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First Gene Therapy Human Trial For Muscular Dystrophy In Progress

ao told Samulski he wanted to put his new minidystrophin gene into an AAV vector and test it.

"He wanted to know if UNC could make the virus, and that's when I told him that we were using this virus for Canavan's disease and for other efforts. We had started improving the vectors, and we had developed some new ones that we thought were better for muscle."

Xiao and Samulski put their projects together and formed Asklepios BioPharmaceutical Inc. (AskBio) in 2003. Along with the rights granted by UNC to Samulski's vector technology, AskBio acquired the intellectual property rights to Xiao's uniquely miniaturized dystrophin gene.

In July 2004, the Muscular Dystrophy Association (MDA) awarded $1.6 million to AskBio to develop gene therapy strategies for DMD.

Following extensive laboratory toxicity experiments required by the U.S. Food and Drug Administration demonstrating that minidystrophin gene transfer was unlikely to harm and could ultimately benefit muscles affected by DMD, approval was granted March 3, 2006, to proceed with the human trial.

"After years of encouraging pre-clinical results, I'm excited that AskBio will help bring this promising new therapy into the clinic, and look forward with a great deal of optimism to offering this initial step toward hope for the DMD community," Samulski said.

--Eurekalert
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