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news.Neuralgene is planning to initiate animal studies to assess new gene therapy agent PRCN-829 being developed as amyotrophic lateral sclerosis (ALS) therapy.
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The therapy agent with AAV9 viral vector will 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 had identified that production of Factor H by fat-derived mesenchymal stem cells may be a key mode of action,"Williams added.
"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 that 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 by the company to target gene delivery to the brain and spinal cord, in addition to genetically engineer stem cells.