Cell and Gene Therapies: Keep Calm and Carry On

A few months ago, we discussed the fact that cell and gene therapies were among the hottest topics of 2019. The development and manufacturing of biologics have shown rapid advancement in recent years – both in technological innovations and volume capacities. At the end of 2019, it was broadly recognised that new approaches such as cell and gene therapies were likely to dominate R&D efforts in the future.

Of course, the end of 2019 came before COVID-19 made its mark on the world at large. Therefore, as with many things, expectations and predictions for cell and gene therapy are being considered anew. Almost all biopharma companies and CROs are experiencing disruptions, and across the whole pharma space it has been estimated that more than 600 clinical trials are being affected by the pandemic. However, there are suggestions that cell and gene therapy trials might be less affected than those in other sectors.

In fact, according to Ly Nguyen-Jatkoe, Commercial Strategy Director at Pharma Intelligence, speaking at a recent web-based event hosted by CPhI, of 642 trials across all therapeutic areas that have been reported to be temporarily suspended, delayed, or terminated due to COVID-19, only 26 trials (6%) are in the cell and gene therapy space. A key factor contributing to this low number is that gene therapies are one-time treatments. Therefore, patients in fully-enrolled trials have already received their treatments and there is no on-going treatment regimen to interrupt. Even for those trials that are still enrolling patients, the fact that these therapies meet high unmet needs means that a single visit to hospital to receive a potentially successful one-time treatment could far outweigh any risks of attending clinic during a pandemic.

Another area of potential concern across the pharma sector is delay in regulatory approvals. While FDA approvals (and many others) are still largely on track at the time of writing this blog, it is feasible to consider the likelihood that product launches may be delayed if medical facilities are restricted in their abilities to see patients and provide new treatments. Again, however, due to them addressing high unmet needs, and their one-time treatment modalities, such issues might not present barriers for any newly-approved gene therapies.

Taking another viewpoint, we might also ask how innovative cell and gene therapies are being employed to treat the virus, and how this is affecting demand for new R&D in the sector. While some of these technologies have been viewed with an element of mistrust by the general public in the past, recent risk analysis data have indicated that – as these technologies may now be leveraged to fight the pandemic – there has been an upsurge in their acceptance. However, during the recent web-based event hosted by CPhI, Ly Nguyen-Jatkoe suggested that while non-genetically modified cell therapies and gene therapies are being developed to treat COVID-19, lack of scalability and expected high costs will most likely hamper their uptake in the real world. There are currently two genetically engineered therapies at preclinical stages, but the time taken to develop these products, alongside their high price tags, is unlikely to make them a competitive treatment option if antivirals, antibodies and vaccines become available.

Of course, we are all hoping for a vaccine. Medical supplies suppliers are also trying for it. During the recent web-based event hosted by CPhI, Helen Gu, Business Development Director at GenScript ProBio, explained that there are currently around 115 COVID-19 vaccines in development, six of which have reached clinical development Phase I or II. Those six advanced candidates include a couple of DNA and mRNA vaccines.

Nucleic acid vaccines have several potential advantages over conventional vaccines, including the ability to induce a wider range of immune response types. This could be particularly important when dealing with a ‘rapid mutator’ such as COVID-19. Therefore, if novel vaccines such as these should prove successful, their success and unmitigated global demand will undoubtedly be virus’s greatest impact on the gene therapy sector.

In summary, it seems that the impact of COVID-19 on cell and gene therapy clinical trials and approvals is likely to be minimal, and the lack of scalability and the high costs of non-genetically modified cell therapies and gene therapies make them unlikely to dominate the market. The greatest potential impact of the pandemic on the cell and gene therapy sector might be through the uptake of a novel nucleic acid vaccine – if one is shown to be successful against the virus in clinical trials.

And of course, even more importantly if this should come to pass, cell and gene therapy will have had the greatest impact on COVID-19, providing the vaccine that we are all hoping for. 

Author biography

Sarah Harding, PhD

Sarah Harding worked as a medical writer and consultant in the pharmaceutical industry for 15 years, for the last 10 years of which she owned and ran her own medical communications agency that provided a range of services to blue-chip Pharma companies. She subsequently began a new career in publishing as Editor of Speciality Chemicals Magazine, and then Editorial Director at Chemicals Knowledge. She now focusses on providing independent writing and consultancy services to the pharmaceutical and speciality chemicals industry.