Feb 20, 2023
InsightAce Analytic |
Biotech companies that use messenger RNA (mRNA) in their therapies are changing how medicine and industry operate. The demand for personalized medicine and the need to quickly respond to emerging pandemics such as COVID-19 has made this approach particularly attractive because it allows for flexibility and speed in the development of new treatments. Which makes some of us ask this question - what is mRNA?
Messenger ribonucleic acid (mRNA) is a type of genetic material that plays a crucial role in synthesizing proteins within cells. It acts as a messenger, carrying the genetic code from DNA to the ribosomes, where proteins are synthesized. mRNA is an essential component gene expression process, by which the genetic information in DNA is used to produce the proteins needed for cellular function.
The mRNA industry has seen significant growth and development over the past decade, with increasing interest and investment in the field. mRNA, or messenger ribonucleic acid, plays a crucial role in translating genetic information from DNA to proteins in cells. This process is important for the proper functioning of all living organisms, and advances in mRNA technology can revolutionize how we approach a wide range of diseases and disorders.
Several driving factors have contributed to the growth and development of the mRNA industry. Some of the key driving factors include:
mRNA technology can revolutionize healthcare by providing a new approach to treating diseases and conditions. This has led to significant investment and research in the field and increased interest from pharmaceutical companies and biotech firms.
The development of mRNA vaccines has been a major driving factor for the mRNA industry. mRNA vaccines deliver a piece of genetic material (mRNA) to the body that encodes for a specific protein or antigen. Once the mRNA is inside the cells, it is translated into the protein, stimulating an immune response. This immune response helps to protect against future infection by the targeted pathogen.
mRNA technology is being explored as a way to treat various types of cancer. Cancer cells often produce abnormal proteins that contribute to the growth and spread of the cancer. By using mRNA technology to target and inhibit the production of these proteins, researchers hope to halt the progression of the cancer.
several genetic disorders are caused by mutations in specific genes that result in the production of abnormal proteins. By delivering a healthy version of the gene through mRNA technology, researchers hope to restore the protein's normal function and improve patient outcomes.
The increasing prevalence of genetic diseases has also contributed to the growth of the mRNA industry. As the population ages, the number of individuals with genetic disorders is expected to increase, likely driving the demand for mRNA-based therapies.
The emergence of COVID-19 has also played a role in driving the growth of the mRNA industry. The instant spread of the virus and the need for effective vaccines has led to significant investment in mRNA technology, with several mRNA-based vaccines for COVID-19 currently in development.
Overall, the mRNA industry is driven by the potential for mRNA technology to revolutionize healthcare and treat various diseases and conditions. The success of mRNA vaccines, the potential for mRNA technology to treat cancer, and the increasing prevalence of genetic diseases are all contributing factors to the growth of the mRNA industry.
There are currently three major categories of mRNA-based agents in development: prophylactic vaccines, therapeutic vaccines, and therapeutic drugs.
Prophylactic vaccines are expected to dominate the mRNA market over the next 15 years due to many pipeline assets and a higher probability of success than other vaccine modalities. Currently, 77% of mRNA companies have at least one prophylactic vaccine in their pipeline. Most prophylactic vaccine revenues are expected to come from COVID-19 products soon. In contrast, in the mid-term-to-long term, other vaccines for diseases like respiratory syncytial virus and influenza are expected to reach a wider population. mRNA vaccines are expected to have a good market share against other vaccine modalities due to their speed of development and high protection rates for COVID-19. The average top sales per pipeline asset for prophylactic mRNA vaccines is estimated to be around USD 800 million - USD 1 billion globally. (Excluding COVID-19 vaccines). While the total market size in 2035 is estimated to be around USD 12-15 billion (including COVID-19 vaccines).
Therapeutic vaccines utilizing mRNA technology are a niche space with significant commercial potential, particularly in the field of immuno-oncology (IO). One key trend in therapeutic vaccines is the focus on immuno-oncology, with many candidates targeting single or multiple antigens. Single-antigen vaccines, such as mRNA-5671 and BNT113, can target difficult-to-drug targets and justify high price premiums in small patient populations. However, they also face competition from peptide-based vaccines at an earlier stage of development. Most therapeutic mRNA vaccines are multi-antigen vaccines, such as PCVs, which have advantages over peptide-based vaccines and have provided initial positive safety and efficacy data. Companies are increasingly focusing on combination strategies with IO agents in earlier-line treatments for patients with a lower disease burden and competent immune systems.
Another trend is the growth in the number of mRNA PCV pipelines for an extensive range of cancer types, driven in part by China's rising role as a hub for IO R&D. However, mRNA therapeutic vaccines also face several challenges, including clinical and regulatory risks, long manufacturing lead times, and the need to address manufacturing and commercial pathway issues to unlock the market potential. Despite these challenges, the market for PCVs is expected to reach USD 7-10 billion in 2035.
