AVR-RD-02

AVR-RD-02 is an investigational ex vivo lentiviral gene therapy developed for the treatment of Gaucher disease. The therapy uses autologous CD34+ enriched hematopoietic stem cells (HSCs) that are genetically modified with a lentiviral vector to introduce a functional copy of the GBA gene, which encodes the enzyme beta-glucocerebrosidase (GCase). This modification aims to restore normal levels of GCase in patients with Gaucher disease, reducing pathological accumulation of glucosylceramide and glucosylsphingosine in tissues. The process involves collecting a patient’s own HSCs, modifying them ex vivo to carry the healthy GBA gene using a non-replicating lentiviral vector, and then reinfusing these cells back into the patient. The goal is durable expression of functional enzyme throughout the body[1][4][5][6][7][8]. Originally developed by AVROBIO and now under Tectonic Therapeutic following a merger, AVR-RD-02 has received orphan drug designation from both FDA and EMA as well as rare pediatric disease designation from FDA[3][4]. Clinical development included phase 1/2 trials for Gaucher disease type 1 but was suspended due to business decisions rather than safety or efficacy concerns[3].

Other names

AVR-RD-02AVR-RD02AVR-RD 02AVROBIO Gaucher gene therapy