Thiogenesis Therapeutics is developing thiol-active compounds, specifically TTI-0102, to enhance intracellular cysteine levels and boost glutathione synthesis, addressing oxidative stress and mitochondrial dysfunction in pediatric patients. The company targets inherited and acquired mitochondrial diseases, including metabolic liver disease and Leigh syndrome, for which there are currently no approved therapies.
Funding
$186.2K raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many inherited and acquired mitochondrial diseases lack effective treatments, leading to oxidative stress and mitochondrial dysfunction, particularly in pediatric patients. Current therapeutic options are limited, leaving a significant unmet need for interventions that can enhance intracellular antioxidant levels and improve mitochondrial function.
Solution
Thiogenesis Therapeutics is developing novel thiol-active compounds, including its lead candidate TTI-0102, designed to elevate intracellular cysteine levels and stimulate glutathione synthesis, a critical antioxidant. These compounds address oxidative stress and mitochondrial dysfunction, key features of mitochondrial diseases. TTI-0102 has demonstrated good tolerability at high doses in human studies and is engineered to overcome limitations associated with previous thiol-based drugs, such as short half-life and gastrointestinal side effects. The company is initially focusing on pediatric diseases with unmet medical needs where oxidative stress plays a central role.
Target Audience
The primary target audience includes pediatric patients suffering from inherited and acquired mitochondrial diseases, such as metabolic liver disease (MASLD/MASH), MELAS, and Leigh syndrome, for which there are currently no approved therapies.
Features
- TTI-0102: A novel chemical entity (NCE) that enhances intracellular cysteine levels, the rate-limiting factor in glutathione synthesis.
- Engineered thiol compounds designed to overcome limitations of previous thiol-based drugs, such as short half-life and gastrointestinal side effects.
- Addresses oxidative stress and mitochondrial dysfunction by boosting glutathione, a master antioxidant that neutralizes free radicals and maintains redox balance.
- Potential application for both inherited and acquired mitochondrial diseases, including metabolic diseases.
- Demonstrated good tolerability at high doses in human volunteer studies.