According to a story from BioSpace, the biotechnology company REGENXBIO, Inc. recently announced that its experimental gene therapy candidate called RGX-181 has been granted Rare Pediatric Disease designation from the US Food and Drug Administration (FDA). This gene therapy is intended to be a single use treatment for the late infantile form of Batten disease which is linked to mutations affecting the CLN2 gene.
Batten disease is a nervous system disorder that tends to appear between the ages of five and ten years. This neurodegenerative disease is caused by mutations which affect the CLN3 gene. Girls tend to display symptoms later than boys, but their disease tends to progress more rapidly. Diagnosis is often difficult. Symptoms include seizures, vision problems, repetitive speech, learning regression or delays, scoliosis, decreased muscle and body fat, changes to personality and behavior, poor coordination, and speech loss. Symptoms generally progress over time. There is only one drug for Batten disease that can slow progression, but it is only effective in certain cases. Most treatment is supportive, and the disease is ultimately lethal. There is a dire need for more effective therapies to improve survival times and outcomes for patients with Batten disease. To learn more about Batten disease, click here.
About Rare Pediatric Disease Designation
RGX-181 had previously earned Orphan Drug designation from the FDA. Rare Pediatric Disease designation is typically reserved for therapies intended to treat a disease that mostly affects people under 18 years of age and is considered rare, meaning that it affects less than 200,000 people in the US. To earn this designation, a potential therapy must address a currently unmet medical need or offer substantial advantages in safety or effectiveness in comparison to currently available treatments.
There is a great need for better disease modifying treatments for all forms of Batten disease, so REGENXBIO’s development of a single use gene therapy would be a dramatic improvement for a disease that is currently considered fatal. RGX-181 uses a proprietary AAV9 vector delivery system called NAV. This system will deliver a corrected gene into the nervous system that should allow patients to start producing their own TPP1, a critical enzyme that is deficient in late infantile Batten disease patients.
