Published: October 2023
These next generation RNA therapeutics and vaccines are currently in late stages of clinical development (Phase I / II and above) and anticipated to receive marketing approval in the coming decade. We analyzed more than 100 next generation RNA therapeutics and RNA vaccines (including self amplifying RNA, self amplifying mRNA, transfer RNA, endless RNA and circular RNA) that enable higher therapeutic efficacy and targeted delivery. Of these, discover the 5 RNA therapeutics and vaccines that have reached late stages of development.
Over the years, RNA therapeutics and RNA vaccines have been studied extensively for their crucial role in gene function and protein expression regulation. Owing to their vast potential in the treatment of a myriad of disease indications, targeting previously deemed undruggable targets, and high therapeutic efficiency, these molecules have transitioned into a compelling treatment alternative for a range of disorders. However, a major challenge in the implementation of these therapies lies in the highly unstable structure of the RNA molecule. The loose prime terminals of RNA molecules allow quick degradation of these molecules by restriction exonucleases. Therefore, to circumvent this challenge and achieve a more stable and efficient line of treatment, pharma companies are actively investing their time and resources in the development of next generation RNA therapeutics.
Next generation RNA therapeutics and vaccines are making their way in to the healthcare industry, being developed as viable treatment options for various life-threatening diseases, which help combat the concerns associated with the instability of conventional RNA-based therapies such as mRNA therapeutics. Further, the novel characteristics conferred by these molecules such as closed loop structure and self-replicating nature have sparked the interest of drug developers engaged in this industry. As a result, more than 30 developers are currently developing next generation RNA therapeutics and vaccines across various stages of development. In this context, a number of next generation molecules are being developed in late stages of clinical development, that are anticipated to receive marketing authorization in the coming 12 years.
Roots Analysis has conducted an exhaustive study on RNA Therapeutics and RNA Vaccines Market featuring the current market landscape and future opportunity associated with the RNA vaccines and RNA therapeutics market, over a span of 12 years.
In order to ensure continued success rate of these modalities, stakeholders are undertaking several partnership and funding initiatives for their progression into advanced stages of development. Further, in the recent years, this market has witnessed several technological advancement efforts, focused on improving the design, development, manufacturing and delivery processes for next generation RNA therapeutics and vaccines. The significant activity of industry stakeholders in this market demonstrates the vast potential of next generation modalities to revolutionize the healthcare industry. The pharmaceutical companies are increasing looking out for smaller biotech companies working on next generation drug modalities, including novel antibody therapies, bacteriophage therapy and more such areas.
The top five RNA therapeutics and RNA vaccines currently in late stages of clinical development (phase I / II and above) along with information on their developers are listed below (in decreasing order of phase of development):
Table 1: List of Top 5 Next Generation RNA Therapeutics and Vaccines
| S. No | Drug Name | Developer | Type of Modality | Type of Molecule | Phase of Development |
| 1 | Gemcovac | Gennova Biotherapeutics | Vaccine | Self-amplifying messenger RNA | Approved |
| 2 | GRANITE (GRT-C901 / GRT-R902) | Gritstone Bio | Vaccine | Self-amplifying messenger RNA | Phase II / III |
| 3 | AVX901 / VRP-HER2 | Alphavax | Vaccine | Self amplifying RNA | Phase II |
| 4 | MTL-CEBPA + Sorafenib | MiNA Therapeutics | Therapeutic | Self activating RNA | Phase II |
| 5 | SLATE KRAS | Gritstone Bio | Vaccine | Self-amplifying messenger RNA | Phase I / II |
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Discover the Details on Top 5 RNA Therapeutics and RNA Vaccines
This article highlights the top 5 RNA therapeutics and vaccines to watch out for in this industry. These drugs were chosen based on their phase of clinical development (Phase I / II and above). It is essential to note that the selection of top drugs can differ substantially according to the defined criteria.
