Gene Therapy For Cystic Fibrosis In Ph II

Targeted Genetics and Stanford Medical Center in the USA have started aPhase II trial of TG's gene therapy product for cystic fibrosis, tgAAV-CFTR. The company recently reported positive Phase I data with the product, demonstrating that it is safe, provides efficient gene transfer and long-term gene persistence (up to 70 days).

The Phase II trial will test the safety and efficacy of TG's aerosol-formulated, adeno-associated virus-vector product in preventing the occurrence of sinusitis in patients with CF. 50 patients will receive tgAAV-CFTR in one sinus, and placebo in the other.