Enzyvant to Develop Novel Biologic Therapy for Complete DiGeorge Syndrome

Monday, January 09, 2017

Enzyvant, a biopharmaceutical company focused on developing innovative treatments for patients with rare diseases, today announced entering into an exclusive worldwide licensing agreement with Duke University to develop RVT-802, a tissue-based therapy for the treatment of complete DiGeorge Syndrome (cDGS), a rare and fatal congenital immunodeficiency disease. In the absence of treatment, children born with cDGS typically succumb to infections within the first two years of life due to their severe immunodeficiency.

"Patients with complete DiGeorge Syndrome lack effective treatment options, but this novel program offers the hope of a life-saving solution," said Chief Executive Officer Dr. Alvin Shih. "It is our responsibility to ensure that the children with this disease who need this treatment are able to receive it."

Under the terms of the agreement, data from Duke's experience will be used to support submission of a Biologics License Application (BLA) with the Food and Drug Administration (FDA) for approval. RVT-802 expands Enzyvant's pipeline of potentially transformative biologic therapies for rare genetic diseases with high unmet need.

Dr. M. Louise Markert, Professor of Pediatrics at Duke University, has led research on the treatment of immunodeficiency in patients with cDGS. The findings of Dr. Markert and her research team have been published in the New England Journal of Medicine as well as numerous other peer-reviewed scientific journals and clinical publications. "This is an encouraging development for the patients and parents confronting this disease," said Dr. Markert. "I look forward to working with Enzyvant to make this life-saving therapy more available to babies who so desperately need it."