According to a story from apnews.com, the gene and cell therapy company Abeona Therapeutics, Inc., has recently announced that the US Food and Drug Administration (FDA) has given the company’s Investigational New Drug application approval. This approval means that the company is allowed to proceed with phase 1/2 clinical trials that will test ABO-202, an experimental, single dose gene therapy that is being developed to treat CLN1 disease, also known as the infantile form of Batten disease.
About CLN1 Disease
CLN1 disease, or infantile Batten disease is a nervous system disorder that tends to appear very early in the life of an affected child. This neurodegenerative disease is caused by mutations which affect the CLN1/PPT1 gene. Girls tend to display symptoms later than boys, but their disease tends to progress more rapidly. This disease is classified as a form of neuronal ceroid lipofuscionsis. 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 currently no available disease modifying treatment for CLN1 disease. 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 CLN1 disease. To learn more about CLN1 disease, click here.
About ABO-202
Preclinical study has suggested that ABO-202 should be well tolerated and should deliver significant clinical benefit to patients. Like many similar forms of gene therapy in development, this therapy uses and AAV vector in order to deliver a corrected copy of the affected gene to both the nervous system and organs. Following this process, it is expected that the cells will be able to produce the relevant PPT1 enzyme on their own as unaffected individuals do.
In an animal model of CLN1 disease, ABO-202 was able to provide extended survival and improve nerve function. These studies also suggested that combined intrathecal (into the spinal canal) and intravenous administration of the therapy was more effective than using a single administration route. This experimental treatment has the potential to dramatically change the treatment landscape for this disease.
