Targeting “Undruggable” Diseases

Theresa Brady
Theresa Brady
June 5, 2020

Our series, Biotechnology: Beyond Imagination, highlights the technology and scientific advancements of BIO Digital participating companies and BIO member companies. Our next feature looks at Alnylam Pharmaceuticals, a biotechnology company working on treatments for diseases that were once thought to be “undruggable”.

Beyond Imagination Member Spotlight: Alnylam Pharmaceuticals

Biotechnology companies around the world make it their mission to solve the world’s most complex medical issues and unmet medical needs—diseases considered “undruggable” by modern science. Alnylam Pharmaceuticals, a Massachusetts-based company, is working to change this for patients with certain genetic, cardio-metabolic, infectious and central nervous system (CNS) and ocular diseases.

Since 2002, Alnylam has worked to pioneer an innovative, new class of medicines called RNA interference (RNAi) therapeutics.


Targeting the Genetic Cause, not the Symptoms

RNA interference (RNAi) medicines selectively target and then “silence” the gene expression that causes specific diseases. Gene expression is the process by which a set of instructions in DNA are converted into proteins by RNA. Messenger RNA (mRNA) carry these instructions out of the cell nucleus into the cytoplasm of the cell where they are used to manufacture new proteins. However, sometimes errors in DNA, known as mutations, can produce faulty RNA (and therefore faulty mRNA) that create disease-causing proteins.

RNAi therapeutics target cells directly and treat the root-genetic cause of disease rather than its symptoms by degrading or “interfering” with the faulty mRNA. Using this approach, Alnylam believes that RNAi therapeutics can potentially silence any disease-causing gene in the genome. In a recent I AM BIO podcast, Dr. John Maraganore, CEO of Alnylam Pharmaceuticals, used an analogy to explain RNAi. He said that if you have a flood in your kitchen, RNAi is like turning “off the faucet causing the flood” rather than most of today’s medicines that only “mop up the floor.” This innovative method allows for the potential to treat many diseases that have previously had no or inadequate treatment options.  

Alnylam already has two FDA-approved drugs on the market to treat rare diseases. The Alnylam team believes that they are just scratching the surface of what’s possible with RNAi treatments, and they don’t plan to slow down their progress anytime soon.

When discussing what lies ahead for Alnylam and RNAi therapeutics, Akshay Vaishnaw, Alnylam’s head of R&D said: “We’re having a significant impact on the lives of patients already but there are so many more that we believe we can help with this technology, and we’re just getting started.”

As Alnylam continues to work on treatments for previously “undruggable” diseases, BIO applauds its research and the potential treatments they will provide to patients around the globe.  

Registered attendees of BIO Digital can attend In an Era of Partisan Stalemate, What Drug Pricing Policies Can the Executive Branch Enact Without Congressional Authority and How will healthcare systems adapt to the Post-COVID-19 World? with Alan Eisenberg, Alnylam’s Vice President of Global Government Relations & Public Policy. Attendees also can attend “Start Me Up”: How Can Europe’s Biotech Sector Combat the Coronavirus? with Anant Murthy, Alnylam’s Vice President of Pricing, Market Access and Policy.