-29 2020-07-29 Medžiagos ir metodai, skirti fridraicho ataksijos gydymui

Country Status (11)

Country Link
US (4) US11459363B2 (lt)
EP (2) EP4004022B1 (lt)
CN (1) CN114450309A (lt)
AU (1) AU2020321901A1 (lt)
BR (1) BR112022001657A2 (lt)
CA (1) CA3149188A1 (lt)
DK (1) DK4004022T3 (lt)
FI (1) FI4004022T3 (lt)
IL (1) IL290132A (lt)
LT (1) LT4004022T (lt)
WO (1) WO2021021931A1 (lt)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021021964A2 (en) 2019-07-29 2021-02-04 Chondrial Therapeutics, Inc. Methods of quantifying frataxin and frataxin fusion proteins
BR112022001657A2 (pt) 2019-07-29 2022-03-22 Univ Indiana Trustees Polipeptídeo de fusão, sequência de ácidos nucleicos, cassete de expressão, vetor de expressão, célula hospedeira, composição farmacêutica, e, métodos para tratar a ataxia de friedreich e para tratar uma doença associada à ataxia de friedreich
KR20220108094A (ko) * 2019-11-25 2022-08-02 라리마 테라퓨틱스, 인코포레이티드 프라탁신 활성의 정량화 방법
US11891420B2 (en) 2020-03-26 2024-02-06 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
US11976100B2 (en) 2020-04-30 2024-05-07 Larimar Therapeutics, Inc. Methods for treating myelin associated diseases and mitochondria associated diseases
CN116867506A (zh) * 2020-12-12 2023-10-10 拉利玛生物医药公司 包括弗里德赖希共济失调蛋白融合蛋白的药物组合物及其使用方法
US20230357851A1 (en) 2022-04-06 2023-11-09 Larimar Therapeutics, Inc. Frataxin-sensitive markers for monitoring frataxin-replacement therapy
US20240142476A1 (en) 2022-05-10 2024-05-02 Larimar Therapeutics, Inc. Frataxin-sensitive lipid markers for monitoring frataxin replacement therapy
WO2023240201A1 (en) 2022-06-08 2023-12-14 Larimar Therapeutics, Inc. Frataxin-sensitive markers for monitoring progression and treatment of leigh syndrome
WO2025174893A1 (en) * 2024-02-12 2025-08-21 Larimar Therapeutics, Inc. Methods of treating friedreich's ataxia

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HUE028111T2 (en) 2010-07-28 2016-11-28 Roberto Testi Frataxin mutants
KR102043422B1 (ko) * 2011-06-09 2019-11-11 큐알엔에이, 인크. 프라탁신 (fxn)에 대한 자연 안티센스 전사체의 저해에 의한 프라탁신 (fxn) 관련된 질환의 치료
EP2750686B1 (en) * 2011-06-17 2017-06-14 Shire Human Genetic Therapies, Inc. Polypeptide comprising frataxin and a c-terminal mitochondria penetrating peptide for use in the treatment of friedreich's ataxia
EP2780376B1 (en) * 2011-11-18 2017-03-08 Université Laval Methods and products for increasing frataxin levels and uses thereof
US8912147B2 (en) * 2013-04-15 2014-12-16 BioBlast Pharma Ltd. Mitochondrial proteins constructs and uses thereof
WO2015183995A2 (en) 2014-05-28 2015-12-03 Stealth Peptides International, Inc. Therapeutic compositions including frataxin, lactoferrin, and mitochondrial energy generating enzymes, and uses thereof
GB201414130D0 (en) * 2014-08-08 2014-09-24 Agency Science Tech & Res Mutants of the bacteriophage lambda integrase
US10617770B2 (en) 2015-04-24 2020-04-14 University Of Florida Research Foundation, Incorporated AAV vector for treatment of Friedreich's ataxia
CN121610569A (zh) * 2019-04-30 2026-03-06 拉利玛生物医药公司 用于测定共济蛋白替代疗法的疗效的共济蛋白敏感性标志物
EP3999102A1 (en) * 2019-07-18 2022-05-25 Larimar Therapeutics, Inc. Use of frataxin for treating leigh syndrome, french canadian type
BR112022001657A2 (pt) 2019-07-29 2022-03-22 Univ Indiana Trustees Polipeptídeo de fusão, sequência de ácidos nucleicos, cassete de expressão, vetor de expressão, célula hospedeira, composição farmacêutica, e, métodos para tratar a ataxia de friedreich e para tratar uma doença associada à ataxia de friedreich
US11891420B2 (en) * 2020-03-26 2024-02-06 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
US11976100B2 (en) * 2020-04-30 2024-05-07 Larimar Therapeutics, Inc. Methods for treating myelin associated diseases and mitochondria associated diseases

Also Published As

Publication number Publication date
DK4004022T3 (da) 2026-04-07
US11459363B2 (en) 2022-10-04
CN114450309A (zh) 2022-05-06
US20230242600A1 (en) 2023-08-03
US20260055149A1 (en) 2026-02-26
US12180253B2 (en) 2024-12-31
WO2021021931A1 (en) 2021-02-04
CA3149188A1 (en) 2021-02-04
BR112022001657A2 (pt) 2022-03-22
EP4004022A1 (en) 2022-06-01
EP4004022B1 (en) 2025-12-31
US20240391966A1 (en) 2024-11-28
IL290132A (en) 2022-03-01
US20210047378A1 (en) 2021-02-18
FI4004022T3 (fi) 2026-03-30
AU2020321901A1 (en) 2022-02-24
EP4729548A2 (en) 2026-04-22

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