Our Pipeline
Global commercial rights for all programs fully owned by Spell Therapeutics.
| Program | Indication | Deficient Enzyme | Major Tissues | Platform | Est. US Patients |
|---|---|---|---|---|---|
| Spell-101 | Pompe Disease | Acid alpha-glucosidase (GAA) | Skeletal muscle, heart, lung | AAV-mediated ERT | ~18,000 |
| Spell-102 | Fabry Disease | Alpha-galactosidase A (α-GAL) | Heart, kidney, nervous system | Classical ERT | ~29,000 |
Pompe disease is caused by a deficiency of acid alpha-glucosidase (GAA), leading to progressive damage in skeletal muscles, heart, and lungs.
Our Spell-101 program uses an AAV vector carrying the IGF2-GAA fusion gene for liver-depot gene therapy — a potential one-time, curative treatment.
The current market leader (Sanofi's Lumizyme/Nexviazyme franchise) generated €316 million in H1 2025, yet patients still face limited efficacy in muscle and lung — precisely where our technology excels.
US Market: ~$1.2B | Global: ~$1.9B
Apply for Fast Track and Rare Disease Designation to accelerate regulatory review.
In vivo ERT studies in Pompe mice at Duke. Supported by NIH R01 grant (~$2.7M over 5 years).
IND-enabling toxicology studies, FDA filing, GMP vector manufacturing, Phase 1/2 trial initiation.
Fabry disease results from a deficiency of alpha-galactosidase A (α-GAL), causing progressive damage to the heart, kidneys, and nervous system.
Renal failure remains the leading cause of death in Fabry patients, yet no approved ERT effectively treats kidney involvement. Spell-102 aims to be the first ERT to meaningfully address Fabry nephropathy — an $800M+ underserved segment.
Combined Fabry ERT market leaders (Fabrazyme, Replagal, Elfabrio) generated over $1.7 billion in 2024.
US Market: ~$1.4B | Global: ~$2.4B
FDA meeting for Fast Track / Rare Disease Designation.
Efficacy studies in Fabry mice at Duke. GLP-grade enzyme manufacturing with ExcellGene (Switzerland).
IND-enabling studies, FDA filing, GMP enzyme manufacturing, Phase 1/2 clinical trial.
Learn about the market opportunity behind these programs.