Visikol will collaborate with Enzyvant and Duke University to develop a novel assay for thymic histology samples. The purpose of this investigational assay is to provide a quantitative technique for assessment of RVT-802 and is an important step in ongoing efforts to support an FDA filing.
RVT-802 is an investigational one-time thymic tissue-based regenerative therapy designed to reconstitute the immune system for the treatment of primary immune deficiency resulting from congenital athymia associated with complete DiGeorge Anomaly (cDGA).
Children with cDGA are born without a thymus gland, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells.
cDGA is rare, affecting approximately 1 in 300,000 infants, and is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
Enzyvant is focused on developing innovative treatments for patients with rare diseases.
The company is currently working on RVT-802, an investigational tissue-based therapy under study for the treatment of primary immunodeficiency associated with complete DiGeorge Anomaly and RVT-801, an investigational enzyme replacement therapy for the treatment of Farber disease.
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