A Synthetic Chemically Modified siRNA Targeting Caspase-2 as a Therapeutic Agent for Ocular europrotection

Elena Feinstein, M.D., Ph.D., CSO, Quark Pharmaceuticals
Caspase-2 is a pro-apoptotic gene specifically expressed and activated in retinal
ganglion cells following acute optic nerve injury or intraocular pressure increase.
Intravitreal administration of QPI1007 (a syntehticsiRNA targeting caspase-2)
demonstrated local RNA interference, lack of inflammatory effects and excellent
preclinical and clinical safety profiles. Neuroprotective activity of QPI1007 has
been demonstrated in five preclinical animal models involving RGC loss. In
human Phase I trials in NAION patients, a single intravitreal injection of QPI1007
within 2 weeks following disease onset prevented further visual deterioration.

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