Abeona Therapeutics Inc. (ABEO) stock gains by 6.63% in the current market trading session. Abeona Therapeutics Inc. is a pharmaceutical firm in the early stages of development that is working on gene and cell treatments for severe illnesses. EB-101, an experimental autologous, gene-corrected cell treatment for recessive dystrophic epidermolysis bullosa in Phase 3 development, is one of Abeona’s clinical initiatives.
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ABO-102 enhanced grey matter, corpus callosum, and amygdala proportions in the brain in three young kids with Sanfilippo Syndrome Type A (MPS IIIA) at 24 months. This has been observed in the magnetic resonance imaging (MRI) findings from the Phase 1/2 Transpher A clinical trial released today by Abeona Therapeutics. The novel findings are reported at the 16th International Symposium on MPS and Related Diseases in an oral presentation.
Sanfilippo syndrome type A (MPS IIIA) is a rare lysosomal storage disorder with no authorized therapy that predominantly affects the central nervous system. ABO-102 is new gene therapy in Phase 1/2 development for Sanfilippo syndrome type A (MPS IIIA) (CNS).
Vishwas Seshadri, Head of Research & Clinical Development of Abeona stated,
In children with MPS IIIA, brain volume reduction is common, and it’s linked to cognitive and physical impairment for a longer period of time. Grey matter is necessary for cognitive growth, the corpus callosum is essential for motor function, and the amygdala is vital for fear learning and social/emotional growth. The new MRI data reveals that ABO-102 has the ability to enhance brain grey matter, corpus callosum, and amygdala volumes, which is compatible with the Transpher A study’s earlier findings of neurocognitive developmental preservation in these three young patients.
The major objectives of the Transpher A research are neurodevelopment and protection. Brain volume, behavior assessments, life quality, enzyme activity in CSF and plasma, heparan sulfate levels in CSF, plasma, and urine, and liver volume are all secondary endpoints.
The Transpher A Study (NCT02716246) is a two-year open-label, dose-escalation Phase 1/2 global clinical study evaluating ABO-102 for the therapy of Sanfilippo syndrome type A individuals (MPS IIIA). Patients from birth to two years of age, or those older than two years with a cognitive developmental quotient of 60% or above, are eligible for the research, which is also known as ABT-001.