RNA Therapeutics: A Novel Approach to Treating Diseases

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs. Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein. RNA aptamers, on the other hand, are short RNA molecules that can bind to a specific protein, affecting its function.

RNA therapeutics include mRNA, siRNA, and antisense oligonucleotides, revolutionizing medicine. mRNA vaccines (e.g., COVID-19 vaccines) instruct cells to produce proteins, while siRNA and antisense oligos regulate gene expression. These therapies hold promise for treating genetic disorders, cancer, and infectious diseases, marking a new era in personalized medicine.

The above figure presents various types of RNA therapeutics

Below, I’ve elaborated on three main categories of RNA therapeutics:

  • mRNA Vaccines and Therapeutics: Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA and is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent. It is important to highlight that IVT mRNAs are structurally similar to natural mRNAs and can be used to express proteins through genetic engineering. Furthermore, these drug candidates can be used to develop precise and individualized therapies that allow patients to produce therapeutic proteins in their own bodies.
  • RNA interference (RNAi) Therapeutics: Fundamentally, RNAi is a natural process of post-transcriptional gene silencing, involving short strands of nucleic acids. Cells use this process to silence and / or inhibit gene expression, via targeted degradation of specific (unwanted) mRNA molecules. From an application perspective, the gene specificity of RNAi is the primary reason for it being used in the development of therapies. In theory, RNAi based therapeutics are capable of treating a myriad of disease indications, such as age-related macular degeneration (AMD), hepatitis C and various forms of cancer, which are actually hard to treat, using conventional pharmacological options.
  • RNA Aptamers: RNA aptamers are single stranded oligonucleotides, that are capable of binding to an array of molecular targets, including proteins, peptides, carbohydrates, small molecules, toxins, and live cells. It is worth mentioning that aptamers are present in a wide range of shapes due to their high versatility and tendency to form helices and single-stranded loops. Additionally, the binding of aptamers and target recognition is driven by tertiary sequence, which determines the degree of three-dimensional, shape-dependent interaction, hydrophobic interactions, base-stacking, and intercalation. Owing to the various advantages offered by aptamers, as compared to antibodies, RNA aptamer drugs have garnered considerable attention of various stakeholders in the healthcare industry and the domain has witnessed a significant rise in the investment activity.

Key Historical Milestones for RNA Therapeutics

Although the concept of RNA therapy has been around since the mid-20th century, the field has witnessed a significant boost in recent years, resulting in approval of therapies across various therapeutic areas. The key milestones and historical evolution of RNA therapeutics have been presented below.

Key historical milestones for RNA therapeutics include the discovery of RNA interference (RNAi) in 1998, the development of small interfering RNA (siRNA) as a therapeutic tool, and the FDA approval of the first RNA-based drug, Onpattro, in 2018. These advancements mark pivotal steps in harnessing RNA for innovative therapeutic applications.

Advantages of RNA Therapeutics

The potential of RNA therapeutics lies in its ability to target diseases at their genetic roots, enabling more precise and effective treatments. One of the major advantages is that RNA therapeutics can bind to the mRNA and lead to its degradation, thereby preventing the translation of disease-causing proteins. Additionally, RNA therapeutics can be easily integrated with existing therapies, making them a promising option for combination therapies. Unlike traditional small molecule drugs that can have off-target effects and cause unintended side effects, RNA therapeutics can be tailored to specific disease targets, making them potentially safer and more effective. As a result, RNA therapeutics has the potential in addressing the unmet needs in the field of oncology, specifically for drug resistance. In addition, by targeting specific genes or proteins, RNA therapeutics can be employed or the treatment of rare genetic disorders. RNA therapeutics offer several advantages over small molecules.

Limitations of RNA Therapeutics

There is a lot of optimism surrounding RNA therapies, however, over time certain concerns have also come into light and are being actively addressed by several medical researchers and drug / therapy developers. Some of the key concerns associated with RNA therapeutics are as below:

  • Delivery-related Concerns: RNA-based molecules are unstable due to their large molecular size and negative charge. These characteristics hinder the passive diffusion of RNA-based therapeutics through the lipidic bilayer of a cell membrane, thereby impeding the efficient delivery of the RNA molecule to the intended target site.
  • Stability-related Concerns: RNA molecules are prone to degradation by ribonucleases, which makes them inherently unstable. This creates hurdles in storing and transporting RNA therapeutics and poses a challenge for their effective use in clinical settings, requiring specialized techniques to maintain the integrity of RNA molecules.
  • Toxicity-related Concerns

Although there have been several advancements related to these therapies, immunogenicity is still a major concern as toxicity can arise from improper target recognition or immunogenic response against RNA-based lead molecule or its delivery system, leading to impaired translation of RNA-based lead into therapeutic proteins.

  • Regulatory Concerns

Regulatory authorities are exposed to an extremely difficult challenge as these therapies need to be regulated closely to ensure patient safety and subsequently, also need to move to market as quickly as possible, requiring a new set of regulatory standards for these therapies.

Future Perspectives

Owing to the recent success of RNA-based therapeutic interventions in combatting the COVID-19 pandemic and their potential to actualize the concept of personalized medicine, the companies involved in the RNA therapeutics domain have witnessed a surge in investment activity. Subsequently, capital investments worth more than USD 16 billion have been made in this domain, with the majority (75%) of the amount raised by the RNA therapeutic companies, so far, through venture capital financing and public offerings. Moreover, recent advancements in manufacturing processes and delivery technologies have made RNA therapeutics more accessible for widespread use. The manufacturing processes have become more cost-effective, and various delivery technologies, such as lipid nanoparticles, have been developed to enable efficient delivery of RNA molecules to target tissues. Given these numerous benefits offered by RNA therapeutics and the ongoing efforts to develop therapies for existing unmet needs, we anticipate RNA therapeutics market to present numerous lucrative investment opportunities in the future.

About Author


Ronit Sharma is an accomplished business research and competitive intelligence professional with over six years of experience in the pharmaceutical and healthcare industry. As a team leader at Roots Analysis, he has authored numerous multidisciplinary market research reports, and led the efforts on several bespoken consulting assignments, providing valuable insights into the latest innovations in healthcare and the digital transformation of the pharmaceutical industry. Ronit’s exceptional analytical skills and strategic thinking in the field contribute to the firm’s intellectual capital, empowering clients to make informed decisions in the dynamic pharmaceutical landscape. With a passion for staying at the forefront of industry advancements, Ronit specializes in identifying emerging opportunities for various stakeholders, leveraging his deep understanding of market trends and technological developments. He is committed to fostering an environment of excellence, creativity, and innovation within his team, encouraging collaboration and empowering team members to bring their best ideas to the table.

Recently, Ronit’s expertise has been recognized by the online news agency, where he was interviewed and featured as an emerging industry leader. Ronit holds a B. Tech degree in Biotechnology from Lovely Professional University and his research and thought leadership can be found in his professional blog and social media profiles, where he shares the latest insights and engages in meaningful discussions with industry peers. To ensure the highest ethical standards, Ronit openly declares no conflicts of interest in his work, ensuring unbiased and trustworthy contributions. His insights undergo rigorous editorial and peer-review processes, establishing his credibility as a thought leader within the pharmaceutical and healthcare domain. Ronit’s exceptional analytical and strategic thinking skills, coupled with his commitment to excellence, make him a valuable asset to any organization in the pharmaceutical and healthcare industry.

Email: ronit.sharma@rootsanalysis.com

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For detailed insights about this domain, check out our report on RNA Therapeutics Fundamental Analysis