When a team of distinguished scientific leaders came together in 2002, they did not know that their vision would forever transform people’s lives. Their vision was to enhance the way medicines treat diseases, and this is what founded Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY). The name is derived from “Alnilam,” which is the bright center star in the constellation Orion’s belt.
The company’s focus is on RNAi therapeutics and has continued to expound on its ATTR amyloidosis programs’ progress. Through an “RNAi Roundtable” webinar, Alnylam outlined the possibility of a biannual subcutaneous dosing regimen for vutrisiran. Data from a clinical study assessing the effects of patisiran and diflunisal was also announced. This is a TTR stabilizer on the cardiac amyloid burden.
Building an Industry-Leading Franchise of Medicines for the Treatment of ATTR Amyloidosis
Alnylam’s focus on the unmet medical needs falls under four Strategic Therapeutic Areas (STArs). They include; cardio-metabolic diseases, genetic medicines, ocular diseases and infectious diseases, and the central nervous system (CNS). Over the years, excitement over the use of the RNAi approach has drawn multiple pharmaceutical players. Millions of dollars have been spent to unlock its power.
As a result, Alnylam feels it has the potential of building an industry that can give back to the pharmaceutical industry a franchise of medicines for the treatment of ATTR amyloidosis. The data presented recently patisiran has the potential to achieve a substantial reduction in cardiac amyloid burden in ATTR amyloidosis. The company also says data obtained from clinical pharmacology has demonstrated some potential in the biannual subcutaneous dosing regimen for vutrisiran.
Innovative Science Perseverance and Passion Put together
Right from the start, Alnylam’s story is all about giving patients hope and light. It has had a share of insurmountable challenges, which include the dissolution of major partnerships. This led to a loss of faith in its technology. However, within its walls remained an unwavering belief, enthusiasm, continued focus, and the drive to make RNAi therapeutics a reality.
Today, the dark ages and insurmountable challenges are histories. Having overcome them, it has been able to build a robust Research & Development engine. Additionally, its deep pipeline of investigational RNAi therapeutics is addressing a broad spectrum of diseases.