Alnylam is developing an entirely new class of medicines based on the breakthrough discovery in biology known as RNA interference, or RNAi. Alnylam believes that RNAi therapeutics are a promising approach to silence disease-causing genes, and represent an opportunity to transform the treatment of many diseases.
In December 2014, we announced a pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease. The strategy builds on our progress in the advancement of RNAi therapeutics, including our “Enhanced Stabilization Chemistry” (ESC)-GalNAc conjugate technology, as a modular and reproducible platform for discovery of innovative medicines. The three STArs will remain focused on liver-expressed and genetically validated or pathogen-derived disease targets, with biomarkers for assessment of clinical activity early in Phase 1 trials. Across our three STArs, we believe that we can address major unmet needs in a wide range of diseases with high-impact, differentiated medicines, and continue to build what we believe to be one of the most robust pipelines in biotech. By executing on this strategy, we believe we have the potential to make a meaningful difference in the lives of patients, and maximize value for shareholders.
In January 2014, Alnylam and Genzyme formed a transformational alliance with Genzyme for the development and commercialization of RNAi therapeutics as genetic medicines. This new alliance will allow for the accelerated and expanded development and commercialization of RNAi therapeutics across the world. Under the terms of this alliance, Alnylam retains broad product rights in North America and Western Europe, while Genzyme obtains the rights to Alnylam’s genetic medicines pipeline in the rest of the world plus co-development and co-commercialization rights or global rights to three programs. In addition, Genzyme will make a $700 million equity investment in Alnylam. Importantly, this new partnership significantly expands our balance sheet to over $1 billion in cash to increase our investment in new RNAi therapeutic programs, while securing a cash runway that we believe will allow us to develop and launch multiple products as breakthrough medicines for patients in need.