Company: Alnylam Pharmaceuticals
Liaison(s): Fred Tremblay, Gautham Sridharan
Alnylam’s platform technology enables the company to target virtually any gene expressed in the liver. Within this paradigm, the Next Wave team within the company has been tasked with the discovery and validation of novel gene-disease pairs amenable to RNAi therapeutic interventions. Of particular interest are indications under the umbrella of the “metabolic syndrome”, which are characterized by a multitude of co-morbidities including obesity, type II diabetes, non-alcoholic fatty liver disease (NAFLD), etc. To facilitate target discovery in this disease space, a bioinformatics approach was taken to identify gene expression profiles of liver-expressed genes in preclinical models of metabolic disease and treatment. Computational tools using Python were utilized to compile a local database of hepatokines, or liver-secreted proteins that could be putative targets for metabolic diseases. In addition, a brief market assessment and deep-dive literature review was performed on a prioritized list of 10 targets based on the bioinformatics exercise.