Over 300 million people worldwide suffer from rare diseases, many with no treatments. Traditional drug discovery ignores these conditions—too niche, too risky. But what if you could test therapies on human muscle engineered to mirror exact disease biology?
Myomaker Bio’s platform does just that. We recently partnered with a biotech to screen 1,200 FDA-approved drugs for ALS using our neuromuscular junction (NMJ) models. Within weeks, we identified a repurposed cardiac drug that improved motor neuron survival by 40%—a breakthrough missed by animal studies.
For orphan diseases, time isn’t just money; it’s lives. Our models cut preclinical timelines by 50%, giving hope to underserved patients. Because every disease deserves a cure.
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