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news.Leiden University Medical Center and Prosensa have reported positive results from the clinical study with RNA-based therapeutic PRO051 in four patients with Duchenne muscular dystrophy, in which PRO051 has restored dystrophin expression in the treated muscle fibers of all four patients tested.
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In this study, Duchenne muscular dystrophy (DMD) patients between 10 and 13 years of age received a single injection of PRO051 (a 2-’O-methyl antisense oligonucleotide) in a small area of a muscle in the lower leg. In a biopsy taken four weeks later, novel dystrophin expression was observed in the vast majority of muscle fibers with protein levels that are expected to be clinically relevant.
Following this first clinical proof-of-mechanism, Prosensa has started the preparations for a Phase I/II clinical study to explore the effects and safety of PRO051 after repeated systemic injections.
Gerard Platenburg, CEO of Prosensa, said: “We are excited to have demonstrated clinical proof-of-mechanism and will be progressing PRO051 into systemic Phase I/II trials in order to assess the broader benefits available to patients.”