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Pre-clinical siRNA data shows targeting of stress response gene may have therapeutic benefit for CNV

Researchers based at Pfizer’s Ophthalmology External Research Unit in San Diego, have published supporting animal data showing that a clinical stage siRNA experimental drug (PF-04523655) is capable of decreasing the incidence of clinical grade 3 or 4 choroidal neovascularization (CNV) lesions by approximately 60% (P=0.053). In addition, the researchers showed a dose dependent inhibition of the siRNA target gene (the RTP801 stress response gene)(P < 0.01). The researchers suggest that the reduced RTP801 expression is consistent with the RNA-induced silencing complex (RISC) mechanism of action and that the data may be useful to clinical study design.