Key points from article :
Casma Therapeutics, backed by $58.5 million from Third Rock Ventures, is focusing on developing drugs to enhance autophagy—a natural process where cells recycle their dysfunctional components via lysosomes. Autophagy, essential for cellular health, was extensively studied by Nobel laureate Yoshinori Ohsumi, who uncovered its workings in bacteria. Casma builds on this research to identify key mechanisms in mammalian cells, aiming to translate these findings into therapeutic solutions.
The company’s initial focus is on lysosomal storage disorders (LSDs), a group of rare genetic diseases where enzyme deficiencies cause toxic buildup of cellular waste in lysosomes. Many LSDs primarily affect the central nervous system, and existing enzyme replacement therapies struggle to address these challenges due to poor penetration into the brain and spinal cord. Casma’s approach involves targeting upstream processes of autophagy, developing small molecules to induce precise recycling of cellular debris.
While some drugs like mTor inhibitors can trigger autophagy, they act on multiple cellular pathways, making them unsuitable for specific treatment needs. Casma’s goal is to create targeted drugs that avoid these unintended effects. However, the company faces significant hurdles, including the lack of reliable biomarkers to assess whether its drugs effectively induce autophagy.
Casma's efforts extend beyond LSDs, with long-term goals to address more common conditions linked to cellular debris buildup, such as Parkinson’s disease, muscle myopathies, liver issues, and inflammation. By tackling rare genetic disorders first, Casma hopes to pave the way for broader applications, leveraging its foundational research to improve treatments for widespread diseases. With a scientific foundation rooted in groundbreaking autophagy studies and a strategic focus on central nervous system disorders, Casma aims to transform therapeutic approaches for diseases driven by cellular dysfunction.
