BR112017002741A2 - peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas - Google Patents

peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas

Info

Publication number
BR112017002741A2
BR112017002741A2 BR112017002741-0A BR112017002741A BR112017002741A2 BR 112017002741 A2 BR112017002741 A2 BR 112017002741A2 BR 112017002741 A BR112017002741 A BR 112017002741A BR 112017002741 A2 BR112017002741 A2 BR 112017002741A2
Authority
BR
Brazil
Prior art keywords
therapeutic agent
mannose
containing peptides
lysosomal
phosphate containing
Prior art date
Application number
BR112017002741-0A
Other languages
English (en)
Inventor
F. Concino Michael
Strack-Logue Bettina
Meiyappan Muthuraman
W. NORTON Angela
Zhang Bohong
Iskenderian Andrea
Yoon LOW Lieh
Lundberg Dianna
Romashko Alla
Hicham Naimy
Original Assignee
Shire Human Genetic Therapies, Inc
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, Inc filed Critical Shire Human Genetic Therapies, Inc
Publication of BR112017002741A2 publication Critical patent/BR112017002741A2/pt

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
    • 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
    • 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
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K38/19—Cytokines; Lymphokines; Interferons
    • A61K38/20—Interleukins [IL]
    • A61K38/204—IL-6
    • 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
    • A61K38/22—Hormones
    • A61K38/30—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • 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
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4703—Inhibitors; Suppressors
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/54—Interleukins [IL]
    • C07K14/5412—IL-6
    • 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
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors (Somatomedins), e.g. IGF-1, IGF-2
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1288—Transferases for other substituted phosphate groups (2.7.8)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/08—Transferases for other substituted phosphate groups (2.7.8)
    • C12Y207/08017—UDP-N-acetylglucosamine--lysosomal-enzyme N-acetylglucosaminephosphotransferase (2.7.8.17)
    • 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
    • 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)

Abstract

um agente terapêutico direcionado incluindo uma enzima lisossômica e uma porção de direcionamento lisossômico que é um peptídeo contendo pelo menos um sítio de glicosilação ligada a n. métodos de produção do agente terapêutico direcionado podem incluir ácidos nucleotídicos que codificam a mesma células hospedeiras que coexpressam gnpt. composições farmacêuticas compreendendo o agente terapêutico direcionado e métodos de utilização das mesmas para o tratamento de uma doença de armazenamento lisossômico.
BR112017002741-0A 2014-08-11 2015-08-11 peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas BR112017002741A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462036082P 2014-08-11 2014-08-11
US62/036,082 2014-08-11
PCT/US2015/044713 WO2016025519A1 (en) 2014-08-11 2015-08-11 Mannose-6-phosphate bearing peptides fused to lysosomal enzymes

Publications (1)

Publication Number Publication Date
BR112017002741A2 true BR112017002741A2 (pt) 2018-02-27

Family

ID=54035297

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112017002741-0A BR112017002741A2 (pt) 2014-08-11 2015-08-11 peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas

Country Status (10)

Country Link
US (2) US10722559B2 (pt)
EP (1) EP3185889B1 (pt)
JP (1) JP6623213B2 (pt)
CN (1) CN106573039A (pt)
AU (1) AU2015301809A1 (pt)
BR (1) BR112017002741A2 (pt)
CA (1) CA2956469A1 (pt)
EA (1) EA201790178A1 (pt)
MX (1) MX2017001898A (pt)
WO (1) WO2016025519A1 (pt)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
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
MA50746A (fr) 2017-10-02 2020-08-12 Denali Therapeutics Inc Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive
CN113301925A (zh) 2018-12-19 2021-08-24 小利兰·斯坦福大学理事会 用于溶酶体靶向的双官能分子以及相关的组合物和方法
JP2022538497A (ja) * 2019-07-02 2022-09-02 エム６ピー セラピューティクス （スイッツァランド） ゲーエムベーハー ライソゾーム蓄積症を処置するためのベクター組成物およびそれを使用する方法
CN111455004B (zh) * 2020-02-29 2023-05-05 浙江农林大学 一种脂肪酶催化在线合成香豆素-3-羧酸-6’-o-d-甘露糖酯的方法
US20230211001A1 (en) * 2020-07-30 2023-07-06 The Board Of Trustees Of The Leland Stanford Junior University Lysosomal Targeting Molecules Comprising Knottin Peptides And Related Compositions And Methods
WO2023150387A1 (en) * 2022-02-07 2023-08-10 M6P Therapeutics, Inc. Compositions comprising acid alpha glucosidase and methods of use thereof

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040142474A1 (en) * 2000-09-14 2004-07-22 Expression Genetics, Inc. Novel cationic lipopolymer as a biocompatible gene delivery agent
US7560424B2 (en) * 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
CA2472937C (en) 2002-01-11 2014-06-17 Biomarin Pharmaceutical, Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
US20110027267A1 (en) * 2007-11-09 2011-02-03 Anaphore, Inc. Fusion Proteins of Mannose Binding Lectins for Treatment of Disease
PL2279210T3 (pl) * 2008-05-07 2017-10-31 Biomarin Pharm Inc Lizosomalne peptydy kierujące i ich zastosowania
EP2588130B1 (en) * 2010-06-25 2016-08-17 Shire Human Genetic Therapies, Inc. Cns delivery of therapeutic agents
JP6063380B2 (ja) 2010-06-25 2017-01-18 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド サンフィリポ症候群ｂ型の処置
EP2975134A3 (en) * 2010-07-22 2016-02-24 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
JP5959114B2 (ja) * 2011-05-19 2016-08-02 学校法人 明治薬科大学 リン酸化糖鎖を含有する組換えヒトサポシンｂタンパク質及びその用途
US9545450B2 (en) * 2011-05-27 2017-01-17 Amicus Therapeutics Inc. Methods for coupling targeting peptides onto recombinant lysosomal enzymes for improved treatments of lysosomal storage diseases
AU2012322811A1 (en) * 2011-10-12 2014-05-01 Synageva Biopharma Corp. Recombinant human NaGlu protein and uses thereof
KR102262882B1 (ko) 2012-11-27 2021-06-10 바이오마린 파머수티컬 인크. 표적화된 치료적 리소좀 효소 융합 단백질들 및 그들의 용도들

