Policy & Regulation
Gene Solutions' mitochondrial therapy platform targeting neurological diseases secures patent
12 September 2025 -

Gene Solutions, a company involved in mitochondrial dysfunction genomics and CNS drug discovery, announced on Thursday the publication of its platform technology patent.

The award covers novel technologies underpinning the company's platform for the identification and development of mitochondrial dysfunction therapeutics. The awarded claims also cover a range of novel therapeutic lead candidate compounds, several of which have shown enhanced selectivity for diseased DNA sequences.

Gene Solutions says that it has developed a new generation of gene regulatory compounds, known as DNA-binding polyamides (DBPAs), that recognise mutated Single Nucleotide Polymorphisms (SNPs) and down regulate transcription. The company's DBPAs exhibit greater than 90-fold selectivity for a desired SNP over wild type sequences, such as the SNP associated with the COXIII gene present in a range of mitochondrial diseases (e.g. mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes, MELAS). The company has developed a combined biophysical and biochemical platform to assess the potency and selectivity of this new generation of DBPA candidates.

"The publication of our platform and lead candidate library patent marks a significant milestone in our commitment to transforming the landscape of neurological disease research and treatment," said James Oury, Gene Solutions board member. "This achievement protects our proprietary inventions and strengthens our position as a collaborator of choice for pharmaceutical and biotechnology partners worldwide."

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