Anti-transferrin receptor (TfR) antibody and uses thereof
Inventors
Subramanian, Romesh R. • Qatanani, Mohammed T. • Weeden, Timothy • Desjardins, Cody A. • Quinn, Brendan
Assignees
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Publication Number
US-12624120-B2
Publication Date
2026-05-12
Expiration Date
Abstract
Aspects of the disclosure relate to antibodies that bind to transferrin receptor (e.g., transferrin receptor 1) and complexes comprising the antibody covalently linked to a molecular payload. Methods of making and using the antibodies are also provided.
Core Innovation
The invention describes an antibody that binds human transferrin receptor, comprising a heavy chain variable region and a light chain variable region containing one of three specified combinations of complementarity-determining regions. Described antibody formats include full-length IgG, IgG1 kappa, Fab, Fab', F(ab')2, scFv, and Fv. The antibody functions as a targeting component covalently linked to molecular payloads, including oligonucleotides, proteins, small molecules, and diagnostic agents.
The disclosure describes selective transferrin receptor 1 binders that exhibit binding to human and non-human-primate transferrin receptor 1 without binding transferrin receptor 2 or nonspecific serum proteins. These antibodies and antibody-payload conjugates mediate cellular uptake, targeted delivery to tissues expressing transferrin receptor such as brain and muscle, DMPK knockdown, and DMD exon skipping. The background section is omitted, so the problem being solved is not explicitly described.
Claims Coverage
The claim coverage includes one independent claim and encompasses three inventive features across the provided claim set.
Human transferrin receptor-binding antibody
The independent claim covers an antibody that binds human transferrin receptor and comprises a heavy chain variable region and a light chain variable region containing one of three specified combinations of CDR-H1, CDR-H2, CDR-H3, CDR-L1, CDR-L2, and CDR-L3.
Antibody formats and binding affinity
Dependent claim elements specify antibody formats including full-length IgG, Fab, Fab', F(ab')2, scFv, and Fv, as well as a defined K_D affinity range for transferrin receptor 1 binding.
Antibody-linked payload complexes and delivery uses
Dependent claim elements describe a complex wherein the antibody is covalently linked to a molecular payload, as well as detection of transferrin receptor in a biological sample and delivery of the molecular payload to a subject's brain or muscle.
The independent claim centers on a human transferrin receptor-binding antibody defined by specified complementarity-determining region combinations, while dependent claim coverage extends to antibody formats, binding affinity, covalently linked molecular payload complexes, biological detection, and targeted delivery.
Stated Advantages
The reported results describe TfR1 binding without TfR2 binding or nonspecific serum-protein binding.
The conjugates are reported to mediate cellular uptake and DMPK knockdown in myotubes, enhance DMD exon skipping in patient myotubes, and reduce DMPK in multiple muscle tissues in vivo.
The reported linker stability is at least 75% at 72 hours across species.
Documented Applications
Detection of transferrin receptor in a biological sample by contacting the sample with the antibody and measuring binding.
Delivery of a molecular payload to a subject's brain or muscle using the antibody-payload complex.
Diagnostic and therapeutic applications in muscle and neurological disorders, including therapeutic oligonucleotide delivery evaluated for DMPK antisense knockdown and Duchenne muscular dystrophy exon-skipping activity.
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