Curing the Disease, Not the Symptom

Mutations that encode for nonsense codons result in truncated proteins that don’t function properly, leading to many different diseases. PTC Therapeutics, a startup in South Plainfield, NJ, has developed a new drug that allows the cell to skip over these nonsense codons to produce functional proteins, thereby treating the disease's cause, not just symptoms. "Since the drug treats the underlying gene-expression problem, it is applicable to a few thousand diseases," says cofounder Allan Jacobson in a story in Tech Review. Early clinical tests on Duchenne muscular dystrophy and cystic fibrosis have been successful, and larger trials are on the horizon.