A single treatment with the potential
for long-lasting results
RGX-381 is our product candidate for the treatment of ocular manifestations of late-infantile neuronal ceroid lipofuscinosis Type 2 (CLN2 disease), a form of Batten disease.
RGX-381 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the retina.
CLN2 disease is a rare, recessive genetic disease caused by a deficiency in TPP1, an enzyme required for the breakdown of specific peptides in the lysosomes of cells.
Disease onset is generally between two to four years of age with initial features of recurrent seizures (epilepsy), language delay, and difficulty coordinating movements (ataxia). Following onset, the disease progresses rapidly, resulting in loss of language and motor functions, seizures, cognitive decline, and premature death by mid-childhood. Ocular manifestations typically begin at age 4, with rapid loss of vision progressing to blindness over the next several years. Children with atypical CLN2 disease may begin to lose vision at a later age.
There is currently no available treatment for the vision loss in CLN2 disease. We believe that delivery of the gene encoding for the TPP1 enzyme via a one-time administration of RGX-381 could provide a durable source of TPP1 activity in the retina, thereby potentially preventing ocular manifestations of the disease.
We expect to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) in the second half of 2020 for RGX-381 to enable initiation of a first-in-human clinical trial for children with CLN2 disease. We have two on-going natural history studies that are enrolling patients. Please visit clinicaltrials.gov for more information.