DMD Research: Exon Skipping Goes Systemic

AVI BioPharma of Portland, Ore., has started the systemic (through the blood) delivery phase of its clinical trial of AVI4658 in Duchenne muscular dystrophy (DMD). The trial is being conducted in the United Kingdom.

In January, the company announced that this laboratory-engineered molecule was safe and well tolerated when injected directly into a foot muscle in boys with DMD. More importantly, the molecule led to production of dystrophin in all trial participants. (Dystrophin is the necessary muscle protein missing in DMD.)(See UK trial shows robust response.)

AVI BioPharma says the systemic-delivery phase of the trial now under way will test the safety and efficacy of administering AVI4658 intravenously into 16 boys with DMD. Systemic delivery is expected to reach several muscles and potentially could improve strength and function.

The 12-week study is being conducted in London and Newcastle Upon Tyne, United Kingdom. Francesco Muntoni at Imperial College London, who has MDA support to conduct research in another muscle disease, is the principal investigator. Support for this trial comes from AVI BioPharma and the British Medical Research Council.

Exon Skipping

AVI4658, a so-called antisense compound, is designed to cause muscle cells to skip over an error in the DNA for the dystrophin protein, utilizing a strategy called "exon skipping," in which specific exons (sections) of genetic instructions are blocked and the surrounding instructions preserved.

In its Feb. 19 press release, AVI said the earlier results and preclinical research suggest that "by skipping [exon 51], a truncated but functional form of the dystrophin protein is produced to amelioerate the disease process, potentially prolonging and improving the quality of life in these patients."

Disease: 
Link: 
GUID: 
1 003
Time Stamp: 
1 257 949 961
Thumbnail: 
http://quest.mda.org/sites/default/files/imagecache/mda_org_frontpage100x75/7%20LeadArt.jpg
Node Type: 
Quest News