Gene Solutions, a pioneer in mitochondrial dysfunction genomics and central nervous system drug discovery, has announced the publication of a key patent for its platform technology. The granted patent encompasses novel innovations that support the company’s proprietary approach to identifying and developing therapies for mitochondrial dysfunction. It also secures protection for a wide array of newly developed therapeutic lead candidate compounds, several of which have demonstrated enhanced selectivity for diseased DNA sequences.
Mitochondria play a central role in human health, producing 90% of the energy required by cells while also regulating critical biological processes such as programmed cell death. When mitochondrial function is impaired, energy production is disrupted, which can trigger the onset of multiple neurodegenerative disorders. The mitochondrial genome (mtDNA) contains specific genes that code for proteins responsible for generating adenosine triphosphate (ATP). Mutations in these genes, known as Single Nucleotide Polymorphisms (SNPs), can reduce ATP levels. When these reductions fall below a critical threshold, mitochondrial dysfunction diseases such as MELAS—mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes—can emerge.
To address these challenges, Gene Solutions has created an innovative class of gene regulatory compounds referred to as DNA-binding polyamides (DBPAs). These compounds are designed to recognize mutated SNPs and suppress transcription. The company’s DBPAs display exceptional precision, with greater than 90-fold selectivity for mutated sequences over normal, or wild-type, DNA. For example, the technology can specifically target mutations in the COXIII gene, which are associated with a range of debilitating mitochondrial disorders. Using a combination of biophysical and biochemical tools, Gene Solutions has established a platform to rigorously evaluate both the potency and selectivity of its DBPA candidates.
James Oury, a member of Gene Solutions’ board, described the publication of this patent as a defining step in advancing research and treatment options for neurological disease. He emphasized that securing these intellectual property rights not only protects the company’s discoveries but also enhances its attractiveness as a partner to pharmaceutical and biotechnology companies worldwide.
With scientific teams spanning the United States and the United Kingdom, Gene Solutions is committed to accelerating therapeutic progress through advanced research, strong collaborations, and a deep focus on improving patient outcomes in mitochondrial dysfunction-related conditions. The discovery program behind this breakthrough was carried out in partnership with the University of Strathclyde, whose multidisciplinary expertise supported the successful application and award of the patent. Gene Solutions maintains exclusive commercialization rights and retains the option to assume full patent ownership if needed by itself or its partners.
