Alnylam: Message of Hope

April 4, 2008
A Message of Hope
Listen to Alnylam's Message of Hope

Alnylam Pharmaceuticals was founded in 2002 and now has approximately 110 employees. Based in Cambridge, Mass, the company is a leader in the development of RNAi therapeutics. Discovered in 1998, RNAi is a naturally occurring tool the body uses to turn off a gene's function when it isn't needed. This property makes RNAi a fantastic weapon against human disease. Using RNAi, Alnylam is developing a deep pipeline of drug products to treat a wide array of important diseases.

DISEASE PROGRAMS USING RNAi

Respiratory Syncytial Virus (RSV)

Alnylam is working to create a therapy for RSV, the most common cause of bronchiolitis and pneumonia among infants and children under 1 year of age. Using a direct RNAi approach, Alnylam will deliver the drug directly to the lungs in order to silence the gene the virus uses to infect the lung cells, neutralizing the virus and preventing further spread and infection. In June 2007, Alnylam initiated a second clinical trial to evaluate the safety, tolerability, and anti-viral activity for its lead therapy in experimentally infected subjects. Data are expected in the the first quarter of 2008.

Hypercholesterolemia
  • About 105 million U.S. adults have cholesterol levels of 200 mg/dL or higher (CDC, May 2007)
  • Familial hypercholesterolemia occurs in approximately 1 in 500 people in the U.S. (~500k people)
  • High cholesterol is a leading cause of heart disease, the #1 cause of death in the United States (CDC, May 2007)
Alnylam is collaborating with researchers at UT Southwestern Medical Center to evaluate new approaches for reducing LDL-cholesterol ( or "bad" cholesterol) levels using RNAi therapeutics directed to a disease target. In a pre-clinical study, this team has shown that their targeted RNAi approach can result in an approximately 50% lowering of LDL cholesterol.

Liver Cancers

Primary liver cancer is one of the most common cancers worldwide, with more than 600,000 people diagnosed each year. Secondary liver cancer, also known as metastatic liver cancer, is cancer that spreads to the liver from another part of the body. Worldwide, more than 500,000 people are diagnosed with secondary liver cancer each year. Alnylam is now in testing of its therapy that enables the efficient silencing of genes associated with liver cancer. In pre-clinical studies, Alnylam has demonstrated the ability to silence two important genes associated with liver cancer, stopping the cancer cells from growing.

Huntington's Disease (HD)
  • According to the Huntington's Disease Society of America (HDSA), one out of every 10,000 Americans has HD
  • Each child of a parent with HD has a 50/50 chance of inheriting the gene that causes HD
  • There are approximately 200,000 Americans "at-risk" of developing the disease
Alnylam and Medtronic are focusing on developing a drug-device combination for the treatment of Huntington's disease. The product is expected to consist of an RNAi therapeutic targeting the Huntington's disease gene that will be delivered by Medtronic's implantable infusion pump.
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