A new Cambridge gene and cell therapy startup, AvroBio, just launched with seed funding from Atlas Venture. The company plans to launch phase 1 trials this year for both acute myeloid leukemia and Fabry disease – a rare inherited lysosomal storage disease.
AvroBio’s lentiviral gene therapy approach works by infusing genetically modified cells into a patient. Its first program, AVR-01, is an immunotherapy approach to acute myeloid leukemia. The company extracts the patient’s cancer cells, and genetically modifies it to express a powerful immune signaling agent. These cells are then transferred back into the patient, and activate killer T cells to attack the remaining cancer in the body.
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AvroBio’s second program, AVR-02, is aimed at Fabry disease – genetically modifying a patient’s cells to add a functional copy of the faulty gene that causes the disease. It modifies hematopoetic stem cells to express an enzyme called alpha-galactosidase – then, when infused back in the patient’s body – they’re meant to produce the enzyme that the patient otherwise lacks.
The underlying research comes from Toronto’s University Health Network.