Akshay Vaishnaw, MD, PhD, President, Research and Development at Alnylam Pharmaceuticals, a company focused on RNA interference (RNAi) to develop new therapeutics for genetic conditions. At the BIO International Convention held in Philadelphia, PA, we talked to Dr Vaishnaw about the company and its RNAi technology.
RNAi is a nature part of our cells. We currently have RNA segments that function to interfere, or block, the synthesis of certain proteins. These segments are called RNAi. Using that knowledge, RNAi can also be used to interfere or block proteins that are leading to rare genetic diseases. For example, the Alnylan’s drug, Patisiran, blocks the synthesis of an abnormal form of transthyretin which is responsible for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis.
To better understand the concept, Dr. Vaishnaw explained how proteins are made. “We have genes in our nucleus those genes give rise to something called messenger RNA which is an intermediary. The messenger RNA is translated to proteins and proteins are things like enzymes and hormones and they do the work of our body. If there’s a mutation in the gene, you get an abnormal messenger RNA and you get an abnormal protein. RNA interference is a way of tackling that intermediary the messenger RNA,” said Dr. Vaishnaw, adding, “with RNA interference we can get rid of that messenger RNA to a substantial degree and reduce the level of the protein that’s produced and causing the disease in inherited disorders.”
“So that’s why RNA interference is such an attractive, powerful, and specific way to attack terrible rare diseases,” noted Dr. Vaishnaw.
In addition to its approved RNAi drugs such as patisiran to treat hAATR and givosiran to treat acute hepatic porphyria, Alnylam is excited about the rest of its pipeline and their ability to learn from past developments to further expand their pipeline, including the ability to develop drugs that can be delivered to the brain. Dr. Vaishnaw said, “We’re very excited and encouraged by our ability to get RNAi to work in the brain and in the eye. And we’re now looking forward to tackling the many terrible diseases that affect those organs, many of them rare diseases like Lou Gehrig’s disease for example in the brain and spinal cord or severe inherited forms of parkinsonism or forms of Alzheimer’s that are inherited.”
For more information, visit www.alnylam.com
