FIRST GENE THERAPY TRIAL FOR
DUCHENNE MD MOVES AHEAD

TUCSON, Ariz., Dec. 12, 2006 — The first U.S. human gene therapy trial targeting Duchenne Muscular Dystrophy (DMD), launched in March at Columbus Children’s Hospital (Ohio), will soon move to a second stage, thanks to an unprecedented $2.5 million grant from the Muscular Dystrophy Association (MDA) and matching funds from Asklepios BioPharmaceutical Inc. (AskBio) of Chapel Hill, N.C., the Association announced today.

In this second stage, AskBio will conduct further large animal studies in canine and non-human primate DMD models to test the safety and effectiveness of systemic delivery of Biostrophin, AskBio’s lead gene therapy candidate for DMD.

The first stage of the project, now nearing completion, has been testing the safety of administering Biostrophin directly into a single muscle in six boys with the disease. Following years of laboratory experiments required by the Food and Drug Administration demonstrating that the mini-gene was unlikely to harm and could ultimately benefit patients with DMD, the team was given a green light to proceed with the human trial on March 3. Results of that trial are expected in mid-2007.

“The next step of this project is important to establish that we can deliver the dystrophin gene to multiple muscles at once,” said Sharon Hesterlee, vice president of Translational Research at MDA. Reaching multiple muscles will be necessary to achieve functional improvements in patients.

DMD is an X-linked genetic disease primarily affecting males that begins in early childhood, causes progressive loss of muscle strength and bulk, and usually leads to death in the 20s from respiratory or cardiac muscle failure. In DMD patients, no dystrophin protein is produced. Biostrophin consists of a genetically engineered replacement dystrophin gene (mini-gene) delivered with Biological Nano Particles (BNPs) derived from rAAV vectors. The dystrophin gene is among the largest known, and miniaturizing it to enable delivery to a patient’s muscles while retaining its therapeutic abilities has been one of the greatest challenges to the field of gene delivery.

The mini-gene used in this research was developed by Xiao Xiao and the BNP technology platform was developed by Jude Samulski, both researchers at the University of North Carolina at Chapel Hill. Xiao and Samulski formed AskBio in 2003.

The $2.5 million grant to AskBio is the largest MDA has ever awarded to a for-profit company. MDA provided $1.6 million for the first phase of the trial.

“MDA has a long-standing commitment of supporting innovative muscular dystrophy research, and we are pleased to be selected for this grant award,” said Sheila Mikhail, chief executive officer of AskBio. “We expect the results of this research to be a significant step in evaluating Biostrophin gene therapy as a potential viable advancement in care for the DMD community.”

MDA (www.mda.org) is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education.

Asklepios Biopharmaceutical, Inc. (www.askbio.com) is a private development-stage biotechnology company engaged in the development of novel gene therapy-mediated protein therapies using its proprietary Biological Nano Particle (BNP) technology platform. AskBio’s Biostrophin project is directly supported by translational grants from MDA.