PPL Reports Progress With Transgenic AAT

PPL Therapeutics of the UK has completed viral and prion removal studies of its lead therapy for cystic fibrosis, alpha1 antitrypsin, and is now in a position to begin Phase I clinical trials, according to Julie Edgar, project manager for AAT at PPL, at the 10th Annual North American Cystic Fibrosis Conference (see adjacent story).

AAT is a protein which functions as an inhibitor of the action of elastase. Several companies have looked at the feasibility of using elastase inhibitors to treat CF and chronic bronchitis by preventing the destructive effects on lung tissue of leukocytes responding to the frequent bacterial infections seen in CF patients.

PPL produces bulk AAT in the milk of transgenic sheep, and has a cost-effective, consistent supply that plasma-derived AAT would be unable to achieve. The new data are important for the further development of PPL's AAT, because of concerns over the relationship between scrapie, a prion infection in sheep, and Creutzfeld-Jacob disease in humans.