AVI-created technology to begin first human trial
Sunday, December 10th, 2006AVI-created technology to begin first human trial
A potential treatment for Duchenne muscular dystrophy using a technology developed by AVI BioPharma Inc. is set to begin its first human clinical trial.
British researchers at Imperial College and Hammersmith Hospital in London are running the study with funding from the British government. Portland-based AVI is supplying the drug.
An AVI spokesman on Thursday said that if results from the preliminary study are positive, the company would file an application with the Food and Drug Administration to begin expanded clinical trials in the U.S. in 2007.
Children born with the incurable disease appear healthy at birth, but by age five, severe muscle deterioration begins. By age 12, many children can no longer walk. The disease is caused by mutations that stop muscle cells from producing a vital protein.
AVI has developed synthetic polymers that bind to specific gene sequences. Australian researchers showed that the polymers can be used to trick the cell’s protein-making machinery to skip mutated gene sequences and resume output of functional muscle protein. The strategy has worked in a mouse model of muscular dystrophy and in test-tube studies using human muscle cells.
A British government gene-therapy regulatory panel in September gave clearance for the study.
– Joe Rojas-Burke
