Design of Mitochondrial Precision Therapeutics Through an AI-guided Synthetic Biology Approach to Enhance Myocyte Resilience and Durability

Mitochondrial dysfunction has been causally linked to aging and sarcopenia, yet current therapies fail to target mitochondria directly. Powerhouse Biology is developing AI-driven synthetic mitochondrial therapeutics to enhance cellular resilience and durability that will translate to improved human health outcomes. This project focuses on designing and validating synthetic mitochondrial assets, leveraging insights from resilient species and applying them to physiologically relevant human myocyte preclinical models.

Building on our expertise in sarcopenia and AI-guided drug discovery, we will computationally design mitochondria-targeted assets inspired by evolutionary adaptations. We will then test their effects on mitochondrial function in human skeletal muscle cells. Finally, we will optimize, and validate synthetic mitochondrial assets that enhance mitochondrial function to meet natural demands.

A successful outcome will validate synthetic mitochondrial enhancement in human cells, providing a foundation for novel therapeutics that prevent rather than cure muscle weakness. The NAM Catalyst Award will accelerate proof-of-concept development of novel AI-guided synthetic designs of mitochondria-centric precision therapeutics.