Alnylam: Stock Story

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Tell us about the company

Richie Vernon:  Imagine a drug that can navigate through the body and into your cells, selectively switching off the faulty, disease-causing parts of your gene, like a light switch. These revolutionary drugs aren't science fiction. They're transforming patients' lives today, and Alnylam Pharmaceuticals is the pioneer of this new drug class.

The company was founded in 2002 by a group of leading scientists who based their work on the Nobel Prize-winning discovery of RNA interference, or RNAi. Over the last two decades, they turned this scientific breakthrough into safe and effective medications. 

What makes this stock special?

Taking a drug from the initial discovery stage to approval for use in patients is the business equivalent of a well-funded expedition to walk over a thin layer of ice covering the world's largest lake. It's long, it's expensive, and it's rarely achieved. The value of Alnylam's RNAi technology is that it makes this task more repeatable than traditional drugs. After success with one RNAi drug, reordering the molecule allows it to be programmed to target other disease-causing genes. This effectively marks a safe path across the ice for others to follow.

This process has meant Alnylam's drug development track record is six times better than the industry average. And it's not just the company's drug research that makes it special. The commercial expertise it has built over the years enables Alnylam to challenge some of the largest big pharma companies.

What could the future hold?

Alnylam has spent its first two decades proving its RNAi drugs can work. Its approved drugs have already changed the lives of patients who suffer certain rare diseases. This success has also provided a consistent stream of cash that Alnylam is investing to deliver treatments for more common diseases, such as hypertension, fatty liver disease, and Alzheimer’s. If Alnylam can maintain its impressive drug development track record in these more common diseases, the potential rewards for patients, the company, and its shareholders are extensive.

Risk factors

This communication was produced and approved in March 2025 and has not been updated subsequently. It represents views held at the time and may not reflect current thinking.

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