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PRINTed GLA
- Development stage
- Preclinical
- Lead developer
- Addition Therapeutics
- Modality
- Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
PRINTed GLA is a preclinical-stage, non-viral, lipid nanoparticle (LNP)-encapsulated genetic medicine being developed by Addition Therapeutics for the treatment of Fabry disease. The therapy utilizes Addition's proprietary PRINT (Precise, RNA-Mediated, Insertion of Transgenes) technology, which leverages retrotransposon machinery (specifically an R2 retrotransposase) to precisely insert a healthy copy of the GLA transgene into a highly conserved ribosomal DNA 'safe-harbor' site in the genome. This precise insertion enables stable, durable, and therapeutic expression of functional alpha-galactosidase A (α-Gal A) in the liver, leading to sustained secretion of the enzyme into the circulation and subsequent reduction of toxic globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3) accumulation in key organs such as the liver, kidney, and heart.
- Other names
- PRINTed GLA medicinePRINTed GLA transgene
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