Biotechnology company Neuralgene will begin animal studies to assess new gene therapy agent PRCN-829, developed to treat amyotrophic lateral sclerosis (ALS).
The company has developed the therapy agent with AAV9 viral vector to deliver multiple genes such as Factor H, neural growth factors and regulators of TDP-43 to treat neurodegenerative disease ALS.
Neuralgene founder and CEO Jason Williams said the technology addresses several key aspects of ALS’s underlying pathology.
"In his stem cell work, Dr Williams identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action," Williams said.
"Our gene therapy will target several of the main underlying mechanisms related to ALS with the hopes of getting a good response in a larger group of patients. However, our platform is versatile, allowing us to change and add different target genes."
New targets referred to as neural growth factors and a protein implicated in ALS named TDP-43 are included during development to the gene therapy, which is based on the discovery that the attack of ALS can be inhibited by few proteins produced by stem cells.
The PRCN-829 gene therapy is designed to target gene delivery to the brain and spinal cord, in addition to genetically engineer stem cells, said the company.
The results from the stem cell therapy for ALS are generally partial and temporary because of the production of the growth factors and other proteins for a short period, while gene therapy is believed to help in overcoming all these factors.
Image: MRI (axial FLAIR) demonstrates increased T2 signal within the posterior part of the internal capsule, consistent with the clinical diagnosis of ALS. Photo: courtesy of Frank Gaillard.