UMMS ALS researcher believes RXi technology will address current hurdles to RNAi-based treatments.

Three research foundations dedicated to amyotrophic lateral sclerosis (ALS) research and drug development will jointly fund an ALS therapeutics collaboration between RXi Pharmaceuticals and researchers at the University of Massachusetts Medical School (UMMS) in Worcester.

The work, by RXi and Robert Brown, M.D., D.Phil, chair of the department of neurology at UMMS, aims to evaluate the use of RXi’s self-delivering rxRNA™ (sd-rxRNA™) compounds in the treatment of ALS. The research will be funded by The Angel Fund, the ALS Therapy Alliance, and Project ALS.

As part of the collaboration, Dr. Brown’s team will investigate the ability of RXi’s sd-rxRNA molecules to effect gene silencing and extend survival in a mouse model of familial ALS. According to Dr. Brown, while an RNAi-based approach to ALS therapy is theoretically promising, in practice it has been limited by issues associated with delivering the molecules. He maintains RXi’s sd-rxRNAs could represent “the unique solution that could be the key to treating neurological disorders such as ALS.”

RXi claims the fact that its proprietary rxRNA compounds are designed specifically for therapeutic use means that in comparison with conventional siRNAs, its molecules have a number of beneficial features. These include higher specificity for the target gene, a greater reliability at blocking immune side effects, up to 100-fold higher activity, and increased resistance to nuclear degradation.

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