RNAi lost and found in translation: a breakthrough therapeutic area
Nature Biotechonology has recently reported on the progress industry is making in understanding the benefits of RNAi as a treatment modality, news that is especially promising for rare diseases patients. RNAi is a part of the “gene silencing” technology where the introduction of small strands of RNA (20-24 base pairs) binds to its complementary RNA in vivo, forming a double stranded RNA stopping the protein formation of that RNA.
While this methodology has been successfully tried and tested in fundamental research, its foray into clinical research has been markedly sketchy. The authors describe how start-ups were developing therapeutic platforms using this technology and big pharma followed suit, which unfortunately, did not bear fruit. However, the initial setbacks were tempered by recent success of Alnylam in using RNAi –with effective delivery vehicle to the liver – in not only knocking down target genes but also causing a therapeutic effect in humans, has been good news for patients suffering from rare liver diseases. The authors present how Alnylam is now courted by Genzyme boosting its ability to bring this therapy to the market a possible dream. The article also describes the case of fellow RNAi specialist Dicerna, who has also netted $USD 92.9 million in an IPO encouraged by the success of Alnylam.
According to the authors, these ventures into newer and advanced methodologies to treat diseases, by looking beyond conventional drugs is the sign of times to come as “companies pursue gene therapy, gene editing and protein misfolding correction, to name a few, are reminding investors of what it was like in the genomics era, to dare to imagine transformative advances”.
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