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Beyond mRNA Vaccines: The Expanding World of RNA-Based Therapeutics

Beyond mRNA Vaccines: The Expanding World of RNA-Based Therapeutics

Mar 21, 2025PAO-25-30-24

RNA-based therapeutics have rapidly emerged as a transformative force in modern medicine, offering precision-targeted treatments for infectious diseases, cancer, genetic disorders, and more. From the breakthrough success of mRNA vaccines to the expanding landscape of small interfering RNA, antisense oligonucleotides, and next-generation modalities like self-amplifying and circular RNA, the field is evolving at an unprecedented pace. However, significant challenges remain, including manufacturing scalability, delivery hurdles, regulatory uncertainties, and cost barriers that could limit widespread adoption. Advances in synthetic biology, AI-driven RNA design, and innovative delivery technologies are poised to address these challenges and expand RNA's therapeutic potential. As investment and regulatory frameworks adapt to the accelerating innovation, RNA-based medicines are positioned to play a central role in the future of precision healthcare, reshaping treatment paradigms across multiple disease areas.

RNA has emerged as one of the most versatile and rapidly evolving therapeutic modalities in modern medicine. Unlike DNA, which serves as the long-term repository of genetic information, RNA acts as a dynamic intermediary, translating genetic instructions into proteins and regulating gene expression. Over the past several decades, researchers have expanded the scope of RNA’s role in therapeutics, leveraging its ability to directly influence cellular processes. The field has gained significant momentum, particularly following the success of messenger RNA (mRNA) vaccines, which demonstrated the potential of RNA-based interventions for preventing infectious diseases and beyond.

The rise of RNA-based therapeutics reflects a convergence of advancements in molecular biology, synthetic chemistry, and nanotechnology. Initial research in RNA-based drugs focused on antisense oligonucleotides (ASOs) and small interfering RNA (siRNA) for gene silencing, leading to the first regulatory approvals in the early 2000s. More recently, self-amplifying RNA, circular RNA, and RNA-editing technologies have expanded the landscape, offering novel mechanisms for targeted intervention at the molecular level. These innovations have broadened the application of RNA beyond infectious disease vaccines to include oncology, rare genetic disorders, autoimmune diseases, and regenerative medicine.

Despite its promise, RNA therapeutics face several challenges that must be addressed to realize their full potential. Manufacturing at scale remains a significant hurdle, with production bottlenecks, high costs, and supply chain constraints limiting widespread accessibility. Delivery is another persistent obstacle, as RNA molecules are inherently unstable and require sophisticated encapsulation strategies, such as lipid nanoparticles or polymeric carriers, to ensure efficient cellular uptake and stability in vivo. Additionally, regulatory frameworks for RNA-based drugs remain in flux, as agencies such as the U.S. Food and Drug Adminstration (FDA) and the European Medicines Agency (EMA) refine their classification and approval pathways for these novel modalities.

Given the breadth of RNA’s potential applications and the hurdles that remain, it is essential to explore the full range of RNA-based therapeutics, the challenges they face from a practical, manufacturing, clinical, and regulatory perspective, and how future advancements might shape the field. By examining these factors, a clearer picture emerges of how RNA-based interventions can be optimized to become more effective, accessible, and integrated into mainstream medicine.

The Historical Context of RNA Therapeutics

The therapeutic application of RNA is rooted in decades of research on its fundamental biological roles. RNA was first recognized as a key intermediary between DNA and protein synthesis in the mid-20th century, with the discovery of mRNA in the early 1960s providing critical insights into gene expression.1 This period also saw the identification of ribosomal RNA and transfer RNA, which solidified RNA’s essential function in translation. While initially regarded primarily as a passive conduit for genetic information, subsequent discoveries revealed its broader regulatory roles, leading to the foundation of RNA-based therapeutics.

The first efforts to leverage RNA for therapeutic purposes began in the 1970s and 1980s, with early experiments on ASOs. These synthetic strands of nucleotides were designed to bind complementary RNA sequences, blocking translation or altering splicing patterns.2 By the late 1980s, the concept gained traction as researchers demonstrated the potential for ASOs to target disease-associated genes, laying the groundwork for their clinical application.

