WO2022032140 VESICLE TARGETING PROTEINS AND USES OF SAME

Settings

Stemming

Single Family Member

Include NPL

Feedback

Goto Application

Publication Number WO/2022/032140

Publication Date 10.02.2022

International Application No. PCT/US2021/045012

International Filing Date 06.08.2021

IPC

CPC

View more classifications

View less classifications

Applicants

AMICUS THERAPEUTICS, INC. [US]/[US]

Inventors

BAUMAN, Joseph
WU, Xiaoyang
MCANANY, Yuliya

Agents

ALEGRIA, Rory P.

Priority Data

63/062,844		07.08.2020		US
63/160,215		12.03.2021		US

Publication Language English (en)

Filing Language English (EN)

Designated States

View all

Title

(EN) VESICLE TARGETING PROTEINS AND USES OF SAME

(FR) PROTÉINES DE CIBLAGE DES VÉSICULES ET LEURS UTILISATIONS

Abstract

(EN) The disclosure provides a novel method for treating genetic disorders where a peptide sequence targets proteins produced via gene therapy into exosomes. These protein-loaded exosomes can enter into non-transduced cells and correct pathology. Also, gene therapy compositions, protein replacement therapy composition, pharmaceutical compositions, methods of treatment, and uses of the gene therapy compositions and the recombinant proteins are also disclosed. The method can also be used to improve in vitro recombinant protein yield.

(FR) L'invention concerne un nouveau procédé de traitement de troubles génétiques selon lequel une séquence peptidique cible des protéines produites par thérapie génique dans des exosomes. Ces exosomes chargés de protéines peuvent pénétrer dans des cellules non transduites et corriger une pathologie. L'invention concerne également des compositions de thérapie génique, une composition de thérapie de remplacement de protéines, des compositions pharmaceutiques, des procédés de traitement et des utilisations des compositions de thérapie génique et des protéines recombinantes. Le procédé peut également être utilisé pour améliorer le rendement de protéines recombinantes in vitro.

Latest bibliographic data on file with the International Bureau

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48	Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9	Medicinal preparations characterised by special physical form
48	Preparations in capsules, e.g. of gelatin, of chocolate
50	Microcapsules
51	Nanocapsules

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
47	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
30	Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
42	Proteins; Polypeptides; Degradation products thereof; Derivatives thereof, e.g. albumin, gelatin or zein

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48	Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

A	HUMAN NECESSITIES
61	MEDICAL OR VETERINARY SCIENCE; HYGIENE
K	PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
9	Medicinal preparations characterised by special physical form
48	Preparations in capsules, e.g. of gelatin, of chocolate
50	Microcapsules
51	Nanocapsules

Processing

National Phase Entries

Authority File

Settings

Feedback

Goto Application

1. WO2022032140 - VESICLE TARGETING PROTEINS AND USES OF SAME

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
14	Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N	MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15	Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09	Recombinant DNA-technology
63	Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79	Vectors or expression systems specially adapted for eukaryotic hosts
85	for animal cells
86	Viral vectors

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Y	ENZYMES
207	Transferases transferring phosphorus-containing groups (2.7)
11	Protein-serine/threonine kinases (2.7.11)

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
2319	Fusion polypeptide
01	containing a localisation/targetting motif
033	containing a motif for targeting to the internal surface of the plasma membrane, e.g. containing a myristoylation motif

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
2319	Fusion polypeptide
01	containing a localisation/targetting motif
033	containing a motif for targeting to the internal surface of the plasma membrane, e.g. containing a myristoylation motif

C	CHEMISTRY; METALLURGY
07	ORGANIC CHEMISTRY
K	PEPTIDES
2319	Fusion polypeptide
01	containing a localisation/targetting motif
055	containing a signal for localisation to secretory granules (for exocytosis)

C	CHEMISTRY; METALLURGY
12	BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
Y	ENZYMES
302	Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
01	Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
0101	Oligo-1,6-glucosidase (3.2.1.10), i.e. sucrase