Statement on Gene Therapy

The FDA reported on July 26 the death of a participant in a rheumatoid arthritis gene therapy trial using an adeno-associated virus (AAV) carrier.  It’s not known if the death was related to the gene therapy trial. 

The Muscular Dystrophy Association is currently supporting a clinical trial using an AAV carrier containing the dystrophin gene in boys with Duchenne muscular dystrophy.  Six patients have received the gene carried by AAV.  The boys are being closely monitored and no adverse effects have been associated with this MDA-sponsored trial. 

MDA is following this incident and working closely with investigators in the field to ensure the safest possible conditions for clinical trial participants.

For more information about the rheumatoid arthritis clinical trial see the FDA statement at www.fda.gov/bbs/topics/NEWS/2007/NEW01672.html