Patient Focused. Results Driven.
Transformative therapies for rare diseases with high unmet needs.
Innovative partnerships that create new opportunities in the rare disease field.
On our commitment to the patients, families, and communities we serve.
Complete DiGeorge Anomaly
Children with complete DiGeorge Anomaly are born without a thymus gland, resulting in primary immunodeficiency. The disease is almost uniformly fatal within the first 24 months of life if untreated.
Farber disease is an ultra-rare lysosomal storage disease caused by a mutation in both alleles of the ASAH1 gene, resulting in the deficiency of the lysosomal enzyme acid ceramidase.
July 28, 2018
Enzyvant announced today the appointments of Andrew Dawson to head human resources and Alex Tracy, PhD to lead development and manufacturing, enhancing the overall capabilities of the company
July 19, 2018
Enzyvant today announced that it has formed a collaboration with PerkinElmer Genomics, a global genomic testing company, to screen for mutations in the ASAH1 gene.
July 9, 2018
Enzyvant, announced today that it has initiated its rolling submission of a Biologics License Application (BLA) for RVT-802 to the U.S. Food and Drug Administration (FDA).