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Thymic histology goes digital
Cambridge, MA—Today Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, has announced the formation of a partnership with Visikol, a contract research organization focused on digital pathology, drug discovery and advanced imaging tools. Under the terms of the agreement, 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.
“Today’s announcement of the partnership with Visikol is a key component to enable development of a medicine to treat complete DiGeorge Anomaly,” said Dr. Alvin Shih, Chief Executive Officer of Enzyvant. “Rare disease drug development requires collaboration and creativity, and we are pleased to commence work with Visikol to incorporate an innovative digital pathology approach into the RVT-802 development process.”
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). RVT-802 is investigational and not approved for commercial use by the FDA or other health regulators at this time. RVT-802 was initially developed by Dr. M. Louise Markert, Professor of Pediatrics at Duke University. Enzyvant partnered with Markert and Duke University in 2016.
Complete DiGeorge Anomaly is a rare disease affecting approximately 1 in 300,000 infants, or roughly 10-20 infants born each year in the US. Children with cDGA are born without a thymus gland, resulting in severe immunodeficiency due to the inability to produce normally functioning T cells, which defend against infection and regulate essential processes in the immune system. cDGA is uniformly fatal if untreated, with death typically occurring in the first 24 months of life due to susceptibility to infection.
Enzyvant plans to initiate the rolling Biologics License Application submission for RVT-802 to the US Food and Drug Administration (FDA) later this year. RVT-802 has received Breakthrough Therapy designation, Regenerative Medicine Advanced Therapy designation, rare pediatric disease designation, and orphan drug designation from the FDA.
“We are excited to work with Enzyvant as they develop RVT-802 into an approvable therapy with the requisite data and CMC procedures,” said Dr. Thomas Villani, Chief Science Officer at Visikol. “We are especially keen to work on this project because it is an ideal opportunity to apply our digital imaging technologies and machine learning capabilities to develop a potentially life-saving therapy.”