TFO Technology

The use of triplex-forming oligonucleotides (TFOs) and peptide nucleic acids (PNAs) to target genome modification is the core technology of Helix Therapeutics. Triplex DNA occurs when oligonucleotides bind as third strands in a sequence-specific manner within the major groove of the double helix. This event stimulates recombination in human cells when combined with donor DNA molecules. This technology has been termed “minimally invasive” gene repair, as gene modification likely occurs in the absence of induced DNA double strand breaks or DNA damage. Using a series of chemical modifications, the intracellular effectiveness of TFOs has been progressively enhanced. Helix Therapeutics is focusing this technology on the treatment of common genetic diseases, including sickle cell disease (SCD),
b-thalassemia, and Gaucher Disease.