Therapy Analysis - microRNA
Looking to the future
In the chequered history of nucleic acid therapeutics, many ‘next-big-thing’ technologies have come and gone, and only a handful of therapeutics have struggled through to the market. Despite over two decades of intense research effort, only two vector-based gene therapies have ever been launched (one in China, one in the Philippines), and the track record for therapeutic oligonucleotides is equally disappointing – one antisense drug and one aptamer. However, the advances in DNA delivery technology resulting from this massive research effort have not been in vain – the lack of visible success on the market belies an increasingly robust late-stage pipeline for gene and oligonucleotide therapies – and certainly the rapid progress of a siRNA therapeutic to Phase III development within a four-year space would never have been achieved with ‘old’ oligonucleotide technologies. Thus it may be a prime time for miRNA inhibitors and mimetics to succeed on the back of these advances.
On the flip side, the very mechanism by which miRNA operates is doubtless destined to turn a few drug development ideas upside-down. The relentless emphasis over the last few decades on increased precision in drug targeting as being the most desirable way forward could certainly be revisited – the smallest fragments of DNA may have the most profound and far-reaching implications in fine-tuning and control in multiple tissues and disease processes. Perhaps we should have been looking between the genes all along, rather than directly at them.
Jan Beal
Biotech Editor
Image courtesy of Narayanese under the GNU free documentation license 1.2
