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Craig C. Mello, PhD |
The breakthrough discovery of RNA interference that earned UMass Medical School Professor Craig C. Mello, PhD, and Andrew Z. Fire, PhD, of Stanford University, the 2006 Nobel Prize in Medicine is yielding its first commercial drug, and possibly a new class of drugs, according to numerous media reports, including stories in Stat News, Forbes and Worcester Business Journal.
Patisiran, developed by Alnylam Pharmaceuticals to treat a rare nerve disorder known as familial amyloid polyneuropathy (FAP), is based on RNAi by using small pieces of genetic code to halt the activity of disease-causing genes. Results of a stage 3 clinical trial of patisiran showed real benefits in treating the disease. Alnylam and its partner Sanofi said they intend to seek market approval in the United States this year, based on the findings.
“We are very proud to report the first ever positive phase 3 results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines. This moment is the culmination of a 15-year journey of tireless work by countless contributors who have overcome enormous scientific and business challenges to make RNAi therapeutics a reality,” said John Maraganore, Alnylam’s CEO, in a statement, in the Stat News article on Sept. 20.
Dr. Mello, Howard Hughes Medical Institute Investigator, the Blais University Chair in Molecular Medicine and distinguished professor in of RNA therapeutics, and Dr. Fire published findings in 1998 that demonstrated that a particular form of RNA—the cellular material responsible for the transmission of genetic information—can silence targeted genes.
Read full media reports, below:
Stat News: Alnylam’s rare disease drug shines in trial, paving way for a brand-new class of medicines
Forbes: After 20 Years, A Drug That Silences Genes Succeeds
Worcester Business Journal: UMass professor's breakthrough paved way for new drug
Related links on UMassMedNow:
Boston Globe: Mello’s RNAi discovery propelling new advancements against disease
Telegram reports on advancements in RNAi treatments
