TPN-101

TPN-101 is a first-in-class, orally bioavailable small molecule nucleoside reverse transcriptase inhibitor (NRTI) that specifically targets the LINE-1 (Long Interspersed Nuclear Element-1) reverse transcriptase. Developed by Transposon Therapeutics, TPN-101 is designed to treat neurodegenerative diseases by inhibiting the activity of retrotransposable elements that become dysregulated and contribute to neuroinflammation and neuronal death. It is a derivative of the HIV medication stavudine (d4T). TPN-101 is currently being evaluated in Phase 2 clinical trials for the treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD), particularly in patients with the C9orf72 gene mutation, as well as Progressive Supranuclear Palsy (PSP) and Aicardi-Goutieres Syndrome (AGS).