AVR-RD-01

AVR-RD-01 is an investigational ex vivo lentiviral gene therapy developed for the treatment of Fabry disease. The therapy utilizes autologous hematopoietic stem cells (HSCs) that are genetically modified with a lentiviral vector to carry a functional copy of the GLA gene, which encodes the enzyme alpha-galactosidase A (α-Gal A). Following a conditioning regimen, the modified cells are re-infused into the patient, where they engraft in the bone marrow and differentiate into various blood cell lineages that continuously produce and secrete the functional enzyme. This process, known as cross-correction, aims to reduce the systemic accumulation of globotriaosylceramide (Gb3) and globotriaosylsphingosine (lyso-Gb3), potentially providing a one-time curative treatment for patients with Fabry disease. Although it showed promising results in Phase 1 and Phase 2 trials, the developer announced the discontinuation of the program in early 2022.

Other names

lentivirus alpha-gal A transduced stem cellsautologous CD34+ cells transduced with lentiviral vector encoding alpha-galactosidase Alentivirus alpha-gal A transduced CD34+ cells