Engineered polypeptides

A technology of constructs and sequences, applied in the direction of peptides, hybrid peptides, specific peptides, etc., can solve problems such as limiting the therapeutic use of macromolecules

Pending Publication Date: 2019-11-26
DENALI THERAPEUTICS INC
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

AI Technical Summary

Problems solved by technology

[0001] The blood-brain barrier (BBB) ​​blocks the peripheral entry of most macromolecules into the brain, thereby limiting the therapeutic use of these macromolecules

Method used

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  • Engineered polypeptides
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  • Engineered polypeptides

Examples

Experimental program
Comparison scheme
Effect test

Embodiment 1

[0409] Example 1. Generation of TfR targets

[0410] DNA encoding the transferrin receptor (TfR) extracellular domain (ECD) (residues 121-760 of human (SEQ ID NO: 235) or cynomolgus monkey (SEQ ID NO: 300) TfR) was cloned into the In mammalian expression vectors with C-terminal cleaved His and Avi tags. The plasmid was transfected into HEK293 cells and allowed to express. The extracellular domain was purified from the collected supernatant using Ni-NTA chromatography followed by size exclusion chromatography to remove any aggregated protein. The yield was about 5 mg per liter of culture. Store protein in 10 mM K 3 PO 4 (pH 6.7), 100 mM KCl, 100 mM NaCl, and 20% glycerol and frozen at -20°C.

[0411] DNA encoding the permuted TfR apical domain (SEQ ID NO: 301) (residues 326-379 and 194-296 of human or cynomolgus TfR) was cloned with an N-terminal His-tag for purification and for in vitro Biotinylated Avi-tagged pET28 vector. The plasmid was co-transformed into BL21(DE3) ...

Embodiment 2

[0414] Example 2. Design and Characterization of Engineered Transferrin Receptor Binding Polypeptides

[0415] This example describes the design, generation and characterization of polypeptides of the invention. For purposes of this example and comparing amino acids that are identical in cloned sequences, "conservative" mutations are considered mutations present in all clones identified (non-conservative amino acid substitutions), while "semi-conservative" mutations are those present in >50% Mutations present in clones.

[0416] Unless otherwise indicated, the positions of amino acid residues in this section are based on the numbering of SEQ ID NO: 1 , the human IgGl wild-type Fc region has three residues from the hinge, PCP, at the amino terminus.

[0417] Design of Peptide Fc Region Library

[0418]By selecting certain solvent-exposed surface patches for modification, constructing surface-display libraries in which the amino acid composition of the selected patches is al...

Embodiment 3

[0575] Example 3. Using Biacore TM Binding characteristics of other CH3C variants identified

[0576] Use Biacore TM T200 instrument, determination of affinity of clonal variants to recombinant TfR apical domain by surface plasmon resonance. Biacore TM S-series CM5 sensor chips were immobilized with anti-human Fab (Human Fab Capture Kit from GE Healthcare). 5 μg / mL of polypeptide-Fab fusions were captured on each flow cell for 1 min and injected into serial 3-fold dilutions of human or cynomolgus apical domains at a flow rate of 30 μL / min at room temperature. Each sample was assayed with 45 minutes of association and 3 minutes of dissociation. After each injection, the chip was regenerated using 10 mM glycine hydrochloride (pH 2.1). Binding responses were corrected for by subtracting RU from the flow cell capturing irrelevant IgG at a similar density. By using Biacore TM T200 evaluation software v3.1, for the response of concentration fitting equilibrium to obtain the...

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Abstract

The present invention provides polypeptides that bind to a blood-brain barrier (BBB) receptor, methods of generating such polypeptides, and methods of using the polypeptides to target a composition toa BBB receptor-expressing cell, e.g., for transport across the BBB. Also provided herein are transferrin receptor (TfR) constructs that comprise a monomeric TfR apical domain or one or more portionsof the TfR apical domain which have been circularly permuted relative to the full-length TfR sequence.

Description

Background technique [0001] The blood-brain barrier (BBB) ​​blocks the peripheral entry of most macromolecules into the brain, thereby limiting the therapeutic use of these macromolecules. Receptors expressed on endothelium, including that of the blood-brain barrier, can mediate the delivery of receptor-bound ligands across the blood-brain barrier. Contents of the invention [0002] In one aspect, the present disclosure provides an isolated recombinant transferrin receptor (TfR) construct comprising a monomeric TfR apical domain, wherein the construct does not comprise a TfR Protease-like domain or helical domain. In one embodiment, the construct displays a conserved epitope or antigen and / or retains the approximate three-dimensional structure of the apical domain of native human TfR, or has a root mean square deviation (RMSD) of less than about 2. In one embodiment, the three-dimensional structure is measured by X-ray crystallography. In one embodiment, the construct com...

Claims

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Application Information

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Patent Type & AuthorityApplications(China)
IPC IPC(8): C07K14/705C07K19/00C12N15/63G01N33/68
CPCC07K14/70582C07K19/00G01N33/68G01N2500/10
Inventor陈晓诚米哈利斯·卡里奥利斯罗伯特·C·威尔斯
OwnerDENALI THERAPEUTICS INC