CA2962081A1 - Exosomes utiles pour traiter une maladie de stockage lysosomal - Google Patents

Exosomes utiles pour traiter une maladie de stockage lysosomal Download PDF

Info

Publication number
CA2962081A1
CA2962081A1 CA2962081A CA2962081A CA2962081A1 CA 2962081 A1 CA2962081 A1 CA 2962081A1 CA 2962081 A CA2962081 A CA 2962081A CA 2962081 A CA2962081 A CA 2962081A CA 2962081 A1 CA2962081 A1 CA 2962081A1
Authority
CA
Canada
Prior art keywords
protein
exosomes
lysosomal
gaa
seq
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
CA2962081A
Other languages
English (en)
Inventor
Mark TARNOPOLSKY
Adeel SAFDAR
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
EXERKINE Corp
Original Assignee
EXERKINE Corp
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 EXERKINE Corp filed Critical EXERKINE Corp
Publication of CA2962081A1 publication Critical patent/CA2962081A1/fr
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5176—Compounds of unknown constitution, e.g. material from plants or animals
    • 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
    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K17/00—Carrier-bound or immobilised peptides; Preparation thereof
    • C07K17/02—Peptides being immobilised on, or in, an organic carrier
    • C07K17/04—Peptides being immobilised on, or in, an organic carrier entrapped within the carrier, e.g. gel, hollow fibre
    • 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
    • C12N11/00—Carrier-bound or immobilised enzymes; Carrier-bound or immobilised microbial cells; Preparation thereof
    • C12N11/02—Enzymes or microbial cells immobilised on or in an organic carrier
    • C12N11/04—Enzymes or microbial cells immobilised on or in an organic carrier entrapped within the carrier, e.g. gel or hollow fibres
    • 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
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/5005—Wall or coating material
    • A61K9/5063—Compounds of unknown constitution, e.g. material from plants or animals
    • A61K9/5068—Cell membranes or bacterial membranes enclosing drugs

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Organic Chemistry (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Obesity (AREA)
  • Biophysics (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Dispersion Chemistry (AREA)
  • Diabetes (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Botany (AREA)
  • Physics & Mathematics (AREA)
  • Nanotechnology (AREA)
  • Optics & Photonics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
CA2962081A 2014-09-26 2015-09-25 Exosomes utiles pour traiter une maladie de stockage lysosomal Abandoned CA2962081A1 (fr)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201462056125P 2014-09-26 2014-09-26
US62/056,125 2014-09-26
US201462082853P 2014-11-21 2014-11-21
US62/082,853 2014-11-21
US201562112940P 2015-02-06 2015-02-06
US62/112,940 2015-02-06
US201562211312P 2015-08-28 2015-08-28
US201562211204P 2015-08-28 2015-08-28
US62/211,312 2015-08-28
US62/211,204 2015-08-28
PCT/CA2015/050959 WO2016044947A1 (fr) 2014-09-26 2015-09-25 Exosomes utiles pour traiter une maladie de stockage lysosomal

Publications (1)

Publication Number Publication Date
CA2962081A1 true CA2962081A1 (fr) 2016-03-31

Family

ID=55580007

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2962081A Abandoned CA2962081A1 (fr) 2014-09-26 2015-09-25 Exosomes utiles pour traiter une maladie de stockage lysosomal

Country Status (2)

