Bringing gene knockdown to cancer treatment
A dozen candidates built on four gene silencing approaches have entered the clinic
A small group of companies aims to take gene knockdown technologies, most often used to treat rare genetic disorders, into the cancer arena. Collectively, the companies have brought at least 12 programs into early clinical testing, with three applying the technology to validated targets and the others using the tools to access new or difficult targets.
Antisense- and siRNA-mediated knockdown account for 10 of the 12 programs, while microRNA (miRNA) and short hairpin RNA (shRNA) technologies, which have yet to lead to an approved drug, each account for one. ...