Jul 12

A respected RNAi therapeutics organization.

‘Further, the new data in a model of ocular amyloidosis reveal additional potential clinical indications in our overall ALN-TTR program. As we obtain final regulatory and ethics committee approvals, we anticipate initiating our Phase I study with ALN-TTR01 in ATTR sufferers, which remains on the right track to start in the next few months.’ Previous pre-clinical studies reported by Alnylam possess demonstrated the power of TTR-particular RNAi therapeutics to mediate potent and long lasting silencing of both wild-type and mutant forms of the TTR gene in rodents and nonhuman primates. In the first half of 2010, Alnylam expects to initiate a Stage I scientific trial with ALN-TTR01 in sufferers with ATTR.The study protocol called for independent evaluators who completed assessments to be unacquainted with all treatment assignments. Statistical Analysis On the basis of previous studies,10-15 we hypothesized that 80 percent of children in the combination-therapy group, 60 percent in either the sertraline group or the cognitive-behavioral-therapy group, and 30 percent in the placebo group would be considered to have had a reply to treatment at week 12. We established that we had a need to enroll 136 topics in each active-treatment group and 70 subjects in the placebo group for the analysis to get a power of 80 percent to detect a minimum difference of 17 percent between any two study groupings in the rate of response, assuming an alpha of 0.05 and a two-tailed test without adjustment for multiple comparisons.