JP7280241B2 - 操作されたトランスフェリン受容体結合ポリペプチド - Google Patents

操作されたトランスフェリン受容体結合ポリペプチド Download PDF

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JP7280241B2
JP7280241B2 JP2020506964A JP2020506964A JP7280241B2 JP 7280241 B2 JP7280241 B2 JP 7280241B2 JP 2020506964 A JP2020506964 A JP 2020506964A JP 2020506964 A JP2020506964 A JP 2020506964A JP 7280241 B2 JP7280241 B2 JP 7280241B2
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seq
sequence
polypeptide
ala
polypeptide comprises
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JP2020530293A5 (https=
JP2020530293A (ja
Inventor
シャオチェン チェン
マーク エス． デニス
ミハリス カリオリス
アダム ピー． シルバーマン
アンキタ スリヴァスタヴァ
ライアン ジェイ． ワッツ
ロバート シー． ウェルズ
ジョイ ユ ズチェロ
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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 [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)
JP2020506964A 2017-08-10 2018-08-10 操作されたトランスフェリン受容体結合ポリペプチド Active JP7280241B2 (ja)

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 (3)

Publication Number Publication Date
JP2020530293A JP2020530293A (ja) 2020-10-22
JP2020530293A5 JP2020530293A5 (https=) 2021-09-16
JP7280241B2 true JP7280241B2 (ja) 2023-05-23

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JP2020506964A Active JP7280241B2 (ja) 2017-08-10 2018-08-10 操作されたトランスフェリン受容体結合ポリペプチド
JP2020507090A Pending JP2020530465A (ja) 2017-08-10 2018-08-10 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法
JP2023107814A Pending JP2023123757A (ja) 2017-08-10 2023-06-30 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法
JP2025090730A Pending JP2025120209A (ja) 2017-08-10 2025-05-30 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法

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JP2020507090A Pending JP2020530465A (ja) 2017-08-10 2018-08-10 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法
JP2023107814A Pending JP2023123757A (ja) 2017-08-10 2023-06-30 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法
JP2025090730A Pending JP2025120209A (ja) 2017-08-10 2025-05-30 トランスフェリン受容体結合タンパク質を使用するための、親和性に基づく方法

Country Status (19)

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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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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
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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
WO2026052652A1 (en) * 2024-09-03 2026-03-12 F. Hoffmann-La Roche Ag Cytokine receptor agonist
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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
TWI821197B (zh) 2023-11-11
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
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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