PE20231931A1 - Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas - Google Patents

Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas

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Publication number
PE20231931A1
PE20231931A1 PE2023001396A PE2023001396A PE20231931A1 PE 20231931 A1 PE20231931 A1 PE 20231931A1 PE 2023001396 A PE2023001396 A PE 2023001396A PE 2023001396 A PE2023001396 A PE 2023001396A PE 20231931 A1 PE20231931 A1 PE 20231931A1
Authority
PE
Peru
Prior art keywords
enzymes
methods
fusion proteins
sgsh
amino acid
Prior art date
Application number
PE2023001396A
Other languages
English (en)
Inventor
Tina Giese
Gunasekaran Kannan
Mihalis Kariolis
Cathal Mahon
Original Assignee
Denali Therapeutics 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 Denali Therapeutics Inc filed Critical Denali Therapeutics Inc
Publication of PE20231931A1 publication Critical patent/PE20231931A1/es

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal 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
    • A61K47/51Medicinal 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
    • A61K47/68Medicinal 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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y310/00Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y310/00Hydrolases acting on sulfur-nitrogen bonds (3.10)
    • C12Y310/01Hydrolases acting on sulfur-nitrogen bonds (3.10) acting on sulfur-nitrogen bonds (3.10.1)
    • C12Y310/01001N-Sulfoglucosamine sulfohydrolase (3.10.1.1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K2039/505Medicinal preparations containing antigens or antibodies comprising antibodies
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/40Immunoglobulins specific features characterized by post-translational modification
    • C07K2317/41Glycosylation, sialylation, or fucosylation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52Constant or Fc region; Isotype
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se refiere a proteinas que pueden transportarse a traves de la barrera hematoencefalica (BHE) que comprenden: a) un primer polipeptido Fc unido a una primera secuencia de aminoacidos de Nsulfoglucosamina sulfohidrolasa (SGSH), una secuencia de aminoacidos variante de SGSH o un fragmento cataliticamente activo de esta; y b) un segundo polipeptido Fc unido a una segunda secuencia de aminoacidos de SGSH, donde el segundo polipeptido Fc puede unirse especificamente a un receptor de transferrina (TfR); y donde el segundo polipeptido Fc comprende una secuencia que tiene una identidad de al menos 80 % con respecto a la SEQ ID NO: 37 y tiene Ala en la posicion 389, de acuerdo con la numeracion EU.
PE2023001396A 2020-10-14 2021-10-13 Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas PE20231931A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063091800P 2020-10-14 2020-10-14
PCT/US2021/054860 WO2022081765A1 (en) 2020-10-14 2021-10-13 Fusion proteins comprising sulfoglucosamine sulfohydrolase enzymes and methods thereof

Publications (1)

Publication Number Publication Date
PE20231931A1 true PE20231931A1 (es) 2023-12-01

Family

ID=78725609

Family Applications (1)

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PE2023001396A PE20231931A1 (es) 2020-10-14 2021-10-13 Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas

Country Status (17)

Country Link
US (2) US11884944B2 (es)
EP (1) EP4229192A1 (es)
JP (1) JP2023545707A (es)
KR (1) KR20230086703A (es)
CN (1) CN116916947A (es)
AR (1) AR123788A1 (es)
AU (1) AU2021362209A1 (es)
CA (1) CA3197506A1 (es)
CL (1) CL2023001034A1 (es)
CO (1) CO2023005260A2 (es)
CR (1) CR20230178A (es)
EC (1) ECSP23035251A (es)
IL (1) IL302029A (es)
MX (1) MX2023004335A (es)
PE (1) PE20231931A1 (es)
TW (1) TW202229547A (es)
WO (1) WO2022081765A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3583120T5 (da) 2017-02-17 2024-09-02 Denali Therapeutics Inc Modificerede transferrinreceptorbindende polypeptider
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
EP4081536A1 (en) 2019-12-23 2022-11-02 Denali Therapeutics Inc. Progranulin variants

