On February 1, 2023 Scenic Biotech, a pioneer in the discovery of genetic modifiers developing therapeutics to treat severe diseases, reported that it has entered into a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health (NIH), to support its therapeutic program for the treatment of Niemann-Pick Type C (NPC) disease, which is a severe, progressive and lethal neurological disorder (Press release, Scenic Biotech, FEB 1, 2023, View Source [SID1234626716]). The goal of the collaboration is to obtain clinical insights into genes that can reduce the severity of NPC. Under the terms of the CRADA, NICHD will grant Scenic Biotech access to its extensive clinical dataset and whole genome DNA sequence data derived from NPC patients. Forbes D. Porter, MD, PhD, Senior Investigator at NICHD, will be the principal investigator for this study based on his long-running clinical protocols that track the natural history of rare genetic disorders, including NPC.

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"The CRADA with the NIH will help advance our work toward identifying genetic modifiers for NPC. Our aim is to develop novel therapeutics that act by suppressing or completely blocking the effect of a disease-causing mutated gene. Through the combined efforts with Dr. Porter’s team, we hope to further enable the discovery of a tailor-made, disease-modifying small molecule therapeutic for this devastating disease," commented Sebastian Nijman, CSO and co-founder of Scenic Biotech.