The RNA Therapeutics Market is estimated to be valued at USD 2.5 Mn in 2024 and is expected to reach USD 948 Mn by 2031, growing at a compound annual growth rate (CAGR) of 68% from 2024 to 2031.

The RNA therapeutics market is witnessing positive growth trends over the past few years. Factors such as increasing investment in RNA-based drug development, growing focuses on rare diseases and genetic disorders, rising prevalence of infectious diseases and cancer are expected to drive the demand for RNA therapeutics during the forecast period. 

Market Driver - Increasing Prevalence of Chronic Diseases Demanding Advanced Therapies

The growing burden of chronic diseases across the world has become a major healthcare challenge. Diseases like cancer, cardiovascular diseases, neurodegenerative disorders are affecting millions of lives. As per WHO, chronic diseases account for over 70% deaths globally each year. The need to effectively treat these complex diseases has accelerated the search for advanced disease modifying therapies.

RNA therapeutics is emerging as one of the most promising areas that can help address unmet needs in chronic disease management. Disease pathogenesis at molecular level involves various genetic and RNA expressions. Traditional drug molecules are not always able to target these underlying abnormalities precisely. RNA-based drugs offer targeted approach to intervene at RNA level and correct the causes of diseases. Technologies like RNA interference (RNAi), antisense oligonucleotides are enabling design of therapies against previously undruggable targets.

Success stories of RNA drugs in clinical trials are boosting industry's confidence in their potential. Patisiran became first RNAi drug approved by FDA for hereditary transthyretin-mediated amyloidosis. Investigational RNA drugs are showing encouraging results for conditions like spinal muscular atrophy, Duchenne muscular dystrophy. As disease understanding improves through research, more RNA drug targets are being identified. This expanding target pipeline is likely to increase success rates of future RNA drug developments.

Market Driver - Growing Investments in RNA-based Research and Development

Advancements in basic research have tremendously increased understanding of biological functions at molecular level involving RNA. This expanding knowledge base is continuously fueling innovation in RNA technologies. Significant number of investments are allowing companies to explore RNA's applications beyond traditional drug development.

Venture capital firms and healthcare investors see immense long-term potential in RNA as a new therapeutic class. Global funding in RNA-based companies exceeded US$ 2.5 Billion in 2021 alone. Several biotech startups focused exclusively on RNA are able to raise large Series A/B rounds indicating investor confidence. Big pharmaceutical players are also actively partnering and acquiring small RNA companies to build capabilities in this area.

In addition to private money, governments and not-for-profit organizations are majorly supporting RNA research through dedicated grants. National Institutes of Health, European Commission and other global health organizations consider RNA an important long term priority area. Their sustained funding is propelling important basic researches, facilitating collaborations and helping build infrastructure.

Growing R&D activity is expanding therapeutic applications of RNA concepts beyond drug development. RNA is being researched for complex gene editing, cell and gene therapy, diagnostics etc. Rapid technology convergence with fields like artificial intelligence, genome sequencing is opening up newer avenues. These multi-disciplinary innovations will create wider commercialization opportunities augmenting the attractiveness of RNA sector.

Market Challenge - High Cost and Complexity of RNA-based Therapeutics

One of the key challenges in the RNA therapeutics market is the high cost and complexity associated with RNA-based drugs. Developing RNA therapies requires sophisticated delivery technologies to effectively deliver nucleic acids such as mRNA to target cells and tissues. The delivery vehicles need to protect the fragile RNA strands from degradation or improper functioning in the body. This adds significantly to the overall development costs of RNA drugs. Furthermore, manufacturing RNA drugs at commercial scale also poses technical challenges. Mass producing therapeutic nucleic acids with required purity and quality needs specialized equipment and expertise. All these factors contribute to making RNA drugs much more expensive to develop and produce than conventional small-molecule drugs. The costs are further increased due to the need for life-long treatments for chronic conditions with RNA therapeutics. Additionally, the complex nature of these molecules means they have a relatively short shelf life requiring careful handling and storage. This complexity and the specialized manufacturing needs of RNA drugs keep them from being affordable and accessible to a majority of patients globally. Overcoming these challenges remains a key priority for players in this segment to realize the full potential of this innovative class of therapeutics.

Market Opportunity - Expanding Applications of RNA Therapeutics in Various Therapeutic Areas

One of the key opportunities for growth in the RNA therapeutics market is the expanding range of applications that these drugs are targeting. Traditionally, RNA drugs were majorly focused on genetic diseases for conditions where replacing or modifying a protein could offer therapeutic benefits. However, ongoing research success has opened up new avenues. RNA technologies are now being explored for developing treatments in more prevalent therapeutic areas like oncology, cardiology, metabolic disorders, pain management and vaccines. Considering the large patient pools and worldwide burden of diseases in these areas, RNA drugs effectively developed here could cater to massive markets.

Additionally, the programmability of RNA allows developing personalized treatments targeting specific mutations or biomarkers of diseases. This brings the possibility of effective and tailored therapies for subgroups within disease conditions. With advancements reducing manufacturing and delivery complexities, more disease applications are expected to be added, thereby majorly boosting the commercial potential of the RNA therapeutics sector over the coming years.

Strategic partnerships have been one of the main strategies adopted by companies to develop and commercialize RNA therapeutics. For example, in 2018, Moderna collaborated with Merck to develop mRNA treatments for cancer and autoimmune disorders. This allowed both companies to leverage their strengths - Moderna's platform and Merck's development expertise. Such partnerships help companies expand their pipelines and access new technologies.

