Alnylam Pharmaceuticals, Inc., an RNAi therapeutics company in Cambridge, announced today the publication of promising pre-clinical results related to its ALN-HTT program, an RNAi therapeutic drug-device combination for the treatment of Huntington’s disease.
The findings – the result of a research partnership between Alnylam and Medtronic, Inc., and supported by CHDI – demonstrate that a small interfering RNA (siRNA) targeting the huntingtin gene, when administered by intrastriatal infusion with convection-enhanced delivery (CED), results in widespread distribution of the siRNA and significant silencing of the huntingtin mRNA throughout the striatum. Furthermore, administration of the RNAi therapeutic was well tolerated in these studies. The authors of the paper include researchers from Alnylam, Medtronic, and the University of Kentucky where the work was conducted.
Huntington’s disease is an autosomal dominant neurodegenerative genetic disorder that causes motor, cognitive, and behavioral dysfunction. It is estimated that 120,000 people in the U.S. have the genetic mutation that causes Huntington’s disease and will experience symptoms during a normal lifetime. The average lifespan for patients after onset of motor dysfunction is approximately 10 to 20 years. There are currently no disease-modifying therapies available to slow the progression of Huntington’s disease.
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development.
RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.