Country Link
CA (1) CA2962081A1 (fr)
WO (1) WO2016044947A1 (fr)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10702581B2 (en) 2015-05-04 2020-07-07 Ilias Biologics Inc. Compositions containing protein loaded exosome and methods for preparing and delivering the same
DK3386534T3 (da) * 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
GB2552460A (en) 2016-07-11 2018-01-31 Evox Therapeutics Ltd CPP-Mediated EV Loading
EP3356522A4 (fr) * 2016-09-30 2019-03-27 Cellex Life Sciences, Incorporated Compositions contenant un exosome chargé d'une protéine, et procédés de préparation et d'administration correspondants
NZ760232A (en) 2017-06-07 2023-05-26 Regeneron Pharma Compositions and methods for internalizing enzymes
EP3648777A4 (fr) * 2017-07-06 2021-04-21 Children's National Medical Center Exosomes et procédés d'utilisation
GB201718681D0 (en) * 2017-11-13 2017-12-27 Evox Therapeutics Ltd Protein engineered extracellular vesicles
GB201810301D0 (en) * 2018-06-22 2018-08-08 Evox Therapeutics Ltd Combinatorial gene therapy
WO2020023594A1 (fr) * 2018-07-24 2020-01-30 Mayo Foundation For Medical Education And Research Compositions et procédés impliquant la transformation de vésicules extracellulaires
KR102233530B1 (ko) * 2018-11-22 2021-03-31 (주)프로스테믹스 엑소좀 및 이의 다양한 용도
WO2020152298A1 (fr) 2019-01-24 2020-07-30 Fundació Hospital Universitari Vall D'hebron - Institut De Recerca Procédé de production d'enzymes
EP4117628A4 (fr) * 2020-03-10 2024-04-10 University of Cincinnati Matériels et méthodes pour le traitement de la maladie de gaucher

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001236713A1 (en) * 2000-02-17 2001-08-27 Genzyme Corporation Methods for treatment of lysosomal storage diseases
US9085778B2 (en) * 2006-05-03 2015-07-21 VL27, Inc. Exosome transfer of nucleic acids to cells
JP5713325B2 (ja) * 2009-04-17 2015-05-07 アイシス イノヴェイション リミテッド 遺伝物質の送達用の組成物

Also Published As

Publication number Publication date
WO2016044947A1 (fr) 2016-03-31

Similar Documents

Publication Publication Date Title
CA2962081A1 (fr) Exosomes utiles pour traiter une maladie de stockage lysosomal
Solomon et al. Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives
TWI701332B (zh) 用於治療溶酶體貯積病之腺相關病毒載體
EP1713904B1 (fr) Production et purification d'arylsulfatase a recombinante
EP3519569B1 (fr) Vecteurs viraux recombinés adéno-associés pour le traitement de la mucopolysaccharidose
US12116577B2 (en) Codon-optimized human NPC1 genes for the treatment of Niemann-Pick type C1 deficiency and related conditions
EP2561069B1 (fr) Enzyme des maladies lysosomales
EP2595650B1 (fr) Procédé de fabrication de n-acetylgalactosamine-6-sulfatase hautement phosphorylé active humain, et son utilisation
TWI482625B (zh) 重組人溶酶體酸性脂肪酶（ｌａｌ）於治療人病患之溶酶體酸性脂肪酶缺乏症之用途
IL149029A (en) High mannose proteins and methods for producing the same
WO2017147720A1 (fr) Méthode de traitement d'un trouble du système nerveux central
US20230332112A1 (en) Novel In Vitro and In Vivo Enrichment Strategy Targeting Lymphocytes Derived from Vector Transduced HSCS for Therapy of Disorders
EP1740204A1 (fr) Utilisation medicale de l'alpha-mannosidase
WO2003068255A1 (fr) Compositions et methodes visant a ameliorer le traitement de substitution enzymatique de maladies lysosomales
US20220184185A1 (en) Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
WO2020152298A1 (fr) Procédé de production d'enzymes
JP2004512313A (ja) 血友病ｂを治療する遺伝子薬及びその調剤する方法
Matassini et al. Carbohydrate‐Based Therapeutics for Lysosomal Storage Disorders
Roy Cell disorders in lysosomal storage diseases
Borgida Lysosomal Storage Diseases: How Gene-Targeted Therapies are Changing What is Treatable
Ashiri Using an engineered human hexosaminidase as an enzyme replacement therapy to treat a mouse model of Tay-Sachs Disease
Massaro Intravenously administered gene therapy for neuronopathic Gaucher disease
Priyadharshini et al. Drug delivery systems in Krabbe disease—present and prospective approaches
AU2023212478A1 (en) THERAPEUTIC mRNA
AU2014204569B9 (en) High mannose proteins and methods of making high mannose proteins

Legal Events

Date Code Title Description
FZDE Dead

Effective date: 20180925