TAVT-18 is being investigated as a potential treatment for infants with tuberous sclerosis complex.
Tuberous Sclerosis Complex (TSC) is an ultra-rare multi-system genetic disease that causes non-cancerous (benign) tumors to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin. It usually affects the central nervous system and can result in a combination of symptoms including seizures.
TSC disease is caused by defects, or mutations, on the TSC1 and TSC2 genes. Scientists believe these proteins act as growth suppressors by inhibiting the activation of a protein called mTOR. Loss of regulation of mTOR occurs in cells lacking either hamartin or tuberin, and this leads to abnormal differentiation and development, and to the generation of enlarged cells, as are seen in TSC brain lesions.1
TAVT-18 is in early clinical development as a treatment for the prevention of seizures in infants with a confirmed diagnosis of TSC. The safety and efficacy of TAVT-18 is being evaluated through an investigator-initiated study.
1 Tuberous Sclerosis Fact Sheet, NINDS, NIH Publication No. 20-NS-1846, 2020.