Gene therapy has been in the headlines of the healthcare sector for many years now. Investment rounds worth hundreds of millions have been closed. Large pharmaceutical companies acquire biotech startups to add new therapies to their portfolios. But what are the actual facts between all the hype?
Find out below which therapies have made it through all phases of clinical trials, what kind of diseases are being addressed, the numbers in market value and investment, and the future outlook based on the current number of research projects.
Current landscape of gene therapies
At present, there are 26 approved gene therapies [1], of which the majority are in the field of oncology. In addition, during the first two quarters of 2023, 2 therapies have been approved (Elevidys and Vyjuvek). Both treat rare diseases with a genetic origin.
The graphic of approved therapies shows a slight upward trend in the number of approved therapies per year in the period from 2015 to 2022. This confirms that gene therapy is starting to take off. It may seem that approving 5 or fewer treatments per year is a small number, but bare in mind that gene therapy is a relatively young technology and that innovation in the biopharmaceutical sector takes time due to strict quality, safety, and efficacy requirements.
Where is gene therapy being used?
With cancer – in its various forms – being one of the most common diseases in the world, it is logical that most innovation is aimed at combating this problem. And gene therapy is no exception to that. However, the other area where research efforts are being focused is on rare diseases. Those that affect a very small number of people. Even so, many of these rare diseases are rare forms of cancer.
Rare diseases have – in more than 80% of cases – a monogenic origin, meaning they are caused by mutations in a single gene [2]. Being able to attack this problem at its origin, and correcting this genetic defect, will in many cases mean ending the disease. And that is exactly what gene therapy can do.
Other areas of great interest are also being tackled – although with a smaller number of projects – such as diseases of metabolic, neurological, cardiovascular, or immunological origin.
Let’s talk numbers: market and investment
In 2022 the CGT (cell and gene therapies) market was $5.3 billion in size. The forecast is promising. According to Deloitte, the market is set to quadruple by 2027, growing to $20 billion [3].
These figures can seem small when compared to the total sales of the global biopharmaceuticals market (about $350 billion in 2022). But, as we have said before, we are at the beginning of a curve that will surely have an exponential trend. CGTs will represent an increasingly large share of the biotech market.
The level of private investment is not to be underestimated. Between 2010 and 2021 the increase in investment in gene therapies has been 59% per year. This means that it has gone from a couple of hundred million a decade ago to almost $30 billion in 2021.
It is estimated that currently one third of all private investment in the life sciences world is earmarked for gene and cell therapies. Given these figures, there is no doubt that many companies see huge opportunities in these two technologies. There is a big market at stake [4].
Clinical trials in progress
After looking at the investment numbers, there is only one more element to predict the future for any area of the pharmaceutical sector: clinical trials. Currently, there are thousands of active clinical trials using some form of gene therapy.
If we take a snapshot in time, in Q2 of 2023 the number of clinical trials with gene therapies totaled 2,075 [5].
The historical success rate of clinical trials for biopharmaceuticals is 7.9%. With the numbers shown in the table, we would expect that about 120 of the therapies in the preclinical phase will reach the market. This would represent a significant increase in the number of therapies currently approved per year.
In addition, the number of registered clinical trials with gene therapies is also on the rise, with China and the United States leading most new projects.
We have seen how gene therapies have already reached the market. They have the potential to cure many types of cancer, as well as several chronic rare diseases for which there was little hope a few years ago. The number of deals between companies, and the value of these, shows that the private sector continues to bet heavily on gene therapy. Finally, we see that research is beginning to be tested in patients with several clinical trials suggesting that we will see a significant increase in the number of approved gene therapies in the coming years.
We do this analysis to see what the next few years hold for us. We will continue to provide the best solutions in pharmaceutical engineering based on our extensive knowledge of process engineering and biotechnology.
At Klinea it is clear to us: one cannot talk about the future of biopharmaceuticals without mentioning gene therapy. And although this means big changes for the traditional pharmaceutical industry, we will be ready to continue innovating together with our customers. If you are interested in learning more about gene therapy processes and how we can support you, contact us: klinea@klinea.eu
Sources:
[3] The Evolution and Future of Cell & Gene Therapy | Deloitte US
[4] Cell & Gene Therapy Market Investment Outlook in 2022 & beyond (cellandgene.com)
[5] Fatemeh Arabi, Vahid Mansouri, Naser Ahmadbeigi, Gene therapy clinical trials, where do we go? An overview, Biomedicine & Pharmacotherapy, Volume 153, 2022, 113324, ISSN 0753-3322, https://doi.org/10.1016/j.biopha.2022.113324
