Collaboration aims to use targeted therapy to block production of causative huntingtin protein.

Lundbeck is teaming up with researchers at the University of Massachusetts Medical School (UMMS) to investigate whether RNAi technology could be applied to slow or even stop the progression of Huntington disease (HD). The ultimate aim would be to selectively suppress production of the abnormal huntingtin (mHtt) protein that causes HD. Lundbeck says the partnership is part of its Huntington disease research initiative, through which the firm is collaborating with academic organizations and companies that have promising compounds in development.

Led by UMMS’ professor of medicine and cell biology Neil Aronin, M.D., the new partnership will evaluate potential dosing regimens for the use of siRNA molecules packaged as shRNA and delivered via an AAV vector. Evaluation studies will most critically involve measuring the distribution volume of the AAV-delivered shRNAmir (miRNA-adapted shRNA) in brain tissue to achieve spread throughout the striatum and nearby cortex.  

“Our core idea is that RNAi can be used to selectively reduce mutant huntingtin production to slow or block the progression of HD, but we also hypothesize that excessive huntingtin silencing may impair neuronal function by interfering with essential signaling events,” Dr. Aronin notes. “This research collaboration allows us to test promising RNAi-based therapeutic vehicles to selectively knock down mutant huntingtin with the goal of restoring normal neuronal function.”

“We’ve followed Dr. Aronin and his team of researchers at the Medical School for some time and have been inspired by their bold exploration of RNAi technology and its potential use as a therapy for HD,” adds Stevin Zorn, Ph.D., evp at Lundbeck Research USA.

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