Published April 14, 2023 | 3 min read
Key Points
- Disruptive technologies in genetic medicine include mRNA, the power behind successful COVID-19 vaccines, and early data shows promise in using it to tackle melanoma.
- After many setbacks, siRNA technology is starting to deliver new treatments, with potential in areas such as cardiomyopathy.
- Gene editing is another fast-moving and highly promising field, and with some 6,000 monogenic diseases, where a single gene is the cause, the potential applications are huge.
- With an estimated 27 genetic medicines already approved, companies who can combine the right choice of intervention with strong execution stand to benefit.
Melanoma is a target for mRNA vaccines
Science has understood the genetic underpinnings of disease for some time. Now it is finally producing the tools that allow us to tackle them, says Luca Issi, Biotechnology Analyst at RBC Capital Markets.
The game-changing power of mRNA technology, for example, is no longer in doubt. Millions of people have benefited, in the form of the COVID-19 vaccine. And now there is hope that mRNA could be used to protect people from other diseases as well.
Issi believes mRNA (messenger ribonucleic acid) is transforming the vaccine industry. Unlike traditional vaccines, mRNA does not entail injecting patients with part of an actual virus, but an artificial version based on genetic code. A key advantage is that these can be manufactured relatively quickly and cheaply.
“Every single patient will get a different cocktail of mRNA – this is the epitome of personalized medicine.”
Luca Issi, Biotechnology Analyst, RBC Capital Markets
Early data shows promising results for mRNA in tackling melanoma, albeit based on small studies. In this process, a patient’s tumor biopsy is compared with healthy tissue to produce mRNA designed to stimulate the immune system in a way that attacks the cancer, explains Issi.
“Every single patient will get a different cocktail of mRNA – this is the epitome of personalized medicine,” says Issi. While more research data is required, he adds, “there is reason to believe that this technology may not be limited to infectious disease and will create value for other indications.”
Investors who stuck with siRNA were rewarded
Meanwhile, advances continue in the related field of small interfering RNA, or siRNA. This technology has existed for many years – Andrew Fire and Craig Mello won a Nobel Prize for its discovery in 2006 – but setbacks in trials hindered development, says Issi.
“Most investors lost hope and left the space. A few decided to stay, and were rewarded in a pretty impressive way,” he notes. An innovation called GalNAc enabled safe delivery of siRNA, and several drugs have now been approved for use.
Today the technology is chiefly used in treatments for liver conditions, but companies are working on applications in other areas, including cardiomyopathy.
“The cumulative value for this field is now $50 billion, if you combine market cap and some of the M&A we have seen in that space,” Issi points out.
“Innovation takes time and never works in a straight line – but disruptive technology can ultimately succeed.”
Luca Issi, Biotechnology Analyst, RBC Capital Markets
Risks of gene editing appear low
Gene editing is another fast-moving and highly promising field. Among the pioneers are CRISPR Therapeutics, which is conducting trials on the treatment of sickle cell disease.
With some 6,000 so-called monogenic diseases, where a single gene is the cause, the potential applications are huge. As a “one and done” procedure – a gene is permanently edited to fix the disease – this technology has disruptive potential, says Issi.
However, development will take time and companies must tread cautiously with this technology. Gene editing cannot be reversed, and we do not fully understand its long-term implications. Concerns include the potential for unintended consequences on other genes and how it may impact the children of patients, though early data suggests the risks in both cases may be low.
Prioritization and execution are key to success
RBC estimates 27 genetic medicine drugs now have approval. “This could be just the tip of the iceberg. We continue to believe that genetic medicine is the next wave of innovation in biotech,” Issi declares.
Across the genetic medicine landscape, the winners may be those who prioritize the right conditions for treatment – and who have the capacity to carry through implementation.
“I think it’s important that with these more innovative technologies, we first go after indications with large unmet medical need, where you can justify taking the risks given the severity of the disease,” Issi concludes. And in a capital-intensive sector and a tough economic backdrop, “old school execution and strategy will matter too.”
