ION306, also known as BIIB115, is an investigational antisense medicine in development for spinal muscular atrophy (SMA). This program has the potential to help address additional unmet needs of patients as well as be administered with long interval dosing. ION306 is designed to target the root cause of SMA by increasing the production of functional SMN protein.
About Spinal Muscular Atrophy
Spinal Muscular Atrophy (SMA) is a rare, genetic, neuromuscular disease that affects individuals of all ages. It is characterized by a loss of motor neurons in the spinal cord and lower brain stem, resulting in progressive muscle atrophy and weakness. SMA is caused by a deficiency in the production of survival motor neuron (SMN) protein due to a damaged or missing SMN1 gene, with a spectrum of disease severity. Some individuals with SMA may never sit; some sit but never walk; and some walk but may lose that ability over time. In the absence of treatment, children with the most severe form of SMA would usually not be expected to reach their second birthday.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.