AU2020346914A1 - Compositions and methods for treatment of Friedreich's Ataxia - Google Patents

Compositions and methods for treatment of Friedreich's Ataxia Download PDF

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Publication number
AU2020346914A1
AU2020346914A1 AU2020346914A AU2020346914A AU2020346914A1 AU 2020346914 A1 AU2020346914 A1 AU 2020346914A1 AU 2020346914 A AU2020346914 A AU 2020346914A AU 2020346914 A AU2020346914 A AU 2020346914A AU 2020346914 A1 AU2020346914 A1 AU 2020346914A1
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Australia
Prior art keywords
fxn
nucleic acid
seq
human
promoter
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Pending
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AU2020346914A
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English (en)
Inventor
Darin Falk
Edgardo Rodriguez
Madhurima SAHA
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Prevail Therapeutics Inc
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Prevail Therapeutics Inc
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Publication date
Application filed by Prevail Therapeutics Inc filed Critical Prevail Therapeutics Inc
Publication of AU2020346914A1 publication Critical patent/AU2020346914A1/en
Assigned to PREVAIL THERAPEUTICS, INC. reassignment PREVAIL THERAPEUTICS, INC. Request for Assignment Assignors: LACERTA THERAPEUTICS, INC
Pending legal-status Critical Current

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Neurosurgery (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AU2020346914A 2019-09-13 2020-09-11 Compositions and methods for treatment of Friedreich's Ataxia Pending AU2020346914A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962899953P 2019-09-13 2019-09-13
US62/899,953 2019-09-13
PCT/US2020/050551 WO2021050991A1 (en) 2019-09-13 2020-09-11 Compositions and methods for treatment of friedreich's ataxia

Publications (1)

Publication Number Publication Date
AU2020346914A1 true AU2020346914A1 (en) 2022-04-21

Family

ID=74865919

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020346914A Pending AU2020346914A1 (en) 2019-09-13 2020-09-11 Compositions and methods for treatment of Friedreich's Ataxia

Country Status (6)

Country Link
US (1) US20220211737A1 (https=)
EP (1) EP4028027A4 (https=)
JP (1) JP2022548270A (https=)
AU (1) AU2020346914A1 (https=)
CA (1) CA3151073A1 (https=)
WO (1) WO2021050991A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4426845A4 (en) * 2021-11-01 2025-10-01 Univ Florida AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH ATAXIA
WO2024097772A1 (en) * 2022-11-01 2024-05-10 Lacerta Therapeutics, Inc. Compositions and methods for treatment of friedreich's ataxia
WO2025157942A2 (en) * 2024-01-23 2025-07-31 Icm (Institut Du Cerveau Et De La Moelle Épinière) Encapsulated engineered cells for treating a disease or condition affecting the nervous system

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3245221A4 (en) * 2015-01-16 2018-06-13 Voyager Therapeutics, Inc. Central nervous system targeting polynucleotides
WO2016172659A1 (en) * 2015-04-24 2016-10-27 University Of Florida Research Foundation, Inc. Aav vector for treatment of friedreich's ataxia
WO2017075338A2 (en) * 2015-10-29 2017-05-04 Voyager Therapeutics, Inc. Delivery of central nervous system targeting polynucleotides
RU2021102893A (ru) * 2015-11-05 2021-03-03 Бамбу Терапьютикс, Инк. Модифицированные гены атаксии фридрейха и векторы для генной терапии
CN110139933B (zh) * 2016-11-09 2024-03-08 英特瑞克斯顿股份有限公司 共济蛋白表达构建体
EP3641834B1 (en) * 2017-06-19 2023-10-04 Translate Bio, Inc. Messenger rna therapy for the treatment of friedreich's ataxia
WO2019000093A1 (en) * 2017-06-29 2019-01-03 UNIVERSITé LAVAL PLATINUM TALE AND USE THEREOF TO INCREASE THE EXPRESSION OF FRATAXIN
WO2019076973A1 (en) * 2017-10-17 2019-04-25 Fundació Institut D'investigació En Ciències De La Salut Germans Trias I Pujol VECTORS FOR THE TREATMENT OF FRIEDREICH ATAXIA

Also Published As

Publication number Publication date
JP2022548270A (ja) 2022-11-17
US20220211737A1 (en) 2022-07-07
EP4028027A1 (en) 2022-07-20
WO2021050991A1 (en) 2021-03-18
EP4028027A4 (en) 2023-11-01
CA3151073A1 (en) 2021-03-18

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Bevan et al. This chapter has been published in the peer-reviewed journal Molecular Therapy. This work is the result of collaboration with the authors listed below. I helped design and perform experiments and write the manuscript, especially all experiments using nonhuman primates.

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PC1 Assignment before grant (sect. 113)

Owner name: PREVAIL THERAPEUTICS, INC.

Free format text: FORMER APPLICANT(S): LACERTA THERAPEUTICS, INC