Core Insights - Thiogenesis Therapeutics, a clinical-stage biotechnology company, is focused on developing sulfur-based prodrugs for rare mitochondrial and metabolic diseases, with its CEO participating in a significant webinar to discuss clinical updates on their lead product candidate, TTI-0102 [1][2]. Company Overview - Thiogenesis Therapeutics, Corp. is publicly traded on the TSX Venture Exchange and OTCQX, based in San Diego, California, and specializes in sulfur-containing prodrugs aimed at treating serious pediatric diseases with unmet medical needs [7]. Clinical Programs - The company is advancing its lead product candidate, TTI-0102, which is designed to address mitochondrial oxidative stress, a key pathological feature of inherited mitochondrial disorders, with no approved therapies currently available [2][6]. - TTI-0102 is undergoing a Phase 2 clinical trial for Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS) and plans to initiate a Phase 2a clinical trial for Leigh syndrome spectrum (LSS) in the U.S. [2][7]. Disease Context - MELAS is an inherited mitochondrial disorder affecting approximately 4.1 per 100,000 of the population globally, characterized by symptoms such as seizures and muscle weakness [4]. - Leigh syndrome spectrum is a rare genetic disease diagnosed in about 1 in 40,000 live births, with symptoms including poor muscle development and respiratory issues, and currently has no approved drugs [5]. Product Details - TTI-0102 is a next-generation cysteamine-based prodrug that aims to improve tolerability and dosing convenience compared to first-generation thiol therapies, with controlled release of cysteamine [6].
Thiogenesis to Present Clinical Updates in MELAS and Leigh Syndrome Spectrum at UMDF Bench-to-Bedside Webinar