Hybridon plans to start clinical trials in August with its genetic antisense drug, GEM 132, for the treatment of cytomegalovirus retinitis disease; it also offered new data on its flagship compound GEM 91 at the Euro-Biotech Forum in Paris, France last month.
Antisense technology utilizes segments of RNA or DNA called oligonucleotides. Current therapies concentrate on the protein once produced whereas antisense drugs target the messenger RNA, the "protein coder," and bind to it, rendering the mRNA useless and disabling it from manufacturing the pathological protein in question.
Hybridon uses advanced chemistry techniques to produce its compounds, changing the backbone of the oligonucleotide from phosphodiester to phosphothioate to make the compound more stable. This results in the drug's increased cellular uptake, potency and duration. The compounds are also sequence-specific, so are more effective in the recognition of a protein, and therefore less toxic to the cell.
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