Motor neuron diease News, Reviews & Resources

A neuroscience company targeting mitochondrial dysfunction has teamed up with an organization developing small molecule disease-modifying medicines for the treatment for chronic neurogenerative disorders. Domainex, an integrated medicines research services partner has been chosen by neuroscience company, NRG Therapeutics, and will be providing fully integrated lead optimization services including assay biology and medicinal computational chemistry. NRG Therapeutics Ltd. received a £2.68 million ($3.26 million) early stage Biomedical Catalyst (BMC) award funded by government backed agency Innovate UK which has made the project possible. NRG has invested more than £1 million ($1.2 million) in the project anticipated to take place over a one-year period. Parkinson's Disease Domainex will be focused on medicines for the treatment of Parkinson’s Disease, motor neuron disease (MND) and other debilitating chronic neurodegenerative disorders. NRG and Domainex will conduct an integrated lead optimization program in order to optimize the pharmaceutical properties of small-molecule mPTP inhibitors, with the ultimate aim of nominating a pre-clinical candidate for the treatment of Parkinson’s or motor neuron disease (MND). This project follows several projects Domainex has worked on that have been funded by Innovate UK, such as a project targeting IKKɛ /TBK1 for the treatment of inflammatory disease. Domainex also received Innovate UK funding to develop its PoLiPa technology. Tom Mander, CEO of Domainex said: “We are thrilled to have been selected by NRG Therapeutics to work on this exciting project. We hope that this program will result in a new treatment for neurodegenerative diseases, such as Parkinson’s, which will ultimately benefit patients who desperately need new treatment options for these devasting diseases.” Mitochondria Work that has already been carried out by NRG and funded by Parkinson’s Virtual Biotech, the drug development arm of Parkinson’s UK, has led to the identification of small molecule lead series, which inhibit the mitochondrial inner membrane, which is critical for cell signaling and regulated cell death. Inhibition of mPTP in brain cells has been shown to be neuroprotective in several preclinical models of Parkinson’s and other neurodegenerative diseases. Mitochondria make the energy cells need to survive and dysfunction occurs in most neurodegenerative diseases, including Parkinson's diseases, Alzheimer's, Huntington's disease, Friedreich's ataxia, and amyotrophic lateral sclerosis. Neil Miller, co-founder and CEO of NRG Therapeutics, said: “Mitochondrial dysfunction is a common underlying pathology in many degenerative diseases and there is a substantial body of preclinical data available which demonstrates that inhibition of the mPTP in the brain prevents neuronal cell death, reduces neuroinflammation and extends survival in animals. We look forward to working with Domainex to progress the development of these compounds into potentially life-changing medicines.”

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