A major breakthrough in RNA biology occurred in the 1990s with the discovery of RNA interference (RNAi), a naturally occurring mechanism by which small RNA molecules regulate gene expression.3 This discovery, recognized with a Nobel Prize in 2006, led to the development of small interfering RNA (siRNA) as a powerful tool for silencing genes implicated in disease. During this period, microRNAs (miRNAs) were also identified as key posttranscriptional regulators, opening new avenues for therapeutic intervention.

The 2000s marked the first clinical translation of RNA-based drugs. Fomivirsen, an antisense drug targeting cytomegalovirus retinitis, became the first FDA-approved RNA therapeutic in 1998. This was followed by advances in siRNA drugs, with patisiran gaining FDA approval in 2018 for the treatment of hereditary transthyretin-mediated amyloidosis. These milestones validated the potential of RNA-based approaches, despite lingering challenges related to stability, delivery, and off-target effects.

The 2010s and beyond ushered in a new era of RNA therapeutics, catalyzed by the rapid development of mRNA vaccines. Research efforts that had been ongoing for decades — particularly in improving RNA stability and developing efficient delivery systems — enabled the unprecedented success of mRNA-based COVID-19 vaccines in 2020.4 This period also saw the emergence of self-amplifying RNA (saRNA), circular RNA (circRNA), and other novel RNA modalities designed to enhance therapeutic durability and efficacy.

Key regulatory milestones have further legitimized RNA therapeutics as a mainstream pharmaceutical approach. The approval of multiple siRNA and ASO therapies demonstrated the feasibility of RNA-based interventions beyond vaccines, while regulatory bodies have adapted guidelines to accommodate these rapidly evolving modalities. Despite remaining hurdles in cost, scalability, and long-term safety assessment, RNA-based therapies now occupy a central role in the future of precision medicine.

The Diverse Landscape of RNA Therapeutics

RNA-based therapeutics have evolved into a diverse field with multiple modalities designed to address a wide range of diseases. Unlike small molecule drugs or protein-based biologics, RNA-based approaches enable direct modulation of gene expression at different stages, offering precision and adaptability. Advances in synthetic chemistry, delivery technologies, and regulatory approvals have paved the way for the clinical adoption of various RNA therapies. Each class of RNA therapeutics has distinct mechanisms, applications, and challenges, reflecting the versatility and complexity of this growing field.

mRNA: Protein Expression for Vaccines and Therapies

mRNA serves as a transient template for protein synthesis, directing cells to produce specific proteins of therapeutic or immunogenic value. The most well-known application of mRNA technology is in vaccines, where synthetic mRNA encoding viral antigens is delivered to cells, prompting an immune response. The success of mRNA-based COVID-19 vaccines, developed by BioNTech-Pfizer and Moderna, validated this platform and accelerated efforts to develop mRNA vaccines for other infectious diseases, such as influenza and respiratory syncytial virus.4

Beyond vaccines, mRNA is being explored for protein replacement therapies, particularly in rare genetic diseases where a deficient or dysfunctional protein needs to be restored. Moderna and CureVac are among the companies advancing mRNA-based treatments for conditions such as cystic fibrosis and enzyme deficiencies.5 One of the major advantages of mRNA is its ability to be rapidly designed and synthesized, making it a flexible platform for precision medicine. However, challenges such as stability, immune activation, and delivery efficiency remain focal points of ongoing research.6

siRNA: Targeted Gene Silencing

siRNA-based therapies leverage the natural RNAi pathway to silence disease-causing genes by degrading their corresponding mRNA transcripts before translation. This mechanism makes siRNA a powerful tool for treating diseases caused by excessive or aberrant gene expression. Patisiran, developed by Alnylam Pharmaceuticals, became the first FDA-approved siRNA therapy for hereditary transthyretin-mediated amyloidosis in 2018, followed by Givosiran for acute hepatic porphyria.7

The potential applications of siRNA extend beyond rare diseases, with ongoing research in metabolic disorders, viral infections, and oncology. One of the main challenges in siRNA therapeutics is delivery, as these molecules are prone to degradation and require sophisticated carriers, such as lipid nanoparticles or conjugates for targeted tissue uptake.8 The continued refinement of delivery platforms is expected to expand the reach of siRNA-based drugs in clinical practice.

