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The phrase comes from "The Orphan Drug Act," the law Congress passed in 1983 to encourage drug makers to develop treatments for relatively rare, but sometimes deadly, diseases the big drug makers were overlooking.Two of the diseases Orphagenix is pursuing are sickle cell anemia and spinal muscular atrophy, says Michael Herr, who joined the company this year as president and chief executive officer.Sickle cell anemia is an inherited blood disorder that affects about 70,000 people in the United States, mostly blacks.

Spinal muscular atrophy is a motor-neuron disorder that affects about 1 in 6,000 newborns.

It's the biggest genetic cause of death among children younger than age 2, according to Families of Spinal Muscular Atrophy, an advocacy group.Herr, who came to Orphagenix from the University City Science Center, a Philadelphia incubator for early-stage life science and technology companies, says both diseases are well suited to the gene-replacement process Kmiec and Parekh-Olmedo developed.The promising process is what attracted Herr to join the company from the Science Center, where he helped biotech start-ups develop business plans and find funding."I was blown away b...

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