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UMass Chan Medical School has licensed a promising siRNA-based technology to M2DS, a startup company dedicated to advancing the technology as a treatment for MECP2 Duplication Syndrome. This marks a major step forward in translating cutting-edge research into clinical reality.
The technology was developed in the lab of Dr. Khvorova, with the project led by Vignesh Hariharan. Early research was made thanks to funding from Cure MDS and the Rett Syndrome Research Trust. This siRNA works by targeting and degrading the messenger RNA responsible for the overproduction of the MeCP2 protein.
In mice studies, a dose reached the brain and spinal cord effectively and may only require once- or twice-yearly administration. By licensing this technology to M2DS, the therapy now enters a pathway where it can receive the support needed to advance toward human trials.
In addition to ongoing clinical trials from Ionis and HuidaGene, this milestone may add a promising addition to the pipeline of potential treatments for this rare and complex disorder.