AAV2-ELP1

AAV2-ELP1 is an investigational adeno-associated virus (AAV) gene therapy developed by Tikun Therapeutics for the treatment of Familial Dysautonomia (FD). The therapy utilizes an AAV2 vector to deliver a functional version of the ELP1 gene (Elongator complex protein 1, formerly known as IKBKAP) to target tissues. FD is a rare neurodegenerative genetic disorder caused by mutations in the ELP1 gene, which leads to a deficiency of the ELP1 protein and subsequent degeneration of sensory and autonomic neurons. AAV2-ELP1 is specifically designed to address the progressive optic neuropathy and vision loss associated with the disease by restoring ELP1 expression in retinal ganglion cells.

Other names

AAV2-ELP1 gene therapyAAV-2-ELP1 gene therapyAAV 2-ELP1 gene therapy