TWI821197B - 經工程改造之轉鐵蛋白受體結合多肽 - Google Patents

經工程改造之轉鐵蛋白受體結合多肽 Download PDF

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TWI821197B
TWI821197B TW107128067A TW107128067A TWI821197B TW I821197 B TWI821197 B TW I821197B TW 107128067 A TW107128067 A TW 107128067A TW 107128067 A TW107128067 A TW 107128067A TW I821197 B TWI821197 B TW I821197B
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seq
sequence
polypeptide
polypeptide comprises
numbered
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TW107128067A
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Chinese (zh)
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TW201920278A (zh
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曉誠 陳
馬克 S 丹尼斯
米漢利斯 卡里歐立斯
亞當 P 席佛曼
安基塔 斯里瓦斯塔娃
萊恩 J 瓦茲
羅伯特 C 威爾斯
喬伊 瑜 蘇奇洛
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美商戴納立製藥公司
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/79Transferrins, e.g. lactoferrins, ovotransferrins
    • 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
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/40Transferrins, e.g. lactoferrins, ovotransferrins
    • 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
    • A61K47/62Medicinal 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
    • A61K47/64Drug-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
    • A61K47/644Transferrin, e.g. a lactoferrin or ovotransferrin
    • 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
    • A61K47/6801Drug-antibody or immunoglobulin conjugates defined by the pharmacologically or therapeutically active agent
    • A61K47/6803Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates
    • A61K47/6811Drugs conjugated to an antibody or immunoglobulin, e.g. cisplatin-antibody conjugates the drug being a protein or peptide, e.g. transferrin or bleomycin
    • 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
    • A61K47/6835Medicinal 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
    • A61K47/6849Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
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    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
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    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
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    • 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
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52Constant or Fc region; Isotype
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
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    • C07K2317/526CH3 domain
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
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    • C07K2317/55Fab or Fab'
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    • C07ORGANIC CHEMISTRY
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    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
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    • C07ORGANIC CHEMISTRY
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    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/94Stability, e.g. half-life, pH, temperature or enzyme-resistance
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2318/00Antibody mimetics or scaffolds
    • C07K2318/20Antigen-binding scaffold molecules wherein the scaffold is not an immunoglobulin variable region or antibody mimetics

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  • 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)
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  • 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)
TW107128067A 2017-08-10 2018-08-10 經工程改造之轉鐵蛋白受體結合多肽 TWI821197B (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
WOPCT/US2018/018371 2018-02-15
??PCT/US2018/018371 2018-02-15
PCT/US2018/018371 WO2018152326A1 (en) 2017-02-17 2018-02-15 Engineered transferrin receptor binding polypeptides

Publications (2)

Publication Number Publication Date
TW201920278A TW201920278A (zh) 2019-06-01
TWI821197B true TWI821197B (zh) 2023-11-11

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RS (1) RS64584B1 (enExample)
SI (1) SI3665192T1 (enExample)
SM (1) SMT202300347T1 (enExample)
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MY205898A (en) 2017-02-17 2024-11-19 Denali Therapeutics Inc Engineered transferrin receptor binding polypeptides
EP3692070A1 (en) 2017-10-02 2020-08-12 Denali Therapeutics Inc. Fusion proteins comprising enzyme replacement therapy enzymes
BR112021002953A2 (pt) * 2018-08-22 2021-05-11 Denali Therapeutics Inc. polipeptídeos anti-her2 e métodos de uso dos mesmos
AR118144A1 (es) 2019-02-20 2021-09-22 Denali Therapeutics Inc Anticuerpos anti-trem2 y métodos para utilizarlos
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MX2022008582A (es) * 2020-01-13 2022-08-10 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos.
AU2021224200A1 (en) * 2020-02-19 2022-09-08 Denali Therapeutics Inc. Engineered anti-HER2 bispecific proteins
KR20230086703A (ko) 2020-10-14 2023-06-15 데날리 테라퓨틱스 인크. 설포글루코사민 설포하이드롤라제 효소를 포함하는 융합 단백질 및 이의 방법
MX2024002753A (es) 2021-09-01 2024-05-20 Biogen Ma Inc Anticuerpos anti receptor de transferrina y usos de los mismos.
WO2023128702A1 (ko) * 2021-12-31 2023-07-06 주식회사 아임뉴런 뇌혈관장벽 투과성 융합 단백질 및 이의 용도
CA3257739A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS CONTAINING A MODIFIED TRANSFERRIN RECEPTOR LOCUS
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
US20240052051A1 (en) 2022-07-29 2024-02-15 Regeneron Pharmaceuticals, Inc. Anti-tfr:payload fusions and methods of use thereof
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
EP4634218A2 (en) * 2022-12-16 2025-10-22 Denali Therapeutics Inc. Methods and compositions related to engineered transferrin receptor-binding molecules
TW202519551A (zh) 2023-07-28 2025-05-16 美商雷傑納榮製藥公司 用於治療酸性神經髓磷脂酶缺乏症之抗TfR:酸性神經髓磷脂酶
TW202513801A (zh) 2023-07-28 2025-04-01 美商雷傑納榮製藥公司 用於治療龐貝氏症之抗TfR:GAA及抗CD63:GAA插入
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
CN111148757B (zh) 2024-06-21
JP2020530293A (ja) 2020-10-22
JP2020530465A (ja) 2020-10-22
JP2025120209A (ja) 2025-08-15
LT3665192T (lt) 2023-10-25
ES2956062T3 (es) 2023-12-12
PL3665192T3 (pl) 2023-11-27
FI3665192T3 (fi) 2023-09-28
US20240024432A1 (en) 2024-01-25
HRP20231118T1 (hr) 2023-12-22
EP3665192B1 (en) 2023-07-12
CN111094336A (zh) 2020-05-01
EP3665194A1 (en) 2020-06-17
JP2023123757A (ja) 2023-09-05
DK3665192T5 (da) 2024-08-26
WO2019032955A1 (en) 2019-02-14
PT3665192T (pt) 2023-09-25
US20230381286A1 (en) 2023-11-30
HUE063021T2 (hu) 2023-12-28
US20200289627A1 (en) 2020-09-17
CA3072035A1 (en) 2019-02-14
DK3665192T3 (da) 2023-10-09
US20200369746A1 (en) 2020-11-26
SI3665192T1 (sl) 2023-11-30
MD3665192T2 (ro) 2023-12-31
WO2019033046A1 (en) 2019-02-14
JP7280241B2 (ja) 2023-05-23
CN111148757A (zh) 2020-05-12
TW201920278A (zh) 2019-06-01
EP3665192A1 (en) 2020-06-17
CA3072051A1 (en) 2019-02-14

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