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ceDNA PKU
- Development stage
- Preclinical
- Lead developer
- Generation Bio
- Modality
- Gene Therapies, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
- Administration
- Intravenous
01
Overview
ceDNA PKU is a non-viral, capsid-free gene therapy program developed by Generation Bio for the treatment of phenylketonuria (PKU). The therapy utilizes a proprietary closed-ended DNA (ceDNA) construct to deliver a functional copy of the phenylalanine hydroxylase (PAH) gene to the liver. This construct is encapsulated in a cell-targeted lipid nanoparticle (ctLNP) designed for hepatocyte-specific delivery. Unlike traditional adeno-associated virus (AAV) vectors, the ceDNA platform is intended to be non-immunogenic, potentially allowing for redosing to maintain therapeutic levels of PAH enzyme activity throughout a patient's life. The primary goal is to reduce toxic levels of phenylalanine in the blood, addressing the underlying cause of PKU.
- Other names
- Generation Bio PKU programPAH ceDNA
02
Targets
PAH (Phenylalanine hydroxylase)
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