Patent Publication (WO 2025 168944 A1) and subsequent PCT filing solidifies R&D leadership in Advanced Neurotherapeutics for Alzheimer’s, Parkinson’s and Related Disorders

CHARLOTTESVILLE, Va., Sept. 11, 2025 /PRNewswire/ — Gene Solutions, a leader in mitochondrial dysfunction genomics and CNS drug discovery, announces 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 sizable range of novel therapeutic lead candidate compounds, a number of which have shown enhanced selectivity for diseased DNA sequences

As mitochondria supply 90% of the energy needed by the body’s cells and regulate a variety of biological functions, including cell death, mitochondrial dysfunction can lead to the onset of a range of neurodegenerative diseases by compromising energy production. Mitochondria contain its own small genome (mtDNA), which comprises genes that encode for proteins associated with adenosine triphosphate (ATP) production. Mitochondrial dysfunction occurs where one or more of these genes contains defects (i.e., mutations called Single Nucleotide Polymorphisms or SNPs), resulting in reduced ATP production. Diseases related to mitochondrial dysfunction arise when these SNPs result in ATP production falling below a critical threshold.

Gene Solutions has developed a new generation of gene regulatory compounds, known as DNA-binding polyamides (DBPAs), that recognize mutated SNPs and down regulate transcription. The company’s DBPAs exhibit > 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.”

With research expertise spanning the USA and UK, Gene Solutions is dedicated to accelerating therapeutic innovation through world-class science, strategic partnerships, and a steadfast focus on improving outcomes for patients suffering from mitochondrial dysfunction disorders.

For more information about the groundbreaking technology, collaborative opportunities, or the company’s therapeutic programs, please visit the Gene Solutions website or contact the company directly.

About Gene Solutions
Gene Solutions is a biotechnology company specializing in therapeutic research for CNS diseases, with a focus on mitochondrial genomics and innovative biomarkers. The company collaborates globally to accelerate drug discovery and diagnostics for conditions such as Alzheimer’s, Parkinson’s, and Schizophrenia.

¹ Gene Solutions (or “the Company”) funded this discovery research project with the University of Strathclyde’s cross-functional chemistry, biology, and molecular diagnostic teams; accordingly, the patent was applied for and awarded to the University of Strathclyde. The Company retains exclusive commercialization rights to the patent and also retains the right to be assigned the patent if so desired by the Company or its partners.

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