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Patient Focused. Results Driven.

OUR MISSION

DEVELOP

Transformative therapies for rare diseases with high unmet needs.

PURSUE

Innovative partnerships that create new opportunities in the rare disease field.

DELIVER

On our commitment to the patients, families, and communities we serve.

OUR PIPELINE

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RVT-802

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.

RVT-801

Farber Disease

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.

February 28, 2018

Re:GenerationHope raises awareness and funds for the complete DiGeorge Anomaly

January 8-10, 2018

Coinciding with the annual J.P. Morgan Healthcare Conference

October 16, 2017

Global study aims to define the natural course of Farber disease

Contact us for more information.