INVESTMENT HACKS

Alnylam Pharmaceuticals, Inc. (ALNY – Research Report) is using RNA interference (RNAi) to develop real-world medications for rare, genetically based diseases. These are typically diseases with odd names or long acronyms, and they don’t get into the news so much. They lack the recognition of more common debilitating diseases like muscular dystrophy or cystic fibrosis, but they are just as debilitating. Alnylam’s work with RNAi offers hope for patients and their families.

In simple terms, the drug technology works by using RNAi molecules to prevent the physical expression of a genetic disease. Genes are expressed by reading DNA instructions to produce protein molecules, which then act in the body. RNAi introduced into a patient’s body will interpose itself in the process, preventing production of incorrect proteins. For an RNAi medication to have a therapeutic effect, it is vital that it is carefully tailored to the exact gene responsible for the disease under treatment.

Cutting-edge science and the promise of making life better: this is a strong foundation for a biotech company. Let’s use the TipRanks’ database to see how Alnylam looks from an investor’s standpoint.

First in its Field

Alnylam is the first major biotech company to focus on RNAi therapies for genetic diseases. This gives the company a leg up, as it faces little competition right now for patents or drug approval.

Even more importantly, Alnylam got a boost during the summer after EU approval of its first drug, Onpattro (brand name for Patisiran), and RNAi medication for the treatment of hereditary ATTR amyloidosis. This is a genetic liver disease that be debilitating and sometimes fatal. Alnylam’s new drug stops the production of the protein responsible for the disease symptoms. Last month, Alnylam announced that it is launching Onpattro for patient use in Germany.

Alnylam also has seven more drugs in development phases, including four in late stage testing. One of those, Fitusiran, is designed to treat hemophilia. This is an important development, as hemophilia is far better recognized by the general public than most of the diseases that Alnylam works on, and so will be much more useful for broad-based fund raising.