BR112022001657A2 - 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 - Google Patents

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

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Publication number
BR112022001657A2
BR112022001657A2 BR112022001657A BR112022001657A BR112022001657A2 BR 112022001657 A2 BR112022001657 A2 BR 112022001657A2 BR 112022001657 A BR112022001657 A BR 112022001657A BR 112022001657 A BR112022001657 A BR 112022001657A BR 112022001657 A2 BR112022001657 A2 BR 112022001657A2
Authority
BR
Brazil
Prior art keywords
ataxia
friedreich
treating
fusion polypeptide
methods
Prior art date
Application number
BR112022001657A
Other languages
English (en)
Inventor
Mark Payne Ronald
Original Assignee
Univ Indiana Trustees
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 Indiana Trustees filed Critical Univ Indiana Trustees
Publication of BR112022001657A2 publication Critical patent/BR112022001657A2/pt

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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)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Psychiatry (AREA)
  • Toxicology (AREA)
  • Hospice & Palliative Care (AREA)
  • Diabetes (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)

Abstract

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. um polipeptídeo de fusão tat-fxn útil no tratamento de indivíduos diagnosticados com ataxia de friedreich, cardiomiopatia hipertrófica ou ambos é descrito, assim como métodos de tratamento e composições farmacêuticas relacionados.
BR112022001657A 2019-07-29 2020-07-29 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 BR112022001657A2 (pt)

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
BR112022001657A2 true BR112022001657A2 (pt) 2022-03-22

Family

ID=72087246

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022001657A BR112022001657A2 (pt) 2019-07-29 2020-07-29 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

Country Status (8)

Country Link
US (2) US11459363B2 (pt)
EP (1) EP4004022A1 (pt)
CN (1) CN114450309A (pt)
AU (1) AU2020321901A1 (pt)
BR (1) BR112022001657A2 (pt)
CA (1) CA3149188A1 (pt)
IL (1) IL290132A (pt)
WO (1) WO2021021931A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4065984A1 (en) * 2019-11-25 2022-10-05 Larimar Therapeutics, Inc. Methods for quantifying frataxin activity
US11891420B2 (en) * 2020-03-26 2024-02-06 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
EP4142768A1 (en) 2020-04-30 2023-03-08 Larimar Therapeutics, Inc. Methods for treating myelin associated diseases and mitochondria associated diseases
EP4259183A1 (en) * 2020-12-12 2023-10-18 Larimar Therapeutics, Inc. Pharmaceutical composition comprising frataxin fusion protein and methods of use thereof
WO2023196937A1 (en) 2022-04-06 2023-10-12 Larimar Therapeutics, Inc. Frataxin-sensitive markers for monitoring frataxin replacement therapy
WO2023220657A1 (en) 2022-05-10 2023-11-16 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

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2598525B1 (en) 2010-07-28 2015-08-19 Roberto Testi Frataxin mutants
CN103620036B (zh) * 2011-06-09 2016-12-21 库尔纳公司 通过抑制共济蛋白(fxn)的天然反义转录物而治疗fxn 相关疾病
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
WO2013071440A1 (en) * 2011-11-18 2013-05-23 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
EP3501532A3 (en) 2014-05-28 2019-07-17 Stealth BioTherapeutics Corp 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
WO2016172659A1 (en) * 2015-04-24 2016-10-27 University Of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia
EP3963099A1 (en) * 2019-04-30 2022-03-09 Larimar Therapeutics, Inc. Frataxin-sensitive markers for determining effectiveness of frataxin replacement therapy
US20220378869A1 (en) * 2019-07-18 2022-12-01 Larimar Therapeutics, Inc. Use of frataxin for treating leigh syndrome, french canadian type
US11891420B2 (en) * 2020-03-26 2024-02-06 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
EP4142768A1 (en) * 2020-04-30 2023-03-08 Larimar Therapeutics, Inc. Methods for treating myelin associated diseases and mitochondria associated diseases

Also Published As

Publication number Publication date
AU2020321901A1 (en) 2022-02-24
US11459363B2 (en) 2022-10-04
WO2021021931A1 (en) 2021-02-04
US20230242600A1 (en) 2023-08-03
CA3149188A1 (en) 2021-02-04
CN114450309A (zh) 2022-05-06
US20210047378A1 (en) 2021-02-18
IL290132A (en) 2022-03-01
EP4004022A1 (en) 2022-06-01

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