Science & Pipeline
Pretzel is focused on identifying and prosecuting novel disease targets associated with dysregulated cellular bioenergetics
Our approaches control replication and transcription of mitochondrial DNA (mtDNA) to modulate disease processes and improve survival, function and quality of life. This unique energetics regulating approach was designed to impede disease progression and severity across high unmet need indications.
Pretzel is building a deep, first-in-class pipeline of novel medicines that impact targets associated with restoring cellular energetics for the treatment of neurodegenerative and rare diseases associated with low mtDNA levels or modifying bioenergetics for the treatment of metabolic conditions such as obesity.
Restoring EnergeticsRare / Neuro
Modulating EnergeticsObesity / Metabolic
PX578
PX578, the lead therapeutic in our energetics restoration franchise, represents a first-in-class approach to targeting and activating the mitochondrial polymerase POLG, with disease modifying potential across rare mtDNA depletion syndromes (MDDS) and broader degenerative diseases. PX578 is in Phase 1 clinical development.
POLRMT
The lead program in our energetics modulation franchise targets POLRMT, the mitochondrial RNA polymerase. This novel liver-targeted mechanism for the treatment of metabolic conditions, including obesity, is in late preclinical development.