ASOs: Modulating Gene Expression

ASOs are short, synthetic nucleotides designed to bind to specific mRNA sequences, altering gene expression through mechanisms such as translational inhibition or splicing modification. This approach has been particularly successful in treating genetic and neurodegenerative diseases. Spinraza, the first FDA-approved ASO therapy, revolutionized the treatment of spinal muscular atrophy by modulating the splicing of the SMN2 gene to restore functional protein levels.2

Other ASO-based drugs, such as Tegsedi for hereditary ATTR amyloidosis and Eteplirsen for Duchenne muscular dystrophy, have demonstrated the feasibility of this platform. Ongoing research aims to refine ASO chemistry to improve stability, reduce off-target effects, and expand their applicability to conditions such as Alzheimer’s and Huntington’s diseases.6

microRNAs (miRNAs): Posttranscriptional Regulators in Disease

miRNAs are endogenous non-coding RNAs that play a crucial role in posttranscriptional gene regulation, influencing a wide array of physiological and pathological processes. Given their ability to fine-tune gene networks, miRNAs have been implicated in cancer, cardiovascular diseases, and metabolic disorders. Therapeutic strategies targeting miRNAs include miRNA mimics to restore downregulated miRNAs and miRNA inhibitors to suppress overactive miRNAs.9

Despite their potential, the clinical translation of miRNA-based therapies has been hindered by challenges related to delivery and specificity. Naked miRNA molecules are rapidly degraded in circulation, necessitating the development of advanced delivery systems to ensure stability and targeted uptake. Research into nanoparticle-based and conjugated miRNA carriers is ongoing to address these limitations.10

circRNA: A Novel Therapeutic Modality

CircRNAs are covalently closed RNA molecules that exhibit enhanced stability compared with linear RNAs, making them attractive for therapeutic applications. Unlike traditional RNA therapies, circRNAs can function as miRNA sponges, regulators of protein translation, or even templates for protein synthesis.11 Their unique properties offer potential in gene therapy, regenerative medicine, and even vaccine development.

While research on circRNAs is still in its early stages, preclinical studies suggest that engineered circRNAs may provide advantages in durability and reduced immunogenicity. One of the key challenges is the scalable production of functional circRNAs, as their synthesis and purification require specialized methodologies distinct from linear RNA production.5

saRNA: Enhancing Potency and Durability

saRNA represents an evolution of the mRNA platform, incorporating replication machinery from alphaviruses to enable prolonged intracellular expression at lower doses. This amplification reduces the amount of RNA required for efficacy, potentially enhancing the potency and durability of the response.12

saRNA is being actively investigated for next-generation vaccines, including those for influenza and personalized cancer immunotherapy. The key advantage of this approach is its ability to induce robust immune responses with smaller doses, which could improve manufacturing efficiency and cost-effectiveness. However, ensuring controlled amplification and minimizing unintended immune activation remain important challenges.4

RNA Aptamers: Targeting Proteins with High Specificity

RNA aptamers are synthetic RNA molecules engineered to bind specific targets, such as proteins or small molecules, with high affinity. Unlike antibodies, which require complex production processes, aptamers can be chemically synthesized, offering advantages in stability, scalability, and reduced immunogenicity.13

Aptamers have found applications in targeted drug delivery, biosensors, and diagnostic assays. One of the first aptamer-based drugs, Pegaptanib, was approved for age-related macular degeneration but was later supplanted by more effective treatments. Continued research into aptamer engineering and conjugation strategies is expected to expand their therapeutic potential in oncology and neurodegenerative diseases.8

RNA-Editing Technologies: A Precise Alternative to Gene Therapy

RNA-editing technologies, particularly those utilizing adenosine deaminases acting on RNA (ADAR), offer a reversible approach to correcting genetic mutations without permanently altering DNA. Unlike CRISPR-based genome editing, RNA editing provides transient modifications, reducing the risk of unintended genetic changes.5

This emerging technology is being explored for treating genetic diseases, neurological disorders, and even cancer. ADAR-mediated editing has demonstrated potential for correcting disease-associated mutations in preclinical models, though challenges such as delivery, efficiency, and specificity must be addressed before clinical translation.9

Challenges in RNA Therapeutic Development

While RNA-based therapeutics have demonstrated tremendous potential, their widespread adoption is constrained by multiple challenges across manufacturing, delivery, clinical translation, regulatory frameworks, cost, and public perception. These hurdles must be addressed to ensure that RNA therapies can achieve their full impact in medicine.

