SRT-344 is a drug candidate being developed for the treatment of spinal muscular atrophy (SMA) by SMaRT Therapeutics. Their website reports that SRT-344 is currently in the pre-clinical testing stage and is expected to enter Phase I clinical trials within the next 12-24 months.
SMA is caused by defects in the Survival Motor Neuron 1 (SMN1) gene which encodes the Survival Motor Neuron (SMN) protein. SMN is critical to the health and survival of nerve cells in the spinal cord that are responsible for neuro-muscular growth and function. An individual with SMA has a missing or mutated SMN1 gene, causing a deficiency in the SMN protein. This protein deficiency has its most severe effect on motor neurons.
SMaRT Therapeutics hope to be able to use their RNA-modulating technology to repair the missing or mutated SMN1 mRNA and SMN protein deficiency. This would make SRT-344 the only SMA drug targeting SMN1 gene. Other drugs currently in clinical trials like Repligen RG3039 and ISIS SMN-Rx target the "backup" SMN2 gene.
I'm looking for more information about this particular treatment-in-formation. If it all worked as projected, what age ranges would be treatable?
ReplyDeleteIf it proves to really work on SMN1 mRNA, then all types of SMA would be treatable. The progression of the disease would probably infulence the success of the treatment.
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