Adding a further feather to the industry’s cap are the latest statistics released by the Cell and Gene Therapy Catapult (CGTC) showing that the number of clinical trials increased by 45% in 2019, compared to the previous year.
In total, there are 127 ongoing trials in the advanced therapies medicinal products (ATMPs) area, representing 12% of ongoing global trials.
Keith Thompson, CEO of the CGTC, noted that the number of trials has been growing by an average of 25% year-on-year since 2013.
However, the report did qualify that the new figures have been generated by a new automated methodology, which uncovered trials missed by previous reviews’ manual searches.
UK seen as a leader
Broader than this, the CGTC noted that there appears to be a growing recognition of the UK’s position as a leader in the field, with a majority of commercially sponsored trials being backed by international organizations.
As the pipeline within the country continues to progress, the proportion of clinical trials that are sponsored by commercial organizations has also increased to 77%, up from 25% in 2013.
In terms of the progression of the trials themselves, the greater part are in the recruitment phase, which is a similar pattern to previous years; however, the CGTC added that there has been a larger than normal shift into this stage of the clinical trial set-up.
Explaining why this is the case, the report stated that this could be as a result of “trials in the UK…quickly moving through planning and regulatory approvals reaching the recruitment phase at a faster rate than previously.”
In terms of what therapeutic candidates are being progressed and the target, the majority are gene therapies and the most common therapeutic area is oncology.
The focus on oncology (39% of trials) is no surprise given the global outlook for this therapeutic area. In addition, the gene therapy area has also experienced landmark approvals, including Novartis’ Zolgensma (onasemnogene abeparvovec) and bluebird’s Zyntelgo (autologous CD34+ cells encoding βA-T87Q-globin gene).
In terms of delivery, in-vivo clinical trials are dominated (89%) by gene therapies utilizing adeno-associated viral (AAV) vectors.
The only negative on the results are this reliance on AAV vectors leading to bottlenecks at the leading producers, with waiting lists for capacity. As the clinical trials move closer to larger scale studies and to commercialization, this will only increase the pressure on supply.