AVR-RD-04

AVR-RD-04 is an investigational gene therapy designed for the treatment of cystinosis, a rare genetic lysosomal storage disorder. The therapy works by ex vivo genetic modification of a patient’s own hematopoietic stem cells (HSCs) using a lentiviral vector to introduce a functional copy of the CTNS gene, which encodes the protein cystinosin. This approach aims to restore normal cystine metabolism and prevent multi-organ damage caused by cystine accumulation. Clinical data indicate that AVR-RD-04 has been well-tolerated with no drug-related adverse events reported and has shown potential benefits in multiple tissues including eyes, skin, gastrointestinal mucosa, and neurocognitive system. The therapy has received orphan drug designation and rare pediatric disease designation from the FDA[2][3][4][5][6][8][9].

Other names

CTNS-RD-04CTNS-RD04CTNS-RD 04