Manufacturing and Scalability

The large-scale production of RNA therapeutics presents significant technical and economic challenges. Unlike small molecules, RNA molecules require enzymatic synthesis and specialized purification steps, making the process inherently complex and expensive.14 One of the primary cost drivers is the reliance on nucleoside triphosphates (NTPs), enzymes such as T7 polymerase for in vitro transcription (IVT), and lipid nanoparticles for formulation, all of which require stringent quality controls.15

Additionally, capacity constraints limit the global manufacturing of RNA-based drugs. Many contract development and manufacturing organizations (CDMOs) have struggled to scale production to meet demand, particularly during the COVID-19 vaccine rollout, highlighting the need for expanded RNA-specific manufacturing hubs.16 Compared with traditional biologics, such as monoclonal antibodies, which rely on well-established fermentation-based production, RNA therapeutics require newer, less mature infrastructure, further complicating scalability. Addressing these issues requires investments in automation, continuous manufacturing, and alternative production platforms to reduce costs and increase output.

Delivery Challenges

Efficient delivery of RNA molecules remains one of the most significant barriers to therapeutic success. Naked RNA is highly unstable, susceptible to rapid degradation by nucleases in biological fluids, and generally incapable of crossing cellular membranes without assistance.13 Lipid nanoparticles (LNPs) have been the most widely used delivery vehicle, as seen in mRNA vaccines, but their application in systemic therapies has limitations, including toxicity concerns and limited tissue targeting.8

To expand RNA therapeutics beyond vaccines, alternative delivery platforms are being explored, including polymeric nanoparticles, lipid conjugates, and exosome-based carriers. A primary goal is organ-specific targeting to enhance drug efficacy while minimizing off-target effects. For example, liver-targeted siRNA therapies have been successfully developed, but expanding RNA-based drugs to other organs, such as the central nervous system or lungs, remains an ongoing challenge.5

Clinical and Translational Hurdles

Despite advancements in RNA technology, variability in patient response remains a concern. Immune activation triggered by exogenous RNA can lead to unintended inflammatory responses, necessitating careful sequence optimization and chemical modifications such as N1-methylpseudouridine substitutions.4 Even when immune reactions are controlled, some RNA therapeutics require repeated dosing, raising questions about durability and long-term safety.9

Another critical challenge is dosing optimization. Unlike traditional drugs with predictable pharmacokinetics, RNA therapies exhibit variability in uptake, translation efficiency, and degradation, requiring extensive clinical optimization. Off-target effects, particularly in gene-silencing approaches, such as siRNA and antisense oligonucleotides, further complicate therapeutic development.6 Continued improvements in RNA design, computational modeling, and high-throughput screening will be necessary to refine dosing strategies and enhance therapeutic consistency.

Regulatory Considerations

Regulatory frameworks for RNA therapeutics are still evolving. While mRNA vaccines received rapid emergency-use authorization during the COVID-19 pandemic, other RNA-based therapies have faced more protracted approval processes due to uncertainty in classification. A key unresolved issue is whether RNA-based treatments should be regulated as biologics, gene therapies, or a separate category altogether.15

Global regulatory agencies differ in their approaches, creating hurdles for companies seeking international approval. The U.S. FDA, for example, applies a biologics-based framework for RNA vaccines but evaluates gene-silencing therapies, such as siRNA, separately. The EMA takes a case-by-case approach, further complicating harmonization efforts.12 As the field matures, regulatory clarity will be essential for streamlining clinical development and ensuring that safety and efficacy assessments keep pace with technological advancements.

Cost and Market Access

The high cost of RNA drug development threatens to limit patient access, particularly in low- and middle-income countries (LMICs). Manufacturing expenses, combined with stringent cold-chain requirements for storage and distribution, contribute to the high price of RNA-based therapies.7 For instance, the cost of mRNA vaccines was significantly higher than traditional protein-based vaccines, raising concerns about equitable access during global health crises.16

Reimbursement policies and healthcare system constraints further complicate market access. Unlike conventional pharmaceuticals with well-defined pricing models, RNA-based therapies — particularly those for rare diseases — often struggle with reimbursement approvals due to their novelty and high upfront costs.17 Governments and private payers will need to develop new pricing strategies and funding mechanisms to support the sustainable integration of RNA therapeutics into healthcare systems worldwide.

Public Perception and Ethical Concerns

Public acceptance of RNA-based therapies has been a double-edged sword. While the COVID-19 pandemic helped familiarize the public with mRNA vaccines, misinformation and vaccine hesitancy remain persistent challenges.14 Skepticism about genetic technologies, often fueled by misinformation about RNA’s ability to alter DNA, continues to create resistance to broader adoption.4

Ethical considerations also come into play, particularly with gene-modifying RNA applications. The ability to precisely edit or silence genes raises concerns about unintended consequences and equitable access to advanced therapies. Additionally, the prospect of using RNA technologies for human enhancement rather than strictly for medical necessity has sparked bioethics debates.5 Policymakers, ethicists, and the scientific community must work together to establish guidelines that balance innovation with ethical responsibility.

Future Outlook for RNA Vaccines and Therapeutics

The rapid advancement of RNA-based vaccines and therapeutics signals a fundamental shift in drug development, positioning RNA at the forefront of precision medicine. As scientific understanding deepens, next-generation RNA technologies are expected to enhance stability, expand applications beyond infectious diseases, and revolutionize manufacturing and delivery strategies. Regulatory frameworks must evolve to accommodate these innovations, while industry investment trends will shape the future market landscape. The continued convergence of RNA therapeutics with gene editing, synthetic biology, and artificial intelligence (AI)-driven drug discovery will likely unlock even greater potential in the years ahead.

Advances in Next-Generation RNA Technologies

Innovations in synthetic biology are refining the chemical properties of RNA molecules to improve their stability, efficiency, and therapeutic potential. One key area of research involves optimizing the chemical modifications of RNA to enhance resistance to enzymatic degradation while maintaining high translational efficiency. The introduction of nucleoside modifications, such as pseudouridine, has already been instrumental in improving the stability of mRNA vaccines, and further refinements could broaden the scope of RNA-based therapies.10

AI is also transforming RNA drug discovery. AI-driven platforms are now being used to design optimized RNA sequences with minimal immunogenicity and improved cellular uptake, accelerating the development pipeline for RNA-based drugs.9 Additionally, advances in computational modeling are enabling the precise prediction of secondary RNA structures, which is critical for engineering highly stable and effective RNA therapeutics.11

Beyond standalone RNA-based interventions, the field is converging with gene editing, synthetic biology, and protein engineering. RNA-editing technologies such as ADAR-mediated editing offer a reversible approach to modifying gene expression without altering DNA, presenting a promising alternative to CRISPR-based gene therapies.8 Meanwhile, RNA circuits — engineered RNA molecules capable of conditional activation — are being explored for highly targeted gene regulation in cancer and neurodegenerative diseases.5

Expansion Beyond Infectious Diseases

The success of mRNA vaccines during the COVID-19 pandemic has catalyzed research into their broader therapeutic applications. One of the most promising areas is personalized cancer vaccines, in which mRNA is used to encode patient-specific tumor antigens, training the immune system to recognize and attack cancer cells. Several clinical trials for mRNA-based cancer immunotherapies are underway, with early data suggesting durable responses in certain tumor types.4

RNA-based therapeutics are also being explored for autoimmune and inflammatory diseases, with efforts focused on using mRNA and siRNA to regulate aberrant immune signaling pathways. Unlike conventional immunosuppressants, RNA therapies offer precision control over gene expression, reducing the risk of broad immune suppression and associated side effects.6