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5863782A (en) 1995-04-19 1999-01-26 Women's And Children's Hospital Synthetic mammalian sulphamidase and genetic sequences encoding same
US6194551B1 (en) 1998-04-02 2001-02-27 Genentech, Inc. Polypeptide variants
EP1463512B1 (en) 2002-01-11 2014-05-28 biOasis Technologies Inc. Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
US20050142141A1 (en) 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
AU2003286870A1 (en) 2003-06-05 2005-01-04 Salk Institute For Biological Studies Targeting polypeptides to the central nervous system
CN105274072A (zh) 2008-01-18 2016-01-27 生物马林药物股份有限公司 制备活性高度磷酸化人溶酶体硫酸酯酶及其应用
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
PT3626257T (pt) 2010-06-25 2021-11-12 Shire Human Genetic Therapies Métodos e composições de arilsulfatase a para a administração no snc
MX2013000320A (es) 2010-06-25 2013-06-05 Shire Human Genetic Therapies Composiciones y metodos para suministro al sistema nervioso central de heparan n-sulfatasa.
HUE041335T2 (hu) 2011-03-29 2019-05-28 Roche Glycart Ag Antitest FC-variánsok
US8722019B2 (en) 2011-08-05 2014-05-13 Bioasis Technologies, Inc. P97 fragments with transfer activity
AU2013296557B2 (en) 2012-07-31 2019-04-18 Bioasis Technologies Inc. Dephosphorylated lysosomal storage disease proteins and methods of use thereof
TR201909156T4 (tr) 2012-08-29 2019-07-22 Hoffmann La Roche Kan beyin bariyeri mekiği.
EP2970433B1 (en) 2013-03-13 2019-09-18 Bioasis Technologies Inc. Fragments of p97 and uses thereof
US20160184458A1 (en) 2013-03-14 2016-06-30 Shire Human Genetic Therapies, Inc. Mrna therapeutic compositions and use to treat diseases and disorders
EP3730516A1 (en) 2013-07-22 2020-10-28 Armagen, Inc. Methods and compositions for increasing enzyme activity in the cns
MX368947B (es) 2014-04-01 2019-10-22 Swedish Orphan Biovitrum Ab Publ Sulfamidasa modificada y su producción.
ES2749383T3 (es) 2014-11-06 2020-03-20 Hoffmann La Roche Variantes de la región Fc con unión al FcRn modificada y procedimientos de uso
EP3221361B1 (en) 2014-11-19 2021-04-21 Genentech, Inc. Anti-transferrin receptor / anti-bace1 multispecific antibodies and methods of use
KR20180020277A (ko) 2015-06-24 2018-02-27 제이씨알 파마 가부시키가이샤 혈액뇌관문을 통과하는 항인간 트랜스페린 수용체 항체
HUE057952T2 (hu) 2015-06-24 2022-06-28 Hoffmann La Roche Anti-transzferrin receptor antitestek testreszabott affinitással
DK3386534T3 (da) 2015-12-08 2020-11-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
AR107483A1 (es) 2016-01-29 2018-05-02 Hanmi Pharm Ind Co Ltd Conjugado de enzimas terapéuticas
AU2017222620B2 (en) 2016-02-24 2022-06-16 Biomarin Pharmaceutical Inc. Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
AU2017311040B2 (en) 2016-08-06 2023-08-31 Ossianix, Inc. In vivo methods for selecting peptides that cross the blood brain barrier, related compositions and methods of use
MX2019002252A (es) 2016-08-25 2019-07-04 Japan Chem Res Metodo para producir una proteina de fusion de anticuerpo.
EP3556399A1 (en) 2016-12-19 2019-10-23 Hanmi Pharm. Co., Ltd. Brain targeting long-acting protein conjugate
