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1. (WO2014194427) TARGETED IDURONATE-2-SULFATASE FUSION PROTEINS
Latest bibliographic data on file with the International Bureau   

Pub. No.: WO/2014/194427 International Application No.: PCT/CA2014/050522
Publication Date: 11.12.2014 International Filing Date: 06.06.2014
IPC:
C12N 9/16 (2006.01) ,A61K 47/48 (2006.01) ,A61K 9/127 (2006.01) ,A61K 9/14 (2006.01) ,A61P 3/00 (2006.01) ,C07K 14/81 (2006.01) ,C07K 19/00 (2006.01) ,C07K 7/08 (2006.01) ,C12N 15/62 (2006.01) ,C12N 9/96 (2006.01) ,C12P 21/02 (2006.01) ,C07K 16/00 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14
Hydrolases (3.)
16
acting on ester bonds (3.1)
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, inert additives
48
the non-active ingredient being chemically bound to the active ingredient, e.g. polymer drug conjugates
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
10
Dispersions; Emulsions
127
Liposomes
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
14
Particulate form, e.g. powders
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
3
Drugs for disorders of the metabolism
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
14
Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
81
Protease inhibitors
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
19
Hybrid peptides
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
7
Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
04
Linear peptides containing only normal peptide links
08
having 12 to 20 amino acids
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; 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
11
DNA or RNA fragments; Modified forms thereof
62
DNA sequences coding for fusion proteins
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9
Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
96
Stabilising an enzyme by forming an adduct or a composition; Forming enzyme conjugates
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
P
FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
21
Preparation of peptides or proteins
02
having a known sequence of two or more amino acids, e.g. glutathione
C CHEMISTRY; METALLURGY
07
ORGANIC CHEMISTRY
K
PEPTIDES
16
Immunoglobulins, e.g. monoclonal or polyclonal antibodies
Applicants:
ANGIOCHEM INC. [CA/CA]; 201 President-Kennedy Avenue Suite PK-2880 Montreal, Québec H2X 3Y7, CA
Inventors:
BOIVIN, Dominique; CA
DEMEULE, Michel; CA
Agent:
GOUDREAU GAGE DUBUC; 2000 McGill College Suite 2200 Montreal, Québec H3A 3H3, CA
Priority Data:
61/831,91306.06.2013US
Title (EN) TARGETED IDURONATE-2-SULFATASE FUSION PROTEINS
(FR) PROTÉINES DE FUSION IDURONATE-2-SULFATASE CIBLÉES
Abstract:
(EN) The present invention is directed to polypeptides and peptidomimetics that include a targeting moiety capable of transporting said polypeptide or peptidomimetic to the lysosome and/or across the blood brain barrier, and a lysosomal enzyme, methods for their production and precursors in their production. The polypeptides and peptidomimetics of the invention are produced using a method which utilizes a precursor comprising a tag. The reduces cleavage within the amino acid sequence of the polypeptide or peptidomimetic compared to levels of cleavage observed in the absence of the tag. These compounds are exemplified by IDS-Angiopep-2 polypeptides and peptidomimetics which can be used to treat MPS-I I. Where the targeting moiety transports the polypeptide or peptidomimetic across the blood brain barrier, these polypeptides and peptidomimetics can treat not only the peripheral disease symptoms, but may also be effective in treating CNS symptoms. Where the targeting moiety transports the polypeptide or peptidomimetic to the lysosomes, it is expected that the polypeptide or peptidomimetic will be more effective than IDS itself.
(FR) Cette invention concerne des polypeptides et des peptidomimétiques qui comprennent un fragment de ciblage capable de transporter ledit polypeptide ou peptidomimétique vers le lysosome et/ou à travers la barrière hémato-encéphalique, et une enzyme lysosomale, des procédés pour les produire et des précurseurs dans leur production. Les polypeptides et les peptidomimétiques selon l'invention sont produits à l'aide d'un procédé qui utilise un précurseur comprenant une étiquette. L'étiquette réduit le clivage au sein de la séquence d'acides aminés du polypeptide ou du peptidomimétique comparativement aux niveaux de clivage observés en l'absence de l'étiquette. Ces composés sont illustrés par les polypeptides et les peptidomimétiques IDS-Angiopep-2 qui peuvent être utilisés pour traiter la MPS-II. Quand le fragment de ciblage transporte le polypeptide ou le peptidomimétique à travers la barrière hémato-encéphalique, ces polypeptides et peptidomimétiques peuvent traiter non seulement les symptômes pathologiques périphériques, mais peuvent également être efficaces pour traiter les symptômes du SNC. Quand le fragment de ciblage transporte le polypeptide ou le peptidomimétique vers les lysosomes, le polypeptide ou le peptidomimétique devrait être plus efficace que l'IDS lui-même.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LT, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Office (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (EPO) (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)