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Gene Therapy Developed for ALS Treatment: New Biotech Company Neuralgene Enters Evaluation Phase
Date:4/12/2013

BOGOTA, Colombia, April 12, 2013 /PRNewswire/ -- Startup biotechnology company Neuralgene (http://neuralgene.com) 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.

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-a )

(Photo: http://photos.prnewswire.com/prnh/20130412/PH93428-b )

Neuralgene's neurotropic AAV-based gene therapy platform for the treatment of neurodegenerative diseases is based on the stem cell work performed by Jason Williams , M.D., founder and CEO of Neuralgene. "This technology addresses several key aspects of the underlying pathology of ALS," said Leonardo Gonzalez , 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
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