Acquiring other players has helped companies strengthen their position in this emerging field. For example, in 2021, Vertex Pharmaceuticals acquired privately held ViaTherapeutics for $160 million upfront. This gave Vertex access to ViaTherapeutics' proprietary lipid nanoparticle delivery platform to develop RNA therapies for CNS and liver diseases. The acquisition enhanced Vertex's capabilities in delivery technologies.

Companies like Alnylam, Ionis and Arrowhead have succeeded by focusing on developing proprietary platform technologies like RNAi, STm and TRiM that can be applied to multiple disease targets. For example, Alnylam's core siRNA technology underlies several of its clinical programs and partnerships, establishing the company as a platform leader in RNAi therapeutics. Such broad platforms attract multiple partners and make these firms go-to solutions in the space.

Early patenting: Aggressive patenting of core platform technologies and candidate programs has helped secure early leadership. As of 2021, Ionis had over 1,000 patents related to antisense therapies. Protecting intellectual property early reduces competition and allows firms to license their technologies widely. This boosts revenue visibility through upfronts and milestones.

Insights, By Type of Modality: Versatility and Efficacy of RNA Therapeutics

In terms of type of modality, RNA therapeutics sub-segment contributes the highest share of 55.6% in the market owing to its versatility and efficacy.

RNA therapeutics has emerged as a promising field in biopharmaceutical drug development owing to the versatility and flexibility it provides. RNA molecules can be designed to target any disease-related genes and be tailored to elicit specific cellular responses. This provides RNA therapeutics an advantage over small molecule drugs and antibodies. RNA therapeutics modulates the expression of target proteins by binding to complementary mRNA sequences and altering protein synthesis. This mechanism of action enables it to be used for treating a wide range of genetic disorders.

Further, RNA therapeutics holds potential for repurposing and reprogramming cellular activities. New delivery technologies allow production of RNA molecules within target cells, overcoming issues related to extracellular molecule stability and cellular uptake faced by early RNA drugs. Continued research on RNA structure optimization and conjugation with novel nanoparticles is expanding possible applications. Discovery of various classes of regulatory RNA has unlocked opportunities for developing therapeutics against previously undruggable targets. Overall, these advantages associated with RNA modality are driving its growth as the dominant segment in this field.

Insights, By Type of Molecule: Self-amplifying Property of Replicating RNA

Replicating RNA sub-segment commands the highest share of 20.3% in the RNA Therapeutics Market owing to its self-amplifying property.

Among different types of RNA molecules, replicating RNA stands out due to its unique ability to self-replicate inside cells. Replicating RNA gets amplified through viral replicase enzymes, yielding thousands of copies from a single input molecule. This self-amplifying property enables it to be highly potent and produce sustained therapeutic effects at low dosage. It overcomes the need for repetitive administration associated with non-replicating gene therapy vectors.

Additionally, replicating RNA has shown promising results for vaccination owing to its mimicking of viral infections. It stimulates both arms of the immune system by enabling antigen production as well as innate immune stimulation. This allows development of vaccines against infectious diseases with just one or two doses. Various replicon vaccine platforms are under research which can be rapidly adapted to emerging viral threats.

Overall, replicating RNA has proven significantly more effective than other RNA types in preclinical disease models due to its self-replicative advantage. This unique characteristic qualifies it as the dominant segment in the market for RNA therapeutics. Continuing innovation is likely to expand its applications in the coming years.

• The RNA therapeutics market is poised for significant growth driven by innovative RNA-based therapies, which offer enhanced therapeutic profiles and target a range of diseases previously considered undruggable. The market landscape features over 35 companies, with notable activity from start-ups and significant research in North America. The potential of self-amplifying RNA and tRNA therapeutics is being actively explored, with several companies engaging in partnerships to advance their pipelines. High funding and investment levels reflect strong interest and confidence in RNA therapeutics, supported by regulatory advancements and promising clinical trial results. The market's rapid evolution is expected to continue, driven by technological advancements and increasing demand for effective therapies.

The major players operating in the RNA Therapeutics Market include Alphavax, Arcturus Therapeutics, Atyr Pharma, Gritstone Bio, HDT Bio, Laronde Therapeutics, MiNA Therapeutics, Orna Therapeutics, Recode Therapeutics, Renegade Therapeutics, Replicate Bioscience, Shape Therapeutics, and VLP Therapeutics

• In February 2023, Tevard Biosciences partnered with Vertex Pharmaceuticals to develop tRNA-based therapies for Duchenne Muscular Dystrophy.
• In January 2024, Circular Genomics raised $8.3 million in series A funding for the world’s first circRNA-based clinical assay to improve depression treatment.
• In January 2023, Esperovax and Gingko Bioworks entered a product development agreement for circular RNA therapeutics targeting colorectal cancer.
• In January 2023, HDT Bio and PACRI developed self-amplifying RNA vaccines and therapeutics.

• By Type of Modality
  • RNA Therapeutics
  • RNA Vaccines
• By Type of Molecule
  • Replicating RNA (repRNA)
  • Self amplifying RNA (saRNA)
  • Self activating RNA (sacRNA)
  • Self activating mRNA (samRNA)
  • Transfer RNA (tRNA)