Our investigational therapies are designed with the intent that the therapeutic protein will be available throughout the patient’s body, including the nervous system, either by correcting specialized blood cells, and/or in the plasma available for take-up into other cells, which is called “cross correction.”
Our process is designed to potentially enable widespread engraftment in the brain of microglia cells containing the therapeutic gene. The goal is to address the CNS symptoms that can arise from lysosomal disorders, such as impaired cognition.
Circulating the therapeutic protein in other hard-to-reach areas, such as the bone and muscle, is also crucial to delivering an effective therapy.
In Pompe disease, for instance, our team is using a proprietary technology, known as a GILT tag, intended to facilitate delivery of the protein so it is readily taken up by the muscle. This is crucial for people living with Pompe disease, a progressive disorder that relentlessly erodes neuromuscular control, leading to major muscle weakness.