Targeted Genetics Corp. was granted orphan drug status for anyformulation of adeno-associated virus (AAV) vector for cysticfibrosis (CF).
The Seattle company received approval from the Recombinant DNAAdvisory Committee of the National Institutes of Health for clinicaltesting of its gene therapy approach in September. The company filedan investigational new drug application in early January, and expectsto start trials in May or June, said Alberta Garvin, the company'smanager, corporate communications.
Orphan drug status, given to diseases affecting fewer than 200,000people in the U.S., allows seven-year exclusivity to the first sponsorof an orphan drug granted marketing approval.
Targeted Genetics' trial will involve 16 patients with CF and mildlung disease at Johns Hopkins Children's Center in Baltimore. Theywill be treated during an 18-day stay at the center, then followed oneyear.
Being granted orphan status "solidifies our position with regard toAAV as it relates to CF," Garvin said.
The company's AAV vector is designed to deliver a normal copy ofthe CF transmembrane conductance regulator gene to lung cells. ThePhase I trial is designed to assess the safety of the product and test itsexpression in the cells of the nose and lungs. _ Jim Shrine
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