Also Published As

Publication number Publication date
CN106573039A (zh) 2017-04-19
EP3185889A1 (en) 2017-07-05
JP6623213B2 (ja) 2019-12-18
WO2016025519A1 (en) 2016-02-18
JP2017529326A (ja) 2017-10-05
MX2017001898A (es) 2017-04-11
EP3185889B1 (en) 2020-07-15
US20200376095A1 (en) 2020-12-03
CA2956469A1 (en) 2016-02-18
AU2015301809A1 (en) 2017-02-02
US10722559B2 (en) 2020-07-28
EA201790178A1 (ru) 2017-07-31
US20170232076A1 (en) 2017-08-17

Similar Documents

Publication Publication Date Title
BR112017002741A2 (pt) peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas
BR112016002753A2 (pt) anticorpos contra o inibidor-1 de ativador de plasminogênio (pai-1) e usos dos mesmos
BR112019023856A2 (pt) proteínas triespecíficas que visam msln e métodos de utilização
CY1116424T1 (el) Παραγωγο αδενινης ως αναστολεας ρι3κ
EA201591281A1 (ru) Рибонуклеиновые кислоты с 4'-тиомодифицированными нуклеотидами и связанные с ними способы
BR112017018728A2 (pt) sistema de vetor para expressar a sequência de codificação de um gene de interesse em uma célula, célula hospedeira, composição farmacêutica, método para tratar e/ou prevenir uma patologia ou doença, uso de uma sequência de nucleotídeo de um sinal de degradação em um sistema de vetor e método para diminuir a expressão de uma proteína na forma truncada
BR112015012152A2 (pt) Proteína de fusão terapêutica direcionada que compreende uma enzima lisossomal, ácido nucleico, composição farmacêutica, método para produzir a dita proteína e usos da proteína para tratar uma doença de armazenamento lisossomal e mucopolissacaridose tipo iiib
EA201890725A1 (ru) Агонисты фарнезоидного х-рецептора и их применение
BR112017011923A2 (pt) bactéria modificada para tratar doenças associadas com hiperamonemia
BR112017007765A2 (pt) composições de adenosina deaminase-2 (ada2), variantes do mesmo e métodos de usar o mesmo
BR212016030926U2 (pt) conjugados de alvo e partículas e formulações dos mesmos
BR112015019995A2 (pt) Combinação terapêutica envolvendo anticorpos contra claudina 18.2 para tratamento de câncer
BR112016017041A2 (pt) Proteína isolada tendo atividade de fosfatase, polinucleotídeo, vetor, célula hospedeira, método para produzir uma proteína e composição
BR112016004095A2 (pt) administração de enzimas de empobrecimento de quinurenina para a terapia contra tumores
BR112012004806B8 (pt) composições imunogênicas que incluem moduladores da atividade de tlr, método para aumento da eficácia da referida composição e uso
EA201991409A2 (ru) Антитела к ингибитору активатора плазминогена 1 (pai-1) и пути их применения
EA201590554A1 (ru) Ингибиторы глюкозилцерамид-синтазы
BR112018005287A2 (pt) fitases modificadas e métodos de usar as mesmas
BR112012020558A2 (pt) moduladores do receptor de androgênio e seus usos
CR20130377A (es) Métodos y productos de fármaco para tratar enfermedad de alzheimer
CO7010783A2 (es) Proteína naglu humana recombinante y usos de la misma
BR112014026057A2 (pt) análogos limitados por anel como inibidores de arginase
BR112013001753A2 (pt) método para preparar uma composição d einter-alfa-inibidor (iaip) eriquecida de plasma, solução aquosa de iaip, e, método para tratar uma doença ou distúrbio associado com disfunção de iaip e associado com atividadede protease de serina no plasma aumentada.
EA201692292A1 (ru) Соединения и композиции для стимулирования хондрогенеза
BR112018071221A2 (pt) composições e métodos para programar células terapêuticas usando nanocarreadores de ácido nucleico alvejados

Legal Events

Date Code Title Description
B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]

Free format text: DE ACORDO COM O ARTIGO 229-C DA LEI NO 10196/2001, QUE MODIFICOU A LEI NO 9279/96, A CONCESSAO DA PATENTE ESTA CONDICIONADA A ANUENCIA PREVIA DA ANVISA. CONSIDERANDO A APROVACAO DOS TERMOS DO PARECER NO 337/PGF/EA/2010, BEM COMO A PORTARIA INTERMINISTERIAL NO 1065 DE 24/05/2012, ENCAMINHA-SE O PRESENTE PEDIDO PARA AS PROVIDENCIAS CABIVEIS.

B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements