Tag: SKY-0515
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Skyhawk’s SKY-0515 Posts 1.59-Point cUHDRS Advantage at 15 Months
Skyhawk’s SKY-0515 showed a 1.59-point cUHDRS advantage over an external control at Month 15, alongside sustained reductions in mutant huntingtin and PMS1 messenger RNA.
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How SKY-0515 Uses RNA Splicing to Lower HTT and PMS1
SKY-0515 modifies RNA splicing to lower full-length huntingtin and PMS1, potentially addressing both toxic-protein production and somatic CAG-repeat expansion.