The paradigmatic gene therapy technique involves slipping new genes into cells on the backs of modified viruses, although other methods exist. Wilmington, Del.-based Orphagenix is championing one of these lesser-known approaches against some lesser-known diseases.
"Many of them are life-threatening and there are no other cures," says Orphagenix president and CEO, Michael Herr. That, and their rarity, will let the company benefit from federal incentives designed to encourage drug development for such conditions, he adds. A key incentive is the Orphan Drug Act of 1983.
Orphagenix is using a family of patented techniques designed to correct point mutations, developed by researchers led by molecular biologist Eric Kmiec of the University of Delaware in Newark. The basic approach, called targeted gene alteration, is to introduce into cells oligonucleotides that ...
