Abeona Therapeutics Inc (NASDAQ: ABEO) is an integrated player in gene therapy. It announced the MRI (Magnetic Resonance Imaging) data from its Phase 1 /2 Transhper A clinical study indicating an increase of grey matter in patients at 24 months compared to the patient without treatment.
New data published: As per the company, MRI (Magnetic Resonance Imaging) data indicates ABO-102 led to an uptick in grey matter, amygdala volumes, and corpus callosum in the brain of the young patients who had undertaken the trial. The increase was observed after 24 months of treatment compared to the patients without treatment. As per the company, the brain volume loss in a young patient (Children) with MPS III A is primarily associated with a long-term cognitive and physical disability. They further stated that new MRI data indicated the potential of AB0-102 to increase brain grey matter, corpus callosum, and amygdala volumes. The successful commercialization of drugs is the key to success for the company and the medical field.
The Transpher A depicted two endpoints- primary endpoints consist of neurodevelopment and safety. While secondary endpoints look after brain volume, quality of life, enzyme activity in (cerebrospinal fluid) CSF and plasma, brain volume, and many more.
About Transpher A: It is an ongoing, two-year, Phase 1/2 global clinical trial assessing ABO-102 to treat patients with Sanfilippo syndrome type A (MPS IIIA). The study is primarily meant for patients from birth until two years of age or patients with over two years of cognitive development.
ABO-102: is unique gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA). The therapy is designed to meet the concern of the underlying SGSH enzyme deficiency. The SGSH enzyme deficiency is responsible for the abnormal accumulation of glycosaminoglycans in the brain and the body. It results in cell damage and physical decline, and neurodevelopmental.
Sanfilippo Syndrome Type A (MPS IIIA): is a fatal lysosomal storage disease with hardly any treatment that affects the CNS. As per the company, the children with MPS IIIA are those with cognitive decline and behavioral abnormalities. MPS IIIA is primarily caused by genetic mutations that result in a deficiency in the SGSH enzyme.