CA3072035A1 - Engineered transferrin receptor binding polypeptides - Google Patents

Engineered transferrin receptor binding polypeptides Download PDF

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Publication number
CA3072035A1
CA3072035A1 CA3072035A CA3072035A CA3072035A1 CA 3072035 A1 CA3072035 A1 CA 3072035A1 CA 3072035 A CA3072035 A CA 3072035A CA 3072035 A CA3072035 A CA 3072035A CA 3072035 A1 CA3072035 A1 CA 3072035A1
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CA
Canada
Prior art keywords
seq
sequence
polypeptide
polypeptide comprises
numbered
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CA3072035A
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English (en)
French (fr)
Inventor
Xiaocheng Chen
Mark S. Dennis
Mihalis Kariolis
Adam P. Silverman
Ankita SRIVASTAVA
Ryan J. Watts
Robert C. Wells
Joy Yu Zuchero
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Denali Therapeutics Inc
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 CA3072035A1 publication Critical patent/CA3072035A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • 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
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • 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
    • 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
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52Constant or Fc region; Isotype
    • C07K2317/526CH3 domain
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/50Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/55Fab or Fab'
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/70Immunoglobulins specific features characterized by effect upon binding to a cell or to an antigen
    • C07K2317/77Internalization into the cell
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2317/00Immunoglobulins specific features
    • C07K2317/90Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/94Stability, 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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  • 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)
CA3072035A 2017-08-10 2018-08-10 Engineered transferrin receptor binding polypeptides Pending CA3072035A1 (en)

Applications Claiming Priority (5)

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
PCT/US2018/046199 WO2019032955A1 (en) 2017-08-10 2018-08-10 MODIFIED TRANSFERRIN RECEPTOR BINDING POLYPEPTIDES

Publications (1)

Publication Number Publication Date
CA3072035A1 true CA3072035A1 (en) 2019-02-14

Family

ID=65272509

Family Applications (2)

Application Number Title Priority Date Filing Date
CA3072035A Pending CA3072035A1 (en) 2017-08-10 2018-08-10 Engineered transferrin receptor binding polypeptides
CA3072051A Pending CA3072051A1 (en) 2017-08-10 2018-08-10 Affinity-based methods for using transferrin receptor-binding proteins

Family Applications After (1)

Application Number Title Priority Date Filing Date
CA3072051A Pending CA3072051A1 (en) 2017-08-10 2018-08-10 Affinity-based methods for using transferrin receptor-binding proteins

Country Status (19)

Country Link
US (4) US20200289627A1 (enExample)
EP (2) EP3665194A4 (enExample)
JP (4) JP7280241B2 (enExample)
CN (2) CN111148757B (enExample)
CA (2) CA3072035A1 (enExample)
DK (1) DK3665192T5 (enExample)
ES (1) ES2956062T3 (enExample)
FI (1) FI3665192T3 (enExample)
HR (1) HRP20231118T1 (enExample)
HU (1) HUE063021T2 (enExample)
LT (1) LT3665192T (enExample)
MD (1) MD3665192T2 (enExample)
PL (1) PL3665192T3 (enExample)
PT (1) PT3665192T (enExample)
RS (1) RS64584B1 (enExample)
SI (1) SI3665192T1 (enExample)
SM (1) SMT202300347T1 (enExample)
TW (1) TWI821197B (enExample)
WO (2) WO2019032955A1 (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
CN120265655A (zh) 2022-07-29 2025-07-04 瑞泽恩制药公司 抗tfr:有效载荷融合物及其使用方法
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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Also Published As

Publication number Publication date
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
CN111148757B (zh) 2024-06-21
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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