MA40791A - Ciblage lysosomal d'enzymes et utilisations associées - Google Patents
Ciblage lysosomal d'enzymes et utilisations associéesInfo
- Publication number
- MA40791A MA40791A MA040791A MA40791A MA40791A MA 40791 A MA40791 A MA 40791A MA 040791 A MA040791 A MA 040791A MA 40791 A MA40791 A MA 40791A MA 40791 A MA40791 A MA 40791A
- Authority
- MA
- Morocco
- Prior art keywords
- enzymes
- lysosomal targeting
- lysosomal
- related uses
- compositions
- Prior art date
Links
Classifications
-
- 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
- A61K47/65—Peptidic linkers, binders or spacers, e.g. peptidic enzyme-labile linkers
-
- 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
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
-
- 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
- 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)
- C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
- C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
- C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
- C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
- C12N9/6424—Serine endopeptidases (3.4.21)
- C12N9/6454—Dibasic site splicing serine proteases, e.g. kexin (3.4.21.61); furin (3.4.21.75) and other proprotein convertases
-
- 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
- C12N9/96—Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
-
- 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)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/50—Fusion polypeptide containing protease site
Abstract
L'invention fournit des compositions et des procédés pour un ciblage lysosomal efficace médié par pcsk9. En particulier, les compositions et les procédés fournis par l'invention peuvent être utilisés pour traiter des maladies de stockage lysosomales telles que pompe disease et sanfilippo syndrome type b, et ils peuvent être utilisés pour cibler des enzymes lysosomales sur les différents muscles du corps humain.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201462068033P | 2014-10-24 | 2014-10-24 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA40791A true MA40791A (fr) | 2017-09-27 |
Family
ID=54478975
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA040791A MA40791A (fr) | 2014-10-24 | 2015-10-23 | Ciblage lysosomal d'enzymes et utilisations associées |
Country Status (4)
Country | Link |
---|---|
US (1) | US10556015B2 (fr) |
EP (1) | EP3220958A1 (fr) |
MA (1) | MA40791A (fr) |
WO (1) | WO2016065319A1 (fr) |
Families Citing this family (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20220133863A1 (en) * | 2010-06-25 | 2022-05-05 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
AU2016365834B2 (en) * | 2015-12-08 | 2023-09-28 | Regeneron Pharmaceuticals, Inc. | Compositions and methods for internalizing enzymes |
KR20240001721A (ko) | 2016-09-12 | 2024-01-03 | 제네똥 | 산-알파 글루코시다제 변이체 및 이의 용도 |
EP3293203A1 (fr) * | 2016-09-12 | 2018-03-14 | Genethon | Variants d'alpha-glucosidase acide et leurs utilisations |
EP3293259A1 (fr) * | 2016-09-12 | 2018-03-14 | Genethon | Variants d'alpha-glucosidase acide et leurs utilisations |
CA3066569A1 (fr) | 2017-06-07 | 2018-12-13 | Regeneron Pharmaceuticals, Inc. | Compositions et methodes pour l'internalisation d'enzymes |
US20200206323A1 (en) * | 2017-09-08 | 2020-07-02 | The Nemours Foundation | An agent, a device and a blood-circulation system for treating lysosomal storage diseases, and a method for treating lysosomal storage diseases |
EP3787748A1 (fr) * | 2018-05-01 | 2021-03-10 | OrfoNeuro ApS | Traitement de la céroïde-lipofuscinose neuronale |
Family Cites Families (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2003102583A1 (fr) | 2002-05-29 | 2003-12-11 | Symbiontics, Inc. | Proteines therapeutiques ciblees |
US7629309B2 (en) | 2002-05-29 | 2009-12-08 | Zystor Therapeutics, Inc. | Targeted therapeutic proteins |
EP1716232B9 (fr) * | 2004-02-10 | 2010-10-13 | ZyStor Therapeutics , Inc. | Alpha-glucosidase acide et fragments de celle-ci |
WO2007128121A1 (fr) * | 2006-05-08 | 2007-11-15 | Adaerata, Limited Partnership | Protéines pcsk9 chimériques, cellules les comprenant et dosages les utilisant |
HUE034850T2 (en) | 2008-05-07 | 2018-03-28 | Biomarin Pharm Inc | Lysosomal targeting peptides and their use |
EP3679942A1 (fr) | 2009-06-17 | 2020-07-15 | BioMarin Pharmaceutical Inc. | Formulations pour des enzymes lysosomales |
ES2643015T3 (es) * | 2010-06-25 | 2017-11-21 | Shire Human Genetic Therapies, Inc. | Tratamiento del Síndrome de Sanfilippo Tipo B |
EP2471929A1 (fr) * | 2010-12-29 | 2012-07-04 | Algenics | Production de protéines glycosylées à forte teneur en mannose stockées dans les plastides de micro-algues |
AU2013352184B2 (en) | 2012-11-27 | 2018-05-31 | Biomarin Pharmaceutical Inc. | Targeted therapeutic lysosomal enzyme fusion proteins and uses thereof |
-
2015
- 2015-10-23 US US15/521,130 patent/US10556015B2/en active Active
- 2015-10-23 WO PCT/US2015/057214 patent/WO2016065319A1/fr active Application Filing
- 2015-10-23 EP EP15791835.0A patent/EP3220958A1/fr active Pending
- 2015-10-23 MA MA040791A patent/MA40791A/fr unknown
Also Published As
Publication number | Publication date |
---|---|
EP3220958A1 (fr) | 2017-09-27 |
US20170333569A1 (en) | 2017-11-23 |
WO2016065319A1 (fr) | 2016-04-28 |
US10556015B2 (en) | 2020-02-11 |
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