The majority of treatments for life-threatening diseases address symptoms rather than root causes. Some delay disease progression and the onset of complications associated with the underlying illness, but are not curative. This is especially true for rare diseases where all too often no effective treatment, or only partial solutions, are available to the patient. The definitive solution is to address the root cause – the defective gene.
At AVROBIO, we seek to directly target the genetic cause by providing a healthy gene using a lentiviral gene delivery system. Our focus is on innovative gene therapies for rare diseases, where a faulty gene results in a lack of an essential protein or enzyme. We identify the defective gene in each disease target, then select a subset of a patient’s own cells to incorporate a fully functional gene and infuse these cells back into the patient. Our investigational, state-of-the-art gene therapies take only three days to manufacture, and can be administered on an outpatient basis. The desired result is durable 24/7 expression of the essential protein or enzyme by the patient’s own cells.