PharmiWeb.com - Global Pharma News & Resources
14-Jan-2026

Pretzel Therapeutics Advances Mitochondrial Medicine with Strategic Growth

Pretzel Therapeutics is gaining attention in biotech circles as it continues advancing its mission to transform treatments for diseases linked to mitochondrial dysfunction. The company’s lead therapy candidate, PX578, is now in Phase 1 clinical development targeting mitochondrial DNA depletion syndromes and potentially broader conditions where energy production in cells falters. Pretzel’s approach focuses on harnessing cellular energetics to restore mitochondrial function and impede disease progression across neurological, metabolic, rare and muscle-related conditions.

Recent clinical progress follows groundbreaking research published in Nature demonstrating novel small molecule activators that can restore mutant mitochondrial DNA polymerase activity, underlining the scientific potential of the company’s pipeline. Pretzel was also in the spotlight recently through its strategic positioning following the winding down of Rome Therapeutics, with aspects of Rome’s science and team folding into Pretzel’s work. The company, headquartered in Waltham, Massachusetts, and supported by global investors, is led by a team of experts in mitochondrial biology and drug development. Pretzel’s work could reshape how debilitating mitochondrial diseases are treated and expand potential applications into age-related neurodegenerative disorders too.