Key points from article :
A new investment from The Mito Fund, the venture philanthropy arm of the United Mitochondrial Disease Foundation (UMDF), is supporting Pretzel Therapeutics, a Massachusetts-based biotech developing therapies for mitochondrial diseases. While the financial terms were not disclosed, the move underscores growing interest in targeting mitochondrial dysfunction—an area with major unmet medical need.
Pretzel focuses on genetic disorders affecting mitochondrial DNA (mtDNA), particularly those involving the POLG gene and mtDNA depletion syndromes. Its lead candidate, PX578, now in Phase 1 trials, aims to restore mitochondrial function by activating POLG, the enzyme responsible for mtDNA replication. By boosting mtDNA levels, PX578 could potentially slow or reverse the progression of energy-deficit diseases, including neurodegenerative and age-related disorders.
In addition to PX578, Pretzel is developing another therapy that targets the mitochondrial RNA polymerase POLRMT. This liver-specific candidate, still in preclinical stages, is being explored as a treatment for metabolic conditions such as obesity, by enhancing energy production at the cellular level.
Dr. Philip Yeske of UMDF emphasized the high potential of PX578 and praised Pretzel’s collaboration with patient advocacy groups. The Mito Fund’s investment was made following thorough scientific and financial vetting, and aligns with its mission to accelerate cures for mitochondrial diseases. Other companies supported by the fund include Napigen, Pierrepont, and Khondrion, all pioneering different angles of mitochondrial therapy development.
