Cambridge, US, based Wave Life Sciences Ltd reported positive proof of mechanism data on 16 October from a Phase 1b/2a study of an RNA editing oligonucleotide drug which restored levels of a critical protein in patients with alpha-1 antitrypsin deficiency, a genetic disease affecting the lung and liver. The estimated prevalence of the disease, with the mutation studied in the trial, is 200,000 in the US and Europe.
The drug, WVE-006, is designed to correct mutated proteins and restore them to normal functions within the body. Data reported by the company are from a first single dose cohort and included two patients with the disease. According to the company, the data are the first-ever clinical demonstration of RNA editing in humans.
Wave has developed oligonucleotides, which are short strands of DNA or RNA that can reduce, restore or modulate RNA by way of several different mechanisms. These include editing, splicing and silencing RNA. The primary function of RNA is to create proteins via translation. This is done by carrying genetic information that is translated by ribosomes into proteins necessary for processes in the cell. “Achieving the first-ever therapeutic RNA editing in humans is a significant milestone for our organisation, for our GSK collaboration and for the entire oligonucleotide field,” Paul Bolno, Wave’s chief executive, said in a prepared statement. GSK entered into an oligonucleotide discovery programme with Wave in 2022. It has an exclusive global licence to WVE-006.
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