ISIS-SMNRx is designed to compensate for the underlyinggenetic defect that leads to SMA. SMA is caused by a loss of or defect in thesurvival motor neuron 1 (SMN1) gene, which leads to a decrease in the proteinsurvival motor neuron (SMN), a protein vital to the health and survival ofnerve cells in the spinal cord responsible for neuromuscular growth andfunction. ISIS-SMNRx, as described on the company's website, "is designed totreat all types of childhood SMA by altering the splicing of a closely relatedgene (SMN2) that leads to the increased production of fully functional SMNprotein." The drug has received Orphan Drug Designation and Fast Track statusfrom the
U.S. Food and Drug Administration, and is currently in a Phase I studyof children ages 2-14 with SMA to determine its safety, tolerability andpharmacokinetic profile.
"SMA is a heartbreaking disease – it can kill childrenbefore their second birthday, and there are currently no therapies to treat thedisease," George A. Scangos, Ph.D., CEO of Biogen Idec, said in a pressrelease. "It is exactly the kind of disease and program that we are focused onat Biogen Idec. The unmet need could not be any greater, the program fits withour mission to bring innovative therapies to patients with serious neurologicdiseases and Isis' antisense compound has the potential to be a highlyeffective, first-to-market therapy for this deadly disease. We have the utmostrespect for Isis' scientific leadership and expertise in antisense technology,and we have crafted a collaboration that brings together our two companies'strengths toward a common goal."
