Duke University Startup
A best-in-class fusion enzyme platform that dramatically improves drug delivery to hard-to-treat tissues — at a fraction of today's cost.
Our Mission
Millions of patients with lysosomal storage diseases face progressive disability despite existing treatments. Current enzyme replacement therapies struggle to reach key tissues like skeletal muscle, kidney, heart, and lung — and cost families up to $750,000 per year.
Spell Therapeutics was founded to change that. Our proprietary IGF2-based fusion enzyme technology overcomes the fundamental limitations of today's therapies, delivering more drug to the tissues that need it most, at dramatically lower cost.
Why Spell
Built on 25+ years of translational research in gene therapy and enzyme replacement therapy by Prof. Baodong Sun at Duke University.
Our technology applies to both classical ERT (today's $10B+ market) and AAV-mediated in vivo ERT (the future of curative gene therapy).
Lead programs in Pompe disease (Spell-101) and Fabry disease (Spell-102), with global commercial rights fully owned by Spell Therapeutics.
Pipeline
AAV-mediated liver-depot gene therapy with IGF2-GAA fusion vector. A potential one-time, curative treatment.
~18,000 estimated US patients
First ERT to meaningfully address Fabry kidney nephropathy — an $800M+ underserved market segment.
~29,000 estimated US patients
Learn More
Get in touch to learn more about our technology, pipeline, and how we're transforming enzyme replacement therapy.
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