Year: 2016-17

Company: Alnylam Pharmaceuticals

Liaison(s): Fred Tremblay, Alfica Sehgal

Alnylam Pharmaceuticals is a pre-revenue biopharmaceutical company founded in 2002, headquartered in Cambridge, Massachusetts. Alnylam was founded on the development of Ribonucleic Acid Interference ( RNAi ), which they are currently using to downregulate genetically-defined target genes in the liver, for the treatment of serious, life-threatening diseases with limited treatment options. The current drug pipeline at Alnylam is dedicated to delivering drugs in 3 Strategic Therapeutic Areas: Genetic Medicine, Cardio-Metabolic Diseases, and Hepatic Infectious Disease. Alnylam is interested in identifying rare liver-based diseases that are targetable by their RNAi technology. In addition, they need a database to store relevant disease information in an organized, searchable, and compatible platform. The Alnylam TMP team was tasked with identifying rare liver diseases that can potentially be targeted with Alnylam’s RNAi technology. To accomplish this task, the team conducted secondary and primary research by utilizing scientific papers from multiple, peer reviewed platforms and speaking with Key Opinion Leaders ( PhDs, MDs, and Foundations ), respectively, to identify diseases and put them into a team-developed database. The database includes all of the diseases we discovered, therapeutic target options for Alnylam’s technology, disease information, and current market landscapes. In addition, the team made recommendations about which diseases might be amenable to Alnylam’s technology. These recommendations should provide a solid framework for Alnylam to develop new therapies and enter new markets.