Another emerging frontier is regenerative medicine and aging-related conditions, where RNA therapies may be used to stimulate tissue repair and counteract cellular aging processes. Self-amplifying RNA and circular RNA platforms are being investigated for their ability to enhance protein production over extended periods, a key advantage for regenerative applications.2

Innovations in Manufacturing and Delivery

Significant efforts are being made to improve RNA drug manufacturing to meet the growing demand while reducing costs and logistical barriers. One of the most pressing challenges is the need for thermostable formulations, particularly for vaccines intended for global distribution. Research into RNA lyophilization and alternative storage conditions aims to eliminate the stringent cold-chain requirements that have historically constrained mRNA vaccine distribution.15

Another transformative development is the push for oral and inhalable RNA drugs, which would circumvent the need for injections and broaden accessibility. Advances in nanoparticle technology are enabling the encapsulation of RNA molecules in protective carriers that can withstand the digestive or respiratory environments, potentially leading to RNA-based pills or inhaled formulations for diseases such as cystic fibrosis and pulmonary fibrosis.8

To accommodate the increasing demand for RNA therapeutics, the industry is also scaling up production capabilities, with new RNA-specific manufacturing hubs being established worldwide. Biopharma companies are investing in continuous RNA manufacturing platforms, which promise higher efficiency and lower costs compared with traditional batch production.16

Regulatory Evolution

As RNA therapies continue to diversify, regulatory agencies must harmonize guidelines to streamline approvals and provide clear frameworks for clinical development. Currently, the classification of RNA therapeutics remains inconsistent, with some products regulated as biologics, others as gene therapies, and emerging modalities falling into regulatory gray areas.12 Standardized guidelines will be essential to facilitate the approval of novel RNA-based drugs.

Fast-track regulatory pathways may be expanded to include critical RNA-based therapies, particularly in oncology and rare diseases where unmet medical needs justify accelerated approval.17 Additionally, the growing use of real-world evidence and adaptive clinical trial designs will likely shape the future of RNA drug approval, allowing for more flexible study designs that can evolve based on emerging data.9

Industry and Investment Trends

The RNA therapeutics sector continues to attract substantial investment, with increasing partnerships between biotech startups and major pharmaceutical companies. Biopharma alliances are playing a key role in advancing RNA research, as seen in collaborations between Moderna and Merck for mRNA-based cancer vaccines and between Alnylam and Novartis for RNA interference therapies.5

Despite some deacceleration following the COVID-19 pandemic, market projections indicate continued growth in RNA-based drug classes, particularly as new indications expand beyond vaccines into personalized medicine, rare diseases, and chronic conditions. Analysts estimate that the global RNA therapeutics market will surpass $15 billion by the end of the decade, driven by technological advancements and expanding commercial applications.4

Government funding and venture capital investments are also fueling RNA innovation, with national agencies and private investors allocating substantial resources to RNA drug development. Countries with strong biomanufacturing capabilities, such as the United States, Germany, and China, are leading the charge in RNA-focused initiatives, while emerging biotech hubs are beginning to establish RNA research programs of their own.16

Looking Ahead

RNA-based technologies have emerged as one of the most transformative advancements in modern medicine, redefining the possibilities of vaccine development, gene regulation, and precision therapeutics. The success of mRNA vaccines during the COVID-19 pandemic showcased the ability of RNA-based platforms to provide rapid, scalable solutions for global health challenges. Beyond vaccines, the field has expanded into siRNA and ASOs for gene silencing, saRNA for enhanced durability, and circRNA for novel regenerative applications. As RNA therapeutics continue to evolve, they hold immense potential for addressing complex diseases, including cancer, autoimmune disorders, and rare genetic conditions.4,10

Despite its promise, the field must overcome several significant challenges to fully realize its potential. Manufacturing scalability remains a key obstacle, with high production costs, supply chain constraints, and limited global capacity slowing the widespread adoption of RNA-based treatments.16 Innovations in synthetic biology and continuous manufacturing must be leveraged to improve efficiency and reduce costs. Delivery limitations also present hurdles, as existing lipid nanoparticle systems, while effective for vaccines, require refinement for targeted RNA therapies addressing non-liver tissues.8

Regulatory frameworks will need to adapt to accommodate the rapid expansion of RNA therapeutics. The current lack of standardized global guidelines has created a complex approval landscape, with regulatory agencies taking varied approaches to classifying and evaluating RNA-based drugs.15 Harmonization efforts and adaptive clinical trial designs will be crucial in ensuring that innovation is balanced with safety and efficacy. Additionally, cost and market access concerns must be addressed to prevent RNA therapies from being prohibitively expensive, particularly in LMICs.