Therapeutics is expected to be a promising area for mRNA products. Still, it is unclear whether they will have clinical advantages over other modalities, and there are also high clinical risks. Opportunities in this area will depend on technological advancements such as delivery systems and gene editing. For protein replacement therapy, mRNA platforms may have delivery advantages compared to standard recombinant protein strategies but face technical challenges such as lack of organ selectivity, the potential need for frequent delivery, and high immunogenicity. Initially, this may limit mRNA therapeutics to a subset of oncology indications where treatment agents utilize the immune system, such as an mRNA encoding a bispecific antibody that attaches to CD3 on T cells and a target antigen on tumour cells. For genetic disorders and rare diseases, mRNA therapeutics will likely need to compete with gene therapies at a more advanced clinical stage in genetic disorders. It may provide patients with a sustained supply of therapeutic proteins.
mRNA therapies have the potential to be highly effective and customized to the specific needs of individual patients, which makes them attractive for the treatment of an extensive range of diseases and conditions.
In addition, the demand for personalized medicine is expected to continue to increase as patients, and healthcare providers seek treatments that are tailored to the specific needs of individual patients. The combination of technological advances and government support is likely to drive the continued growth of the mRNA industry and the development of new mRNA-based therapies.
It is also likely that mRNA therapies will increasingly be used in combination with other personalized medicine approaches, such as gene editing and cell therapies, to provide more targeted and effective treatments.
Some mRNA therapies are being developed to stimulate the production of immune cells that can recognize and attack cancer cells. For example, Moderna's mRNA-based therapy, MRNA-4287, is being examined in a clinical trial for the treatment of solid tumours.
mRNA therapies have the potential to treat genetic disorders by providing a functional copy of a missing or faulty gene. For example, BioNTech and Genmab are collaborating on the development of an mRNA-based therapy for the treatment of Gaucher disease, which is a rare genetic disorder that affects the metabolism of fats.
mRNA therapies are being developed to stimulate the production of proteins involved in repairing damaged heart tissue. For example, the biotech company BioCardia is developing an mRNA-based therapy to treat heart failure.
mRNA-based vaccines have the potential to be customized to the specific needs of individual patients. For example, Moderna is developing an mRNA-based vaccine that can be customized to protect against emerging infectious diseases, such as COVID-19.
Advancements in technology have made it easier to design and produce mRNA therapies, which has helped to drive the development of new treatments.
As our understanding of genetics has increased, researchers have been able to identify specific genetic factors that are involved in the development of many diseases and conditions. This has led to the development of mRNA therapies that are targeted at specific genetic pathways, which could make them more effective and safer than traditional treatments.
There is increasing demand for personalized medicine as patients and healthcare providers seek treatments that are tailored to the precise needs of individual patients. mRNA therapies have the potential to meet this demand by providing customized treatments that are effective and have fewer side effects.
Many governments are supporting the development of mRNA therapies through funding and regulatory initiatives. For instance, the US National Institutes of Health (NIH) has a program called "mRNA Therapeutics: From Bench to Bedside" that is focused on advancing the development of mRNA therapies.
mRNA-based therapies have the potential to revolutionize the way we treat many diseases and conditions, and there is strong demand for these therapies from both patients and healthcare providers. But, mRNA is a fragile molecule that is sensitive to degradation, so it is important to develop methods that can effectively isolate mRNA from other cellular components while preserving its integrity. As a result, mRNA extraction and purification technologies have become increasingly important for the production of mRNA-based therapies, and the market for these technologies is expected to continue to grow.
There are several approaches that are being used or developed for the extraction and purification of mRNA:
Researchers have developed new methods and technologies that improve the efficiency of mRNA extraction and purification. For example, magnetic bead-based methods have been developed that allow for the rapid and efficient purification of mRNA from biological samples.
There has been a shift away from traditional methods, such as guanidine hydrochloride lysis and acid guanidinium thiocyanate-phenol-chloroform extraction, towards newer techniques that are faster and more efficient, such as silica-based purification and magnetic bead-based methods. These newer techniques often yield higher-quality mRNA samples with fewer contaminants, which makes them more suitable for use in downstream applications.
There has been a trend towards the automation of mRNA extraction and purification processes. Automated systems can reduce the time and labour required for these procedures. They can greatly increase the speed and accuracy of mRNA extraction and purification processes and have become increasingly popular in both research and commercial settings. Automated systems can also help to reduce the risk of contamination and improve the reproducibility of results.
Researchers have developed new methods and technologies that improve the sensitivity of mRNA detection and quantification. For example, real-time PCR (polymerase chain reaction) methods have been developed that allow for the detection and quantification of mRNA at very low levels.
There has been a craze towards the development of high-throughput mRNA extraction and purification methods. These methods allow researchers to process large numbers of samples quickly and efficiently, which is important for large-scale studies and the development of mRNA-based therapies.
Researchers have developed new methods and technologies that improve the specificity of mRNA extraction and purification. For example, methods have been developed that allow for the enrichment of specific mRNA isoforms or splice variants.
Overall, these trends have led to the development of more efficient, reliable, and sensitive methods for mRNA extraction and purification, which has facilitated the growth of the mRNA industry and the development of new mRNA-based therapies.