Sickle cell anemia and spinal muscular atrophy are genetic diseases that are serious, but relatively uncommon.

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.

[New company President and CEO Michael] 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 two researchers found a way to introduce a minuscule fragment of DNA into a cell where a portion of the DNA is damaged.

The introduced DNA binds to the damaged portion of the DNA, triggering the cell to heal itself. What is introduced into the cell is eventually released out of the cell.

The article credits the Orphan Drug Act, passed in 1983, to Orphagenix’s interest in SMA. The law provides a host of incentives, from a streamlined review process to a tax credit that allows companies to recoup as much as 50 percent of their research-and-development costs.

You can read the entire article by clicking here.