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.
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