The evolution and promise of gene therapies

Dr David Baram from gene therapy company EmendoBio provides a snapshot of the history of therapeutic genetic engineering, explaining the early pitfalls and reasons for recent renewed optimism.

A long and winding path

The early years of gene therapy were characterised by an era of overwhelming optimism, with many believing that advances in DNA manipulation would mean the realisation of curative therapies for multiple disease states. Unfortunately, numerous clinical trial failures shattered this expectation early on, leaving the great potential of gene therapies as a cure-all for human genetic diseases unfulfilled.

At the outset, the application of gene therapy technologies was expected to be straightforward, but tragically, we quickly realised that this was not necessarily true. Notable early clinical trial failures were especially devastating, including the death of Jessie Gelsinger and the development of leukaemia in numerous children as a result of treatments. With these tragic deaths and illnesses, the associated off-target effects derailed the therapeutic potential of genetic modification, sowing seeds of doubt about how realistic the dream of gene therapies was — can it ever really be safe to manipulate the genome?