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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Priority claimed from PCT/US2018/018371 external-priority patent/WO2018152326A1/en
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    • 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
    • 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
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2881—Immunoglobulins [IG], 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 [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IG], 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)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Neurosurgery (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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 WO2018152326A1 (en) 2017-02-17 2018-02-15 Engineered transferrin receptor binding polypeptides
??PCT/US2018/018371 2018-02-15

Publications (2)

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

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US (4) US20200369746A1 (https=)
EP (2) EP3665192B1 (https=)
JP (4) JP7280241B2 (https=)
CN (2) CN111094336A (https=)
CA (2) CA3072035A1 (https=)
DK (1) DK3665192T5 (https=)
ES (1) ES2956062T3 (https=)
FI (1) FI3665192T3 (https=)
HR (1) HRP20231118T1 (https=)
HU (1) HUE063021T2 (https=)
LT (1) LT3665192T (https=)
MD (1) MD3665192T2 (https=)
PL (1) PL3665192T3 (https=)
PT (1) PT3665192T (https=)
RS (1) RS64584B1 (https=)
SI (1) SI3665192T1 (https=)
SM (1) SMT202300347T1 (https=)
TW (1) TWI821197B (https=)
WO (2) WO2019032955A1 (https=)

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BR112019016989A2 (pt) 2017-02-17 2020-05-26 Denali Therapeutics Inc. Polipeptídeos de ligação ao receptor de transferrina manipulados
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KR20210074279A (ko) * 2018-08-22 2021-06-21 데날리 테라퓨틱스 인크. 항-her2 폴리펩타이드 및 이의 사용방법
PH12021552002A1 (en) 2019-02-20 2022-09-19 Denali Therapeutics Inc Anti-trem2 antibodies and methods of use thereof
CA3165846A1 (en) 2019-12-23 2021-07-01 Denali Therapeutics Inc. Progranulin variants
PE20221465A1 (es) * 2020-01-13 2022-09-21 Denali Therapeutics Inc Anticuerpos anti-trem2 y metodos para utilizarlos
CN115361972A (zh) * 2020-02-19 2022-11-18 戴纳立制药公司 工程化抗her2双特异性蛋白
PE20231931A1 (es) 2020-10-14 2023-12-01 Denali Therapeutics Inc Proteinas de fusion que comprenden enzimas sulfoglucosamina sulfohidrolasa y metodos de estas
EP4396231A1 (en) 2021-09-01 2024-07-10 Biogen MA Inc. Anti-transferrin receptor antibodies and uses thereof
AU2022428997A1 (en) * 2021-12-31 2024-08-15 Imnewrun, Inc. Blood-brain barrier permeable fusion protein and uses thereof
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
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
CA3263494A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. ANTI-TFR FUSIONS: PAYLOAD AND THEIR METHODS OF USE
AU2023316646A1 (en) 2022-07-29 2024-12-12 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified transferrin receptor locus
WO2024130116A2 (en) * 2022-12-16 2024-06-20 Denali Therapeutics Inc. Methods and compositions related to engineered transferrin receptor‑binding molecules
AU2024317483A1 (en) 2023-07-28 2026-01-29 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
TW202542190A (zh) 2023-12-20 2025-11-01 美商戴納立製藥公司 包含酸性α—葡萄糖苷酶之融合蛋白及其方法
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US20200369746A1 (en) 2020-11-26
US20240024432A1 (en) 2024-01-25
US20230381286A1 (en) 2023-11-30
RS64584B1 (sr) 2023-10-31
HUE063021T2 (hu) 2023-12-28
EP3665192B1 (en) 2023-07-12
SMT202300347T1 (it) 2023-11-13
WO2019032955A1 (en) 2019-02-14
DK3665192T5 (da) 2024-08-26
CN111094336A (zh) 2020-05-01
EP3665194A1 (en) 2020-06-17
DK3665192T3 (da) 2023-10-09
EP3665192A1 (en) 2020-06-17
HRP20231118T1 (hr) 2023-12-22
CA3072035A1 (en) 2019-02-14
CA3072051A1 (en) 2019-02-14
ES2956062T3 (es) 2023-12-12
JP7280241B2 (ja) 2023-05-23
JP2020530465A (ja) 2020-10-22
CN111148757B (zh) 2024-06-21
EP3665194A4 (en) 2021-07-07
JP2025120209A (ja) 2025-08-15
MD3665192T2 (ro) 2023-12-31
FI3665192T3 (fi) 2023-09-28
US20200289627A1 (en) 2020-09-17
SI3665192T1 (sl) 2023-11-30
PL3665192T3 (pl) 2023-11-27
CN111148757A (zh) 2020-05-12
LT3665192T (lt) 2023-10-25
TW201920278A (zh) 2019-06-01
JP2020530293A (ja) 2020-10-22
PT3665192T (pt) 2023-09-25
JP2023123757A (ja) 2023-09-05
WO2019033046A1 (en) 2019-02-14

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