scAAV9/JeT-GAN for GAN

This trial tests a new treatment for Giant Axonal Neuropathy (GAN), a rare and serious disorder affecting nerves and potentially leading to severe health issues. The study explores the safety of injecting a specific gene therapy, scAAV9/JeT-GAN, directly into the left vagus nerve to address symptoms related to the autonomic nervous system. Suitable candidates for this trial include those with confirmed GAN who have previously tried a related treatment through spinal injection. Participants must attend all study visits and adhere to study procedures. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative therapy.

The trial protocol does not specify if you need to stop taking your current medications. However, if you have a chronic illness requiring medication that the Principal Investigator believes poses unnecessary risks for the gene transfer, you may not be eligible to participate.

Research has shown that scAAV9/JeT-GAN underwent testing using intrathecal administration, which delivers the treatment directly into the spinal canal. In that study, participants generally tolerated the treatment well. Over approximately 68.7 months, 682 adverse events were reported, but the treatment did not cause serious harm in most cases.

Unlike current treatments for Giant Axonal Neuropathy (GAN), which primarily focus on managing symptoms, scAAV9/JeT-GAN directly targets the root cause of the disease. This treatment is unique because it uses a modified viral vector, adeno-associated virus serotype 9 (AAV9), to deliver optimized human gigaxonin DNA directly into the cells. Gigaxonin is the protein mutated in GAN, and by restoring its function, scAAV9/JeT-GAN aims to address the underlying genetic defect rather than just alleviating symptoms. Researchers are excited about this approach because it offers the potential for a more effective, long-term solution for patients with GAN.

Research has shown that the scAAV9/JeT-GAN treatment may help with Giant Axonal Neuropathy (GAN). Previously, administering this gene therapy into the spinal canal slowed the disease and improved movement for some patients. This treatment uses a safe virus to deliver a healthy version of the gigaxonin gene, which is faulty in GAN. Although some side effects occurred, patients generally tolerated the therapy well. In this trial, participants will receive a single injection of scAAV9/JeT-GAN directly into the vagus nerve to test a new method aimed at addressing more symptoms related to the autonomic nervous system.¹²³⁴⁶