According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics, Inc. has recently announced that its experimental gene therapy ABO-202 has earned Fast Track designation from the US Food and Drug Administration (FDA). ABO-202 is in development as a treatment for CLN1 disease, also known as infantile Batten disease. The company is currently in the midst of designing a phase 1/2 clinical trial to test the investigational gene therapy.
About Infantile Batten Disease
Infantile Batten disease is a nervous system disorder that tends to appear very early in the patient’s life, often within the first year. This neurodegenerative disease is caused by mutations which affect the CLN1 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 no disease altering treatment for infantile Batten disease. Most treatment is supportive, and the disease is ultimately lethal; lifespan is often less than eight years. There is a dire need for more effective therapies to improve survival times and outcomes for patients with infantile Batten disease. To learn more about Batten disease, click here.
About Fast Track Designation
Fast Track designation is a special process that is meant to speed up the development of drugs that have the potential to treat severe, life-threatening diseases and/or satisfy currently unmet medical needs. With the ultimate goal of a faster development cycle, the designation allows the recipient company to engage in more frequent communication and collaboration with the FDA. Recipients may also be eligible for Rolling Review, Accelerated Approval, and Priority Review if other relevant criteria are met.
ABO-202 has also earned Orphan drug status in both the US and EU and well as Rare Pediatric Disease designation from the FDA. The drug utilizes an AAV9 viral vector system that is intended to deliver a functional copy of the affected gene in just a single dose. Animal models have demonstrated encouraging results so far.