According to a story from Chemical & Engineering News, the American Society of Gene & Cell Therapy annual meeting was more crowded than usual this year. In fact every event at the meeting was overrun with people to the extent that tents had to be set up outside of the venue to accommodate them all with live streams. The excitement is a reflection of the massive spotlight that gene therapy has gotten in recent years as the first forms of this innovative treatment approach enter the market.
The size of the annual meeting has doubled in the last two years. This year, it is estimated that around 4,500 people are in attendance. The excitement around gene therapy is perfectly understandable as it carries the capability to treat genetic diseases far more effectively than any earlier methods. Diseases that at one point were considered untreatable and lethal could suddenly become far less daunting.
As the potential of gene therapy became more apparent, the popularity in undertaking such as therapy from drug companies has also increased. At this juncture, there are over 100 clinical trials that are testing a gene therapy of some kind. Many of these therapies are also being developed for severe rare diseases.
The results from these treatments can also be remarkable and even emotionally powerful. An example at the event includes a child with X-linked myotubular myopathy, a devastating genetic disorder that causes extreme muscle weakness. Prior to therapy, the boy couldn’t really move and needed a ventilator to breathe. A later video shows the same patient moving and full of energy after a successful gene therapy treatment. It is hard to deny the miraculous nature of the some of the results.
There are still a myriad of ways in which gene therapy is in need of improvement. The AAV, or adeno-associated virus, delivery system that many gene therapies use definitely isn’t perfect yet.
Many of the attendees are business people and investors in addition to the more traditional audience of scientists and academics, who are committed to watching the latest developments closely. The age of gene therapy may very well soon be at hand.