Looking ahead, the future of RNA-based medicine is exceptionally bright. Advances in AI-driven RNA design, improved delivery platforms, and next-generation modifications will continue to refine the efficacy and stability of these therapies. The convergence of RNA therapeutics with gene editing, synthetic biology, and protein engineering will further expand the range of treatable conditions. As biopharma investment in RNA research accelerates, and clinical successes accumulate, RNA-based drugs are poised to play a central role in the next era of precision medicine.5

Ultimately, the continued development of RNA technologies represents not just an evolution in therapeutics but a paradigm shift in how diseases are treated. By addressing current barriers in production, regulation, and accessibility, RNA-based treatments have the potential to reshape global healthcare, providing innovative solutions for some of the most challenging medical conditions of our time.

Our parent company, That’s Nice, is committed to supporting the companies and innovators driving the next wave of pharma and biotech innovation. To celebrate That’s Nice’s 30th anniversary, Pharma’s Almanac is diving into 30 groundbreaking advancements, trends, and breakthroughs that have shaped the life sciences, highlighting the industry-defining milestones our agency has had the pleasure of growing alongside. Here’s to 30 years of innovation and the future ahead!

References

1. Jacob F. and J. Monod. "Genetic Regulatory Mechanisms in the Synthesis of Proteins." J. Mol. Biol. 3: 318-356 (1961). h

2. Mollica, Luca, et al.An overview of structural approaches to study therapeutic RNAs.” Front. Mol. Biosci. 27 Oct. 2022.

3. Fire, Andrew, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans.Nature. 391: 806–811 (1998).

4. Lu, Ruei-Min, et al.Current landscape of mRNA technologies and delivery systems for new modality therapeutics.” Journal of Biomedical Science. 31: 89 (2024).

5. Goleij, Pouya, et al.Chapter Three – Types of RNA Therapeutics.” Progress in Molecular Biology and Translational Science. 203: 41–63 (2024).

6. Kim, Young-Kook.RNA therapy: rich history, various applications and unlimited future prospects.” Experimental & Molecular Medicine. 54: 455–465 (2022).

7. Curreri, Alexander, et al.RNA therapeutics in the clinic.” Bioengineering & Translational Medicine. 25 Jun. 2022.

8. Chatterjee, Srijan, et al.An insight of different classes of RNA-based therapeutic, nanodelivery and clinical status: Current landscape.” Current Research in Biotechnology. 6: 100150 (2023).

9. Tani, Hidenori.Recent Advances and Prospects in RNA Drug Development.” Int. J. Mol. Sci. 25: 12284 (2024).

10. Zhu, Yiran, et al. “RNA-based therapeutics: an overview and prospectus.” Cell Death & Disease. 13: 633 (2022).

11. Sparmann, Anke and Jörg Vogel.RNA‐based medicine: from molecular mechanisms to therapy.” EMBO J. 42: e114760 (2023).

12. Overview and Outlook for RNA-Based Therapies. Avalere. May 2024.

13. Damase, Tulsi Ram, et al.The Limitless Future of RNA Therapeutics.” Front. Bioeng. Biotechnol. 17 Mar. 2021.

14. “RNA Therapeutics – the Promise, the Challenges, and the Future.” Vickers Pulse. 1 Jul. 2022.

15. Guerriaud, Mathieu and Evelyne Kohli.RNA-based drugs and regulation: Toward a necessary evolution of the definitions issued by the European union legislation.” Front. Med (Lausanne). 9: 1012497 (2022).

16. Elharrar, Vanessa and Louis Cicchini. Getting Ahead in mRNA Vaccine Development with an Experienced CRO Partner.” PPD / Thermo Fisher Scientific Insights Hub. 21 Jul. 2022.