Focused On Rare Illnesses, North Carolina Startup Aceragen Goes After Farber Disease

A new North Carolina-based biopharmaceutical company with a focus on rare illnesses hopes to be the first to find a treatment for Farber disease. This extremely rare genetic disorder results in the buildup of a material in the body called ceramide that can inflame and damage many different tissues. Joints, connective tissue, the lungs and

Aceragen Launches with Acquisition of Enzyvant’s Investigational Therapy for Farber Disease and $35 Million Product Financing with NovaQuest

Acquires worldwide rights to RVT-801, a novel enzyme replacement therapy for treatment of Farber disease NovaQuest investment to fund product and clinical development Protocol for potential single registration study reviewed with FDA and EMA Program has been granted rare pediatric disease status, fast track and orphan designations, and is eligible for a priority review voucher