Exegenesis Bio, a leading provider of epigenetics experimental and analytical services headquartered in Hangzhou, has announced that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its proprietary gene therapy, EXG110, which is indicated for the treatment of Fabry disease. This designation highlights the potential of EXG110 in addressing a rare and serious medical condition.
Understanding Fabry Disease and Its Impact
Fabry disease, also known as alpha-galactosidase-A deficiency, is an inherited neurological disorder characterized by the enzyme alpha-galactosidase-A’s inability to efficiently break down lipids in the body. The mutated gene results in the accumulation of lipids to harmful levels within the body’s autonomic nervous system, affecting vital functions such as breathing, cardiovascular health, vision, and kidney function. The traditional treatment for Fabry disease involves enzyme replacement therapy, which requires patients to undergo an infusion of agarase every two weeks for the rest of their lives, underscoring the need for more sustainable therapeutic solutions.
EXG110: A Novel Gene Therapy Approach
EXG110, a groundbreaking adeno-associated viruses (AAV)-based gene therapy, is anticipated to offer long-lasting therapeutic effects through a single intravenous injection. This innovative approach to treatment has the potential to significantly improve the quality of life for both adults and children suffering from Fabry disease by reducing the frequency and invasiveness of treatment.
Investigator-Initiated Trial and Promising Safety Profile
The drug initiated an Investigator-Initiated Trial (ITT) in August of this year, marking a significant milestone in its clinical development. Early results have demonstrated a good safety profile in the first subject, indicating the promising potential of EXG110 as a treatment for Fabry disease patients.-Fineline Info & Tech
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