Non-Human Animals Comprising a Modified Transferrin Receptor Locus
Summary
The USPTO published patent application US20260107929A1 on April 23, 2026, filed July 28, 2023 under application no. 19099378. The application covers non-human animal cells and non-human animals comprising a humanized Tfrc gene at an endogenous Tfrc locus, which express a human TfR protein or fragments thereof. The application also discloses non-human animal cells and animals comprising a humanized Tfrc gene combined with a knockout mutation in a Gaa gene, intended for screening anti-human-TfR binding protein-based therapies for Pompe disease.
“Non-human animal cells and non-human animals comprising a humanized Tfrc gene and a knockout mutation in a Gaa gene, e.g., at an endogenous Gaa locus, and methods of using such non-human animal cells and non-human animals are also provided.”
About this source
GovPing monitors USPTO Patent Applications - Biotech (C12N) for new healthcare & life sciences regulatory changes. Every update since tracking began is archived, classified, and available as free RSS or email alerts — 268 changes logged to date.
What changed
The USPTO published patent application US20260107929A1 covering non-human animal cells and non-human animals engineered with a humanized Tfrc gene at the endogenous Tfrc locus. The disclosed invention enables expression of human TfR protein or fragments thereof and further encompasses combinations with Gaa gene knockout mutations for therapeutic screening applications targeting Pompe disease. Inventors include Andrew J. Murphy, Yajun Tang, Qing Fang, Maria Praggastis, Jun Zhong, Katherine Cygnar, and Alexander O. Mujica.
Pharmaceutical and biotechnology companies engaged in rare disease therapeutic development, particularly Pompe disease programs involving TfR-targeting modalities, should review this published application for potential licensing considerations or freedom-to-operate implications. The disclosed animal models represent a specific screening platform for anti-human-TfR binding protein-based therapies and may inform related research programs.
Archived snapshot
Apr 24, 2026GovPing captured this document from the original source. If the source has since changed or been removed, this is the text as it existed at that time.
NON-HUMAN ANIMALS COMPRISING A MODIFIED TRANSFERRIN RECEPTOR LOCUS
Application US20260107929A1 Kind: A1 Apr 23, 2026
Inventors
Andrew J. Murphy, Yajun Tang, Qing Fang, Maria Praggastis, Jun Zhong, Katherine Cygnar, Alexander O. Mujica
Abstract
Non-human animal cells and non-human animals comprising a humanized Tfrc gene, e.g., at an endogenous Tfrc locus, and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized Tfrc gene express a human TfR protein or fragments thereof. Non-human animal cells and non-human animals comprising a humanized Tfrc gene and a knockout mutation in a Gaa gene, e.g., at an endogenous Gaa locus, and methods of using such non-human animal cells and non-human animals are also provided. Such animals are useful to screen anti-human-TfR binding protein based therapies of, e.g., Pompe disease.
CPC Classifications
A01K 67/0276 A01K 67/0271 C07K 14/47 C12N 5/0602 C12N 15/10 C12N 15/11 A01K 2207/12 A01K 2207/15 A01K 2217/075 A01K 2227/105 A01K 2267/0306 C12N 2510/00
Filing Date
2023-07-28
Application No.
19099378
Related changes
Get daily alerts for USPTO Patent Applications - Biotech (C12N)
Daily digest delivered to your inbox.
Free. Unsubscribe anytime.
Source
About this page
Every important government, regulator, and court update from around the world. One place. Real-time. Free. Our mission
Source document text, dates, docket IDs, and authority are extracted directly from USPTO.
The summary, classification, recommended actions, deadlines, and penalty information are AI-generated from the original text and may contain errors. Always verify against the source document.
Classification
Who this affects
Taxonomy
Browse Categories
Get alerts for this source
We'll email you when USPTO Patent Applications - Biotech (C12N) publishes new changes.
Subscribed!
Optional. Filters your digest to exactly the updates that matter to you.