Science
Advancing Novel Treatments for Patients with Rare Neuromuscular Disease
Duchenne muscular dystrophy (DMD) is a severe genetic disorder that affects one in 4000 boys worldwide. Current treatments for DMD are costly and offer limited efficacy, safety and durability, failing to significantly alter the disease's progression and associated mortality.
Current Status of SH-0322 Development
StrongHolt Therapeutics is developing SH-0322, a next-generation gene therapy designed to overcome the limitations of existing treatments. Our innovative approach ensures tissue-specific delivery, stability under mechanical load, and a minimized immune response. Preclinical testing has shown that SH-0322 can normalize skeletal muscle function and the cardiac phenotype of DMD with a single injection, providing a safer and more effective alternative to current therapies.