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Fab'-siHPRT
- Development stage
- Unknown
- Lead developer
- GenAhead Bio
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
- Administration
- Intramuscular, Intravenous
01
Overview
Fab'-siHPRT is a preclinical research-stage antibody-siRNA conjugate (ARC) developed by GenAhead Bio. It consists of an anti-transferrin receptor (TfR/CD71) Fab' antibody fragment covalently linked to a small interfering RNA (siRNA) targeting hypoxanthine-guanine phosphoribosyltransferase (HPRT). Designed as a model construct to demonstrate GenAhead Bio's nucleic acid delivery platform, Fab'-siHPRT targets TfR to facilitate receptor-mediated endocytosis and deliver the siRNA payload into cells. This construct has been shown to achieve robust and sustained gene silencing of the HPRT reporter gene in cardiac and skeletal muscle tissues following intravenous or intramuscular administration.
- Other names
- anti-TfR (CD71) antibody fragment - siHPRTanti-TfR antibody fragment-HPRT siRNA conjugateanti-TfR antibody fragment-siHPRTanti-TfR/CD71 Fab' fragment-HPRT siRNA conjugate
02
Targets
TFRC (Transferrin Receptor)
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