PL4004022T3 - Substancje i sposoby leczenia ataksji friedreicha - Google Patents

Substancje i sposoby leczenia ataksji friedreicha

Info

Publication number
PL4004022T3
PL4004022T3 PL20757116.7T PL20757116T PL4004022T3 PL 4004022 T3 PL4004022 T3 PL 4004022T3 PL 20757116 T PL20757116 T PL 20757116T PL 4004022 T3 PL4004022 T3 PL 4004022T3
Authority
PL
Poland
Prior art keywords
ataxia
materials
methods
treating friedreich
friedreich
Prior art date
Application number
PL20757116.7T
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 PL4004022T3 publication Critical patent/PL4004022T3/pl

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)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (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)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Pain & Pain Management (AREA)
  • Physics & Mathematics (AREA)
PL20757116.7T 2019-07-29 2020-07-29 Substancje i sposoby leczenia ataksji friedreicha PL4004022T3 (pl)

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
PL4004022T3 true PL4004022T3 (pl) 2026-07-06

Family

ID=72087246

Family Applications (1)

Application Number Title Priority Date Filing Date
PL20757116.7T PL4004022T3 (pl) 2019-07-29 2020-07-29 Substancje i sposoby leczenia ataksji friedreicha

Country Status (18)

Country Link
US (4) US11459363B2 (pl)
EP (2) EP4729548A3 (pl)
CN (1) CN114450309A (pl)
AU (2) AU2020321901B2 (pl)
BR (1) BR112022001657A2 (pl)
CA (1) CA3149188A1 (pl)
DK (1) DK4004022T3 (pl)
ES (1) ES3066806T3 (pl)
FI (1) FI4004022T3 (pl)
HR (1) HRP20260453T1 (pl)
IL (1) IL290132A (pl)
LT (1) LT4004022T (pl)
PL (1) PL4004022T3 (pl)
PT (1) PT4004022T (pl)
RS (1) RS67940B1 (pl)
SI (1) SI4004022T1 (pl)
SM (1) SMT202600182T1 (pl)
WO (1) WO2021021931A1 (pl)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK4004022T3 (da) 2019-07-29 2026-04-07 Univ Indiana Trustees Materialer og fremgangsmåder til behandling af friedreichs ataksi
US12566178B2 (en) 2019-07-29 2026-03-03 Larimar Therapeutics, Inc. Methods of quantifying frataxin and frataxin fusion proteins
US11988675B2 (en) 2019-11-25 2024-05-21 Larimar Therapeutics, Inc. Methods for quantifying frataxin activity
WO2021195597A2 (en) * 2020-03-26 2021-09-30 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
BR112022021823A2 (pt) 2020-04-30 2023-01-17 Larimar Therapeutics Inc Métodos para tratar doenças associadas à mielina e doenças associadas à mitocôndria
US12390509B2 (en) 2020-12-12 2025-08-19 Larimar Therapeutics, Inc. Pharmaceutical composition comprising frataxin fusion protein and methods of use thereof
CA3247402A1 (en) 2022-04-06 2023-10-12 Larimar Therapeutics, Inc. FRATAXIN-SENSITIVE MARKERS FOR MONITORING FRATAXIN REPLACEMENT THERAPY
AU2023269794A1 (en) 2022-05-10 2024-11-21 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
US20250276037A1 (en) * 2024-02-12 2025-09-04 Larimar Therapeutics, Inc. Methods of treating friedreich's ataxia
WO2026136463A1 (en) * 2024-12-16 2026-06-25 Larimar Therapeutics, Inc. Tat-fxn fusion protein for use in the treatment of friedreich's ataxia

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2598525E (pt) 2010-07-28 2015-11-02 Testi Roberto Mutantes de frataxina
WO2012170771A1 (en) * 2011-06-09 2012-12-13 Curna, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
WO2012174452A1 (en) * 2011-06-17 2012-12-20 Shire Human Genetic Therapies, Inc. Mitochondrial targeting and therapeutic use thereof
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
US10576124B2 (en) 2014-05-28 2020-03-03 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
EP3286321A4 (en) * 2015-04-24 2018-10-17 University of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia
CA3138566A1 (en) * 2019-04-30 2020-11-05 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
DK4004022T3 (da) 2019-07-29 2026-04-07 Univ Indiana Trustees Materialer og fremgangsmåder til behandling af friedreichs ataksi
WO2021195597A2 (en) * 2020-03-26 2021-09-30 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
BR112022021823A2 (pt) * 2020-04-30 2023-01-17 Larimar Therapeutics Inc Métodos para tratar doenças associadas à mielina e doenças associadas à mitocôndria

Also Published As

Publication number Publication date
US20260055149A1 (en) 2026-02-26
AU2026205535A1 (en) 2026-07-30
ES3066806T3 (en) 2026-05-18
US20240391966A1 (en) 2024-11-28
HRP20260453T1 (hr) 2026-06-05
US12180253B2 (en) 2024-12-31
BR112022001657A2 (pt) 2022-03-22
EP4729548A3 (en) 2026-06-24
US20230242600A1 (en) 2023-08-03
CN114450309A (zh) 2022-05-06
SI4004022T1 (sl) 2026-06-30
SMT202600182T1 (it) 2026-05-12
LT4004022T (lt) 2026-04-27
AU2020321901B2 (en) 2026-07-16
FI4004022T3 (fi) 2026-03-30
EP4004022B1 (en) 2025-12-31
EP4729548A2 (en) 2026-04-22
PT4004022T (pt) 2026-04-10
RS67940B1 (sr) 2026-04-30
IL290132A (en) 2022-03-01
CA3149188A1 (en) 2021-02-04
US11459363B2 (en) 2022-10-04
US20210047378A1 (en) 2021-02-18
EP4004022A1 (en) 2022-06-01
WO2021021931A1 (en) 2021-02-04
DK4004022T3 (da) 2026-04-07
AU2020321901A1 (en) 2022-02-24