CHDI
CHDI Foundation continues collaboration with Medtronic to advance siRNA therapeutics to treat Huntington’s disease
New York City, CHDI Foundation, Inc. has announced it will continue its collaboration with Medtronic, Inc. (NYSE: MDT) to advance a novel drug-device combination therapy for the treatment of Huntington’s disease (HD). This therapeutic approach uses a siRNA that is targeted towards the huntingtin gene, a mutated form of which causes HD. The siRNA is delivered directly to the central nervous system (CNS) using an implantable drug infusion system developed by Medtronic, a world leader in medical device technology. CHDI is a not-for-profit drug development organization exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease.
“We are very pleased to be continuing our collaboration with Medtronic to develop this therapeutic approach for Huntington’s disease,” said Robi Blumenstein, President of CHDI Management. “Strategies to lower huntingtin levels are an important part of CHDI’s therapeutic portfolio and we are now anticipating the steps required to clinically test this particular siRNA approach in patients.”
Medtronic’s proprietary infusion system uses an implantable, battery-powered drug pump that stores and delivers the siRNA directly to a targeted area in the brain through a specially-designed, investigational catheter connected to the pump. Pre-clinical research currently underway to assess the safety of the proposed therapy represents a promising step towards taking this as well as other potentially disease-modifying therapeutic approaches into the clinic. Ultimately, clinical trials will be needed to address the still-unanswered question of whether lowering huntingtin can be beneficial in slowing disease progression.
“Medtronic is excited to continue our collaboration with CHDI and our research partners to advance research which may one day lead to a novel approach for the treatment of Huntington’s disease,” said Lothar Krinke, Ph.D., vice president and general manager for the Deep Brain Stimulation business in Medtronic’s Neuromodulation division. “Pre-clinical work to combine an innovative therapeutic strategy with Medtronic’s state-of-the-art drug delivery technology has generated promising results, and we look forward to advancing our efforts to find a treatment for this devastating neurodegenerative disease.”
Medtronic and CHDI will continue to work with their research partners to publish data and communicate significant findings when appropriate.
About Huntington’s disease
Huntington’s disease is an inherited neurodegenerative disorder caused by a mutation in the huntingtin gene. The defect causes a DNA sequence called a CAG repeat to occur many more times than normal. Each child of a parent with a mutation in the huntingtin gene has a 50% chance of inheriting the mutation. As a result of carrying the mutation, an individual’s brain cells degenerate leading to behavioral, cognitive, and motor impairments that, over the course of the disease, significantly reduce the individual’s quality of life and ultimately cause death within 15 to 25 years of overt symptom onset. There are currently no therapeutics approved that slow the progression of Huntington’s disease. It is estimated that the disorder affects about 30,000 people in the United States and at least 150,000 others have a 50% risk of developing Huntington’s disease at some point.
About RNA Interference (RNAi)
RNAi (RNA interference) is a biological process that was only recently discovered in 1998; Drs Andrew Fire and Craig Mello were awarded the Nobel Prize for Physiology or Medicine in 2006 for their groundbreaking discovery. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals; it is a fundamental breakthrough in understanding how genes are turned on and off in cells. By harnessing the natural biological process of RNAi occurring in our cells, a major new class of medicines, known as RNAi therapeutics, is now being developed. The molecules that mediate RNAi, called small interfering RNAs (siRNAs), can ‘silence’ or switch off specific mRNAs, which then prevents the production of disease-causing proteins like mutant huntingtin.
About CHDI Foundation, Inc.
CHDI Foundation, Inc. is a privately-funded, not-for-profit, biomedical research organization that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington’s disease (HD). As a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as quickly as possible. Our scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing strategic scientific direction to ensure that our common goals remain in focus. More information about CHDI can be found at www.chdifoundation.org.