The startup develops targeted medical remedies for Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke (MELAS), focusing on enhancing neuronal and mitochondrial function while improving cerebrovascular hemodynamics and inflammatory responses. Their treatments aim to alleviate both central and peripheral symptoms of mitochondrial diseases, facilitating timely patient care.
Funding
$81M 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
Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like episodes (MELAS) is a rare genetic disease with no approved treatments, leading to significant morbidity and reduced life expectancy. MELAS affects multiple organ systems, particularly those with high energy demands such as the brain and muscles, resulting in debilitating symptoms.
Solution
Tisento Therapeutics is developing zagociguat, a first-in-class, brain-penetrant soluble guanylate cyclase (sGC) stimulator, as a potential treatment for MELAS and other genetic mitochondrial diseases. Zagociguat is an oral, once-daily investigational medicine designed to rebalance dysregulated cellular pathways and restore mitochondrial energy production. By stimulating sGC, zagociguat aims to improve neuronal function, cerebrovascular hemodynamics, and inflammatory responses, addressing both central and peripheral symptoms of mitochondrial dysfunction. Tisento's approach is informed by patient perspectives, ensuring clinical studies measure the most relevant and meaningful aspects of MELAS. The company is currently enrolling participants in a global Phase 2b clinical trial (PRIZM) to evaluate the safety and efficacy of zagociguat in adults with MELAS.
Target Audience
The primary target audience includes individuals diagnosed with MELAS and other genetic mitochondrial diseases, as well as their families and healthcare providers specializing in mitochondrial disorders.
Features
- Oral, once-daily administration for convenient dosing
- Brain-penetrant sGC stimulator to target central nervous system manifestations
- Mechanism of action designed to rebalance dysregulated cellular pathways in MELAS
- Potential to improve neuronal function, mitochondrial function, and blood flow in the brain
- Clinical development program informed by patient interview studies to ensure relevant endpoints
- Currently in Phase 2b clinical trial (PRIZM) evaluating safety and efficacy in adults with MELAS