DK4004022T3 - Materialer og fremgangsmåder til behandling af friedreichs ataksi - Google Patents

Materialer og fremgangsmåder til behandling af friedreichs ataksi

Info

Publication number
DK4004022T3
DK4004022T3 DK20757116.7T DK20757116T DK4004022T3 DK 4004022 T3 DK4004022 T3 DK 4004022T3 DK 20757116 T DK20757116 T DK 20757116T DK 4004022 T3 DK4004022 T3 DK 4004022T3
Authority
DK
Denmark
Prior art keywords
ataxysia
materials
methods
treating friedreich
friedreich
Prior art date
Application number
DK20757116.7T
Other languages
English (en)
Inventor
Ronald Mark Payne
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
Application granted granted Critical
Publication of DK4004022T3 publication Critical patent/DK4004022T3/da

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)
DK20757116.7T 2019-07-29 2020-07-29 Materialer og fremgangsmåder til behandling af friedreichs ataksi DK4004022T3 (da)

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
DK4004022T3 true DK4004022T3 (da) 2026-04-07

Family

ID=72087246

Family Applications (1)

Application Number Title Priority Date Filing Date
DK20757116.7T DK4004022T3 (da) 2019-07-29 2020-07-29 Materialer og fremgangsmåder til behandling af friedreichs ataksi

Country Status (14)

Country Link
US (4) US11459363B2 (da)
EP (2) EP4004022B1 (da)
CN (1) CN114450309A (da)
AU (1) AU2020321901A1 (da)
BR (1) BR112022001657A2 (da)
CA (1) CA3149188A1 (da)
DK (1) DK4004022T3 (da)
FI (1) FI4004022T3 (da)
IL (1) IL290132A (da)
LT (1) LT4004022T (da)
PT (1) PT4004022T (da)
RS (1) RS67940B1 (da)
SM (1) SMT202600182T1 (da)
WO (1) WO2021021931A1 (da)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11459363B2 (en) 2019-07-29 2022-10-04 The Trustees Of Indiana University Materials and methods for treating Friedreich's Ataxia
WO2021021964A2 (en) 2019-07-29 2021-02-04 Chondrial 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
EP4126228A2 (en) 2020-03-26 2023-02-08 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
WO2021222865A1 (en) 2020-04-30 2021-11-04 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
CN119365780A (zh) 2022-04-06 2025-01-24 拉利玛生物医药公司 用于监测共济蛋白替代疗法的共济蛋白敏感性标志物
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

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3012267A1 (en) 2010-07-28 2016-04-27 Roberto Testi Frataxin mutants
RU2620980C2 (ru) * 2011-06-09 2017-05-30 Курна, Инк. Лечение заболеваний, связанных с фратаксином (fxn), путем ингибирования природного антисмыслового транскрипта fxn
WO2012174452A1 (en) * 2011-06-17 2012-12-20 Shire Human Genetic Therapies, Inc. Mitochondrial targeting and therapeutic use thereof
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
US10617770B2 (en) * 2015-04-24 2020-04-14 University Of Florida Research Foundation, Incorporated AAV vector for treatment of Friedreich's ataxia
SG11202111824UA (en) * 2019-04-30 2021-11-29 Larimar Therapeutics Inc Frataxin-sensitive markers for determining effectiveness of frataxin replacement therapy
WO2021011929A1 (en) * 2019-07-18 2021-01-21 Chondrial Therapeutics, Inc. Use of frataxin for treating leigh syndrome, french canadian type
US11459363B2 (en) 2019-07-29 2022-10-04 The Trustees Of Indiana University Materials and methods for treating Friedreich's Ataxia
EP4126228A2 (en) * 2020-03-26 2023-02-08 Larimar Therapeutics, Inc. Molecules for organelle-specific protein delivery
WO2021222865A1 (en) * 2020-04-30 2021-11-04 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
PT4004022T (pt) 2026-04-10
US20260055149A1 (en) 2026-02-26
US12180253B2 (en) 2024-12-31
WO2021021931A1 (en) 2021-02-04
EP4729548A2 (en) 2026-04-22
US20230242600A1 (en) 2023-08-03
EP4004022A1 (en) 2022-06-01
LT4004022T (lt) 2026-04-27
SMT202600182T1 (it) 2026-05-12
EP4004022B1 (en) 2025-12-31
CN114450309A (zh) 2022-05-06
US11459363B2 (en) 2022-10-04
US20240391966A1 (en) 2024-11-28
FI4004022T3 (fi) 2026-03-30
BR112022001657A2 (pt) 2022-03-22
CA3149188A1 (en) 2021-02-04
US20210047378A1 (en) 2021-02-18
IL290132A (en) 2022-03-01
RS67940B1 (sr) 2026-04-30

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