BR112019009316A2 (pt) 2016-12-26 2019-08-06 Japan Chem Res anticorpo antirreceptor de transferrina humana, proteína de fusão, fragmento de dna, vetor de expressão, célula de mamífero, complexo de composto farmacologicamente ativo e de anticorpo antirreceptor de transferrina humana, usos do anticorpo antirreceptor de transferrina humana e da proteína de fusão, e, métodos para tratamento de um distúrbio do sistema nervoso central e de uma doença do sistema nervoso central que acompanha a doença de pompe, a síndrome de hurler ou a síndrome de hurler-scheie.
KR102471458B1 (ko) 2016-12-28 2022-11-25 제이씨알 파마 가부시키가이샤 동결건조 제제
MX2019009817A (es) 2017-02-17 2019-11-21 Denali Therapeutics Inc Anticuerpos anti-tau y metodos de uso de los mismos.
DK3583120T5 (da) 2017-02-17 2024-09-02 Denali Therapeutics Inc Modificerede transferrinreceptorbindende polypeptider
SG11201907419PA (en) 2017-02-17 2019-09-27 Denali Therapeutics Inc Transferrin receptor transgenic models
GB201702863D0 (en) 2017-02-22 2017-04-05 Evox Therapeutics Ltd Improved loading of EVs with therapeutic proteins
CN111094559A (zh) 2017-07-07 2020-05-01 韩美药品株式会社 新型治疗酶融合蛋白及其用途
HUE063021T2 (hu) 2017-08-10 2023-12-28 Denali Therapeutics Inc Mesterségesen elõállított transzferrin receptor-kötõ polipeptidek
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
WO2019094608A1 (en) 2017-11-08 2019-05-16 Denali Therapeutics Inc. Anti-bace1 antibodies and methods of use thereof
BR112020012346A2 (pt) 2017-12-22 2020-11-24 Hanmi Pharm. Co., Ltd. proteína de fusão enzimática terapêutica tendo uma nova estrutura e uso da mesma
CN111741977A (zh) 2018-01-10 2020-10-02 戴纳立制药公司 转铁蛋白受体结合性多肽和其用途
WO2019145500A1 (en) 2018-01-26 2019-08-01 Swedish Orphan Biovitrum Ab (Publ) Method of treatment
MX2020012518A (es) 2018-06-18 2021-02-16 Denali Therapeutics Inc Proteinas de fusion que comprenden progranulina.
EP3850007A4 (en) 2018-08-16 2022-08-10 Denali Therapeutics Inc. MODIFIED B-SPECIFIC PROTEINS
JP2021534220A (ja) 2018-08-22 2021-12-09 デナリ セラピューティクス インコーポレイテッドDenali Therapeutics Inc. 抗her2ポリペプチド及びそれらの使用の方法
MX2021011612A (es) 2019-04-03 2021-12-10 Denali Therapeutics Inc Formulaciones de moleculas proteicas que comprenden iduronato 2-sulfatasa.
TW202138384A (zh) 2019-12-23 2021-10-16 美商戴納立製藥公司 顆粒蛋白前體變異體
EP4081536A1 (en) 2019-12-23 2022-11-02 Denali Therapeutics Inc. Progranulin variants
WO2021146256A1 (en) 2020-01-13 2021-07-22 Denali Therapeutics Inc. Anti-trem2 antibodies and methods of use thereof
EP4090682A1 (en) 2020-01-13 2022-11-23 Denali Therapeutics Inc. Anti-trem2 antibodies and methods of use thereof
EP4100419A4 (en) 2020-02-07 2024-05-29 Denali Therapeutics Inc. METHODS OF TREATING HUNTER SYNDROME
BR112022016232A2 (pt) 2020-02-19 2022-11-16 Denali Therapeutics Inc Proteínas biespecíficas anti-her2 manipuladas

Also Published As

Publication number Publication date
TW202229547A (zh) 2022-08-01
MX2023004335A (es) 2023-05-04
US11884944B2 (en) 2024-01-30
CL2023001034A1 (es) 2023-10-06
CR20230178A (es) 2023-07-26
JP2023545707A (ja) 2023-10-31
EP4229192A1 (en) 2023-08-23
CN116916947A (zh) 2023-10-20
WO2022081765A1 (en) 2022-04-21
US20230062800A1 (en) 2023-03-02
KR20230086703A (ko) 2023-06-15
CA3197506A1 (en) 2022-04-21
IL302029A (en) 2023-06-01
AR123788A1 (es) 2023-01-11
ECSP23035251A (es) 2023-10-31
CO2023005260A2 (es) 2023-05-19
AU2021362209A1 (en) 2023-05-18
US20240150736A1 (en) 2024-05-09

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