CN111148757B - 工程改造的转铁蛋白受体结合多肽 - Google Patents

工程改造的转铁蛋白受体结合多肽 Download PDF

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Publication number
CN111148757B
CN111148757B CN201880063660.0A CN201880063660A CN111148757B CN 111148757 B CN111148757 B CN 111148757B CN 201880063660 A CN201880063660 A CN 201880063660A CN 111148757 B CN111148757 B CN 111148757B
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seq
sequence
polypeptide
polypeptide consists
pcp
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Chinese (zh)
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CN111148757A (zh
Inventor
陈晓诚
马克·S·丹尼斯
米哈利斯·卡里奥利斯
亚当·P·西尔弗曼
安基塔·斯利瓦斯塔瓦
赖安·J·沃茨
罗伯特·C·威尔斯
乔伊·余·祖切罗
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Denali Therapeutics Inc
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Denali Therapeutics Inc
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    • 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
    • A61K38/40—Transferrins, e.g. lactoferrins, ovotransferrins
    • 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
    • A61K47/644—Transferrin, e.g. a lactoferrin or ovotransferrin
    • 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
    • A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6801—Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811—Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • 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
    • A61K47/68—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—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 an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • C07K14/79—Transferrins, e.g. lactoferrins, ovotransferrins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD71
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52—Constant or Fc region; Isotype
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52—Constant or Fc region; Isotype
    • C07K2317/526—CH3 domain
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55—Fab or Fab'
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/70—Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77—Internalization into the cell
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/94—Stability, e.g. half-life, pH, temperature or enzyme-resistance
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2318/00—Antibody mimetics or scaffolds
    • C07K2318/20—Antigen-binding scaffold molecules wherein the scaffold is not an immunoglobulin variable region or antibody mimetics

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
CN201880063660.0A 2017-08-10 2018-08-10 工程改造的转铁蛋白受体结合多肽 Active CN111148757B (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762543658P 2017-08-10 2017-08-10
US62/543,658 2017-08-10
US201762583314P 2017-11-08 2017-11-08
US62/583,314 2017-11-08
USPCT/US2018/018371 2018-02-15
PCT/US2018/018371 WO2018152326A1 (en) 2017-02-17 2018-02-15 Engineered transferrin receptor binding polypeptides
PCT/US2018/046199 WO2019032955A1 (en) 2017-08-10 2018-08-10 MODIFIED TRANSFERRIN RECEPTOR BINDING POLYPEPTIDES

Publications (2)

Publication Number Publication Date
CN111148757A CN111148757A (zh) 2020-05-12
CN111148757B true CN111148757B (zh) 2024-06-21

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CN201880063660.0A Active CN111148757B (zh) 2017-08-10 2018-08-10 工程改造的转铁蛋白受体结合多肽
CN201880053177.4A Pending CN111094336A (zh) 2017-08-10 2018-08-10 使用转铁蛋白受体结合蛋白的基于亲和力的方法

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DK (1) DK3665192T5 (enExample)
ES (1) ES2956062T3 (enExample)
FI (1) FI3665192T3 (enExample)
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PL (1) PL3665192T3 (enExample)
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SM (1) SMT202300347T1 (enExample)
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FI3583120T3 (fi) 2017-02-17 2023-01-13 Muunneltuja transferriinireseptoria sitovia polypeptidejä
CA3076369A1 (en) 2017-10-02 2019-04-11 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
KR20210074279A (ko) * 2018-08-22 2021-06-21 데날리 테라퓨틱스 인크. 항-her2 폴리펩타이드 및 이의 사용방법
EP3927743A1 (en) 2019-02-20 2021-12-29 Denali Therapeutics Inc. Anti-trem2 antibodies and methods of use thereof
AU2020411480B2 (en) 2019-12-23 2023-12-21 Denali Therapeutics Inc. Progranulin variants
KR20220131246A (ko) * 2020-01-13 2022-09-27 데날리 테라퓨틱스 인크. 항-trem2 항체 및 이의 사용 방법
CA3170338A1 (en) * 2020-02-19 2021-08-26 Denali Therapeutics Inc. Engineered anti-her2 bispecific proteins
MA71661A (fr) 2020-10-14 2025-05-30 Denali Therapeutics Inc. Protéines de fusion comprenant des enzymes sulfoglucosamine sulfohydrolase et méthodes associées
CR20240139A (es) 2021-09-01 2024-06-14 Biogen Ma Inc Anticuerpos del receptor antitransferrina y usos de los mismos
US20250066449A1 (en) * 2021-12-31 2025-02-27 Imnewrun, Inc. Blood-brain barrier permeable fusion protein and uses thereof
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JP2025525792A (ja) 2022-07-29 2025-08-07 リジェネロン・ファーマシューティカルズ・インコーポレイテッド トランスフェリン受容体１に対して再標的化されるウイルス粒子
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
IL317261A (en) 2022-07-29 2025-01-01 Regeneron Pharma Non-human animals containing a modified transferrin receptor locus
EP4634218A2 (en) * 2022-12-16 2025-10-22 Denali Therapeutics Inc. Methods and compositions related to engineered transferrin receptor-binding molecules
WO2025029657A2 (en) 2023-07-28 2025-02-06 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertion for treatment of pompe disease
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
WO2025137213A1 (en) 2023-12-20 2025-06-26 Denali Therapeutics Inc. Fusion proteins comprising acid alpha-glucosidase enzymes and methods thereof

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EP3665194A4 (en) 2021-07-07
RS64584B1 (sr) 2023-10-31
EP3665194A1 (en) 2020-06-17
PT3665192T (pt) 2023-09-25
TWI821197B (zh) 2023-11-11
HUE063021T2 (hu) 2023-12-28
CA3072035A1 (en) 2019-02-14
SMT202300347T1 (it) 2023-11-13
WO2019033046A1 (en) 2019-02-14
JP2025120209A (ja) 2025-08-15
PL3665192T3 (pl) 2023-11-27
JP7280241B2 (ja) 2023-05-23
FI3665192T3 (fi) 2023-09-28
HRP20231118T1 (hr) 2023-12-22
US20200369746A1 (en) 2020-11-26
SI3665192T1 (sl) 2023-11-30
DK3665192T3 (da) 2023-10-09
CN111148757A (zh) 2020-05-12
MD3665192T2 (ro) 2023-12-31
WO2019032955A1 (en) 2019-02-14
EP3665192B1 (en) 2023-07-12
US20240024432A1 (en) 2024-01-25
DK3665192T5 (da) 2024-08-26
JP2020530293A (ja) 2020-10-22
CN111094336A (zh) 2020-05-01
US20230381286A1 (en) 2023-11-30
JP2023123757A (ja) 2023-09-05
JP2020530465A (ja) 2020-10-22
US20200289627A1 (en) 2020-09-17
LT3665192T (lt) 2023-10-25
EP3665192A1 (en) 2020-06-17
ES2956062T3 (es) 2023-12-12
CA3072051A1 (en) 2019-02-14
TW201920278A (zh) 2019-06-01

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