TS-000497 — Huntington’s Disease (HD) is a late onset progressive neurodegenerative disorder that results in death in 10-15 years after the first sign of symptoms. Existing oligonucleotides (AONs) based therapies are imperfect as they knockdown wildtype protein, require consistent re-injections, and use potentially harmful molecules. Gene therapy experts at Nationwide Children’s have devised a gene therapy approach that uses a specific snRNA to stably and safely reduce the highly pathogenic protein HTT. By enabling a continuous expression of the therapeutic RNA in the nervous system (and other targets), this technology may delay the age of onset, slow symptom progression, and reduce symptom severity of HD. Hence, it has the potential to become the optimal therapeutic strategy for the treatment of HD.

TS-000470 — CuATSM is a small molecule which facilitates the delivery of copper to cells containing damaged mitochondria, the cell compartments responsible for the production of energy. Currently, CuATSM has shown success in clinical trials for treating patients with ALS. Neurodegenerative disease experts at Nationwide Children’s Hospital have elucidated novel mechanisms of action through which the small molecule can treat disorders caused by oxidative stress, mitochondrial dysfunction and elevation of stress response systems. Through these studies, our disease experts have identified several new indications which could be treated with this small molecule compound.