April 12, 2013
/PRNewswire/ -- Startup biotechnology company Neuralgene (
) has announced that it will begin animal studies in May to evaluate the efficacy of PRCN-829, its new gene therapy agent for the treatment of amyotrophic lateral sclerosis (ALS). PRCN-829 is the first gene therapy for sporadic ALS.
Neuralgene's neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by
, M.D., founder and CEO of Neuralgene. "This technology addresses several key aspects of the underlying pathology of ALS," said
, M.D., clinical researcher for Neuralgene. "In his stem cell work, Dr. Williams had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action."
The gene therapy is based on Dr. Williams' discovery that certain proteins produced by stem cells inhibit the attack of ALS. During the development of the gene therapy, he added new targets: neural growth factors and a protein implicated in ALS named TDP-43. "When Dr. Williams demonstrated the concept behind stem cells and how to address the treatment of ALS using gene therapy, we immediately knew that this was a revolutionary new concept," said Dr. Gonzalez.
The PRCN-829 gene therapy is designed to not only target gene delivery to the brain and spinal cord, but also to genetically engineer stem cells. The AAV9 viral vector delivers multiple genes, which include Factor H (a regulator of complement activity), neural growth factors and regulators of TDP-43, to the neural cells. Initial animal studies have demonstrated the safety of the gene therapy platform.
"The problem with stem cell therapy for ALS is that the results are generally partial and temporary," stated Dr. Williams. "This is because the stem cells produce the growth factors and other proteins for a short period, but then cease. Several stem cell studies have confirmed this. Now with gene therapy, we can increase those factors by a millionfold or greater so that recuperation lasts for many years or maybe is even lifelong."