IRBM cited in UniQure publication on using HTT-lowering therapies to slow down neurodegeneration in Huntington’s Disease
UniQure has just published a study in Science Translational Medicine using a Huntingtin (HTT)-lowering therapy.…
One of the studies included in the edition detailed the design, synthesis and evaluation of HIV-1 nucleocapsid protein inhibitors (NCIs). This protein is highly conserved in HIV-1 strains and is involved in multiple steps of the HIV-1 replication cycle, thus becoming a desirable drug discovery target for treating HIV, particularly the drug resistant forms.
We designed and developed bifunctional NCIs that simultaneously target the hydrophobic pocket and N-terminal domain of the nucleocapsid protein. This was confirmed by in vitro inhibition assays and characterization of the mechanism of action in infected cells lines. Furthermore, multiple assays helped confirm the antiviral activity of these NCIs, with no cytotoxicity for the most potent compounds identified.
These compounds represent a valuable starting point for future optimization, demonstrating exciting potential for the treatment of HIV.
Read more here.