Asklepios, a startup biotechnology firm focused on delivery of gene therapies, has received a $1.6 million grant from the Muscular Dystrophy Association to pursue possible gene therapies to treat muscular dystrophy.
The MDA said the grant is the largest the group has awarded to a private company in its history of more than 50 years.
The specific target for the Asklepios project is Duchenne muscular dystrophy, which is a fatal disease that strikes children.
Asklepios says it will place a proprietary, miniaturized dystrophin gene into a particle that will then deliver the gene to human muscles. The MDA said flaws in the gene for the protein dystrophin has long been identified as the underlying cause of muscular dystrophy.
“Without dystrophin, muscle cells become fragile, leading to progressive muscle weakness, resulting in wheelchair reliance by the teens and death from cardiac or respiratory complications by age 30,” the MDA said in its announcement. “The disease affects males almost exclusively.”
Asklepios describes itself as a company focused on the disease therapies delivered to genes through the design of proprietary biological nano particles.
Richard Jude Samulski, director of the Gene Therapy Center at the University of North Carolina, and Xiao Xiao, a biologist in the Department of Molecular Genetics & Biochemistry at the University of Pittsburgh, are lead reasearchers at Asklepios.
Asklepios will also conduct toxicology testing required by the Food and Drug Administration. If the method proves to be safe, Asklepios said it would launch a clinical trial including a small number of patients.
“The financial support provided by MDA will enable Asklepios to aggressively pursue the treatment of DMD on an accelerated pace, for benefit of all of the patients with this disease,” said Sheila Mikhail, CEO of Asklepios, in a statement.
The MDA has been an advocate of gene therapy for nearly 20 years.
“This contract represents a real milestone in MDA’s research program,” said Sharon Hesterlee, MDA’s director of research development, in a statement. “Enlisting the aid of a for-profit company should increase the efficiency and speed of drug development for neuromuscular disease.”
The MDA said gene therapy poses an alternative to other treatments that can’t stop the disease.
“Currently, the best medical therapy can only slow the progressive muscle weakness of Duchenne,” said neurologist Valerie Cwik, MDA’s medical director, in a statement. “There is no treatment to stop or reverse this process. Gene therapy strategies, potentially, could compensate for the causative genetic defect, providing a much more effective form of treatment.”
Another gene therapy project is underway at the University of Florida, MDA said.