After 17 years of effort, and billions of dollars raised and invested in R&D, Alnylam Pharmaceuticals is now introducing an entirely new class of medicines to transform the lives of patients with rare diseases. Here's what Alnylam is doing in Holland and what the future holds according to them.
Alnylam has led the translation of RNAi (RNA interference) from Nobel Prize-winning discovery into an entirely new class of medicines. In 2018, our first medicine, ONPATTRO¬Æ (patisiran), became the world‚Äôs first approved RNAi therapeutic. Our second medicine, GIVLAARITM (givosiran), was approved by the FDA in 2019. We are advancing a robust pipeline of innovative RNAi-based medicines in four therapeutic areas: genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS) and ocular diseases.