About Us
Building on Duke University's decades of leadership in lysosomal storage disease research to deliver transformative therapies to patients worldwide.
Our Company
Spell Therapeutics is developing next-generation enzyme replacement therapies (ERTs) for patients with lysosomal storage diseases (LSDs). We are building on a breakthrough invention by Prof. Baodong Sun, a world-renowned expert with over 25 years of experience at Duke in developing translatable therapies for rare genetic diseases.
Prof. Sun's extensive track record includes pioneering publications in gene therapy and ERT, numerous patent-protected inventions, and significant research funding from the NIH and major pharmaceutical partners.
The Problem
Lysosomal storage diseases are a group of over 70 progressive, debilitating inherited metabolic disorders caused by deficiencies of lysosomal enzymes. They present early in childhood or adulthood, and the most severe forms can be fatal within the first year of life.
Individually rare, but collectively affecting approximately 1 in 5,000 live births worldwide. Common LSDs include Pompe disease, Fabry disease, Gaucher disease, MPS, and Niemann–Pick disease.
Commonly affected organs include the heart, skeletal muscle, kidney, lung, liver, spleen, skeletal system, and brain.
Current ERTs rely on the M6PR pathway, which delivers enzymes poorly to skeletal muscle, kidney, heart valves, and lung.
Biweekly IV infusions cost $300,000–$750,000/patient/year, causing anti-drug antibodies and significant infusion burden.
Even with ongoing ERT, many patients continue to experience progressive decline and disability.
Our Heritage
Duke University faculty led the discovery, translational science, and clinical development of the first approved ERT for Pompe disease (Myozyme/Lumizyme), which achieved $1.43 billion in global sales in 2022.
Duke researchers have also pioneered AAV gene therapy clinical trials for Pompe disease. Prof. Sun recently received an NIH R01 grant (~$2.7 million over 5 years) to develop clinical-candidate AAV vectors for Pompe disease.