MA40460A - Ciblage lysosomial et utilisation correspondante - Google Patents

Ciblage lysosomial et utilisation correspondante

Info

Publication number
MA40460A
MA40460A MA040460A MA40460A MA40460A MA 40460 A MA40460 A MA 40460A MA 040460 A MA040460 A MA 040460A MA 40460 A MA40460 A MA 40460A MA 40460 A MA40460 A MA 40460A
Authority
MA
Morocco
Prior art keywords
lysosomal targeting
compositions
methods
lysosomal
sort1
Prior art date
Application number
MA040460A
Other languages
English (en)
Inventor
Michael F Concino
Jianwen Feng
Kevin Holmes
Andrea Iskenderian
Muthuraman Meiyappan
Angela W Norton
Jing Pan
Bettina Strack-Logue
Bohong Zhang
Original Assignee
Shire Human Genetic Therapies
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 Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of MA40460A publication Critical patent/MA40460A/fr

Links

Classifications

    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)

Abstract

L'invention concerne des compositions et des procédés de ciblage lysosomial efficace à médiation par sort1. En particulier, les compositions et les procédés fournis par la présente invention peuvent être utilisés pour traiter les maladies lysosomiales telles que le syndrome de sanfilippo de type b
MA040460A 2014-08-11 2015-08-11 Ciblage lysosomial et utilisation correspondante MA40460A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201462036084P 2014-08-11 2014-08-11

Publications (1)

Publication Number Publication Date
MA40460A true MA40460A (fr) 2016-02-18

Family

ID=54011889

Family Applications (1)

Application Number Title Priority Date Filing Date
MA040460A MA40460A (fr) 2014-08-11 2015-08-11 Ciblage lysosomial et utilisation correspondante

Country Status (4)

Country Link
US (1) US10603364B2 (fr)
EP (1) EP3193942B1 (fr)
MA (1) MA40460A (fr)
WO (1) WO2016025523A1 (fr)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT3626257T (pt) 2010-06-25 2021-11-12 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
US20220133863A1 (en) * 2010-06-25 2022-05-05 Shire Human Genetic Therapies, Inc. Treatment of sanfilippo syndrome type b
SI3280441T1 (sl) 2015-04-07 2022-01-31 Alector Llc Protitelesa anti-sortilin in metoda njihove uporabe
US10849992B1 (en) 2015-04-07 2020-12-01 Alector Llc Methods of screening for sortilin binding antagonists
WO2019070891A1 (fr) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomaux
WO2019070894A1 (fr) * 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Thérapies géniques pour troubles lysosomiaux
FI3618928T3 (fi) 2018-07-13 2023-04-21 Alector Llc Anti-sortiliini-vasta-aineita ja menetelmiä niiden käyttämiseksi
JP7571044B2 (ja) 2019-04-10 2024-10-22 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
AU2020361703A1 (en) * 2019-10-10 2022-04-28 Amicus Therapeutics, Inc. Variant IGF2 constructs
CN114555808A (zh) * 2019-10-22 2022-05-27 吉尼松公司 嵌合多肽及其用途
US20240197700A1 (en) * 2021-03-29 2024-06-20 Sanford Health Methods and Compositions for Treating Lysosomal Storage Disorders
EP4363437A1 (fr) * 2021-07-01 2024-05-08 Amicus Therapeutics, Inc. Variants de neurotensine et protéines marquées comprenant un propeptide de neurotensine ou de sortiline
EP4460509A1 (fr) * 2022-01-05 2024-11-13 Helix Nanotechnologies Inc Compositions comprenant une séquence prepro de facteur alpha et leurs utilisations
WO2025087397A1 (fr) * 2023-10-25 2025-05-01 Rona Bioscience, Limited Ligands pour l'administration extrahépatique d'arnsi

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4634665A (en) 1980-02-25 1987-01-06 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US5179017A (en) 1980-02-25 1993-01-12 The Trustees Of Columbia University In The City Of New York Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US4399216A (en) 1980-02-25 1983-08-16 The Trustees Of Columbia University Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials
US4656134A (en) 1982-01-11 1987-04-07 Board Of Trustees Of Leland Stanford Jr. University Gene amplification in eukaryotic cells
US5149636A (en) 1982-03-15 1992-09-22 Trustees Of Columbia University In The City Of New York Method for introducing cloned, amplifiable genes into eucaryotic cells and for producing proteinaceous products
GB8601597D0 (en) 1986-01-23 1986-02-26 Wilson R H Nucleotide sequences
US4956288A (en) 1988-04-22 1990-09-11 Biogen, Inc. Method for producing cells containing stably integrated foreign DNA at a high copy number, the cells produced by this method, and the use of these cells to produce the polypeptides coded for by the foreign DNA
US20030007963A1 (en) 1998-12-07 2003-01-09 Van Bree Johannes B. M. M. Treatment of pompe's disease
US6118045A (en) 1995-08-02 2000-09-12 Pharming B.V. Lysosomal proteins produced in the milk of transgenic animals
CN103269709A (zh) 2010-06-25 2013-08-28 夏尔人类遗传性治疗公司 Sanfilippo综合症类型B的治疗
WO2012122042A2 (fr) 2011-03-04 2012-09-13 Shire Human Genetic Therapies, Inc. Lieurs peptidiques pour compositions de polypeptide et procédés pour les utiliser

Also Published As

Publication number Publication date
EP3193942B1 (fr) 2020-03-25
WO2016025523A1 (fr) 2016-02-18
EP3193942A1 (fr) 2017-07-26
US10603364B2 (en) 2020-03-31
US20170246263A1 (en) 2017-08-31

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