CRISPR and Trends in the Gene Therapy Space

15 Oct

By Cole Bunn, Research Analyst, LSN


The allure of gene therapy is very strong, mostly due to its potential to cure, not just treat symptoms, of any disease with a genetic basis. The so-called “magic bullet” treatment—that if perfected, would revolutionize medicine (not to mention several other industries)—has drawn a lot of attention since its inception, and rightfully so. The field has seen many advances as well as setbacks, leaving investors and other industry stakeholders with a lot to ponder and speculate. All things considered, most leaders in the life science industry believe that major breakthroughs are right around the corner, including big pharma who has partnered with several of the biotechs developing these therapies.

The relatively recent gene editing technologies that have surfaced, with the CRISPR/Cas9 technology getting the most attention (more on this later), have once again put the spotlight on gene therapy. Given that gene therapy efforts are still moving forward, we have decided to utilize the Life Science Nation (LSN) company and investor platforms to create a sample of the space and look at some of the current trends.

The LSN company platform, which tracks 30,000 biotech, medtech and healthcare IT companies around the globe, provides a nice sample of what is happening in the industry. Using the platform to look at gene therapy companies by country, the LSN platform found 68 companies that were operating in the gene therapy space. As shown in Figure 1, the U.S. led the way with 35 gene therapy-related companies, with most others spread out fairly evenly across Western Europe.

figure 1

Figure 1 | Companies with Gene Therapy Assets by Country Data from LSN Company Platform | October 13, 2015

Treatment of genetic disease is usually what comes to mind when thinking about gene therapy, but this technique also has utility in treating infectious disease. Gene therapy for infectious diseases requires the introduction of genes designed to specifically block or inhibit the gene expression or function of gene products, such that the replication of the infectious agent is blocked or limited [1]. In addition, gene therapy techniques have several uses in research, including studying protein function and creating genetically modified cell and animal models.

The rekindled interest in the viability of these treatments, given new technologies and more clinical data, is bringing an influx of capital into the space. Using the LSN company platform, we’ve looked at a sample of gene therapy financings. Figure 2 shows the number of funded gene therapy assets, by indication, from the five largest funding rounds over the past three years.

figure 2

Figure 2 | Funded Gene Therapy Assets by Indication Data from the LSN Company Platform | October 13, 2015 *Data of the five largest gene therapy funding rounds ranged from $45M-$75M

The new gene editing technologies, CRIPSR/Cas9 in particular, has excited researchers and certainly peaked the interest of some noteworthy investors and big corporations alike. So what’s the hype? What is CRISPR?

The CRISPR/Cas system is a prokaryotic immune system that confers resistance to foreign genetic elements such as plasmids and phages [2]. The gene editing technology known as “CRISPR” is a bacterial immune system that researchers have learned how to exploit in order to edit specific genes of interest in plants and animals. In nature, the system works by chopping up pieces of invading viral DNA, inserting some of these sequences into its own genome so that the next time the viral DNA is encountered, it is recognized (based on the specific DNA sequence – this is KEY to technology) and destroyed via the Cas enzyme. Simply put, using the system to actually edit genes works like this – researchers are able to guide the Cas enzyme to a specific gene sequence, which cuts the DNA strand in the specified location, allowing for editing/correcting the sequence.

The CRISPR technology has brought a lot of excitement along with it due to its targeted nature and compact system, among other advantages over conventional gene therapy technologies. However, this new technology isn’t exempt from some of the roadblocks that previous gene therapies encountered, most notably delivery of the gene editing therapeutic itself.

It is yet to be seen if gene therapy will be a realized therapy, but there is no doubting the enormous potential of the field, given some of the inherent issues are resolved (not to mention ethical implications and possible pricing scenarios). There are a lot of gene therapy hopefuls out there and most everyone from angel investors to big pharma have a horse in the race. If you’d like to learn more, we find this article useful to read. The LSN team will keep you informed as we continue to track trends in the gene therapy field.

  1. Bruce Bunnell and Richard Morgan, Gene Therapy for Infectious Diseases. Clinical Microbiology Reviews (1998) 11(1): 42–56.
  2. CRISPR.  Wikipedia: The Free Encyclopedia. Wikimedia Foundation, Inc., 10/14/2015.

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