Gemcovac, a samRNA molecule, is the only approved self-amplifying mRNA therapy, till date. Co-developed by HDT Bio and Gennova Biopharmaceuticals, Gemcovac received emergency use authorization in India for treatment of COVID-19. The vaccine is currently in phase II trial in Brazil, which initiated in January 2023 and the study is likely to be completed by September 2023. The safety and efficacy profiles of the vaccine can be validated from its phase II / III clinical trial study results demonstrating an equivalent response rate to AstraZeneca vaccine and much lower rates of adverse events as compared to other COVID-19 vaccines.
Gemcovac-OM is an Omicron specific booster vaccine which was launched in June 2023. The vaccine is thermostable, as such, it does not require cold chain storage.
Developer Details:
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Established in 2018, HDT Bio is a small, US-based clinical stage biopharmaceutical company engaged in the development of innovative immune therapies by leveraging its proprietary technology platform. The company is focused on developing a pipeline of therapeutic candidates using its AMPLIFY vaccine platform, primarily targeting infectious diseases. It is worth highlighting that since 2019, the company has raised a sum of USD 68 million, across five funding instances for the advancement of its therapeutic pipeline.
Recent Developments:
In August 2023, Coalition for Epidemic Preparedness Innovations (CEPI) announced funding of $3.6 million to Gennova Biopharmaceuticals for optimization of the samRNA platform as a preparation against unknow pathogenic threats in the future.1
In January 2023, the company entered into an agreement with Pan African Cancer Research Institute (PACRI) in order to apply the former’s advanced RNA technology and delivery systems for the development of vaccines for the treatment of cancer, across Africa.
In November 2022, the company entered into another technology utilization agreement with The University of Texas Medical Branch (UTMB), in order to apply its proprietary technology for the development of vaccines targeting Crimean-Congo hemorrhagic fever virus (CCHFV) and Nipah virus (NiV). It is worth highlighting that the company has inked a few other delas such as product development agreement in 2022. Further information related to partnerships and funding instances is available in the full report.
GRT-R902 is a self-amplifying RNA vector, being used to develop GRANITE along with GRT-C901, an adenoviral vector.2 GRANITE is being evaluated in phase II / III clinical trials in Vietnam for the treatment of influenza. The phase II / III clinical trial of the drug initiated in February 2022 and the study is likely to be completed by March 2027. Considering that the drug has received fast track designation from the US Food and Drug Administration, the likely approval year for the drug in the US is 2028. Further, the safety and efficacy profiles of the drug can be validated from its phase II / III clinical trial study results demonstrating the lack of high tumor mutation burden in patients who achieve a molecular response.
Developer Details:
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Established in 2015, Gritstone bio is a mid-sized, US-based biotechnology company engaged in the development of next generation vaccines, primarily targeting cancer and infectious diseases. The company has an in-house pipeline of five vaccine programs, evaluating their potential in the treatment for microsatellite-stable colorectal cancer, KRAS-mutated solid tumors, COVID-19 and Human Immunodeficiency Virus (HIV). Further, the company possess a proprietary vaccine platform for the drug design, development and delivery of its next generation RNA vaccines. It is worth highlighting that since 2019, the company has raised a sum of USD 430 million, across seven funding instances in order to fund its ongoing research and clinical development initiatives of its oncology and infectious disease programs.
AVX901 / VRP-HER2 is a self-amplifying RNA vaccine, being evaluated in phase II clinical trials in the US for the treatment of HER2+ breast cancer. The phase II clinical trial of the vaccine initiated in March 2019 and the study is likely to be completed by December 2024. The vaccine is currently being evaluated as a combination therapy with pembrolizumab. The anticipated approval year for the drug in the US is 2029. Further, the safety and efficacy profiles of the drug can be validated from its phase I clinical trial study results demonstrating a well-tolerated safety profile, with no reported cases of adverse events or potential immunogenicity associated with therapy administration.
Developer Details:
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Established in 1997, AlphaVax is a mid-sized, US-based biotechnology company engaged in the development of vaccines, primarily targeting a wide range of infectious diseases and cancer indications. The company has an in-house pipeline of nine next generation RNA vaccine programs, evaluating their potential across preclinical and clinical stages of development. Further, the company possess a proprietary vaccine platform for the drug development, manufacturing and delivery of its next generation RNA vaccines. It is worth highlighting that the company’s current business strategy includes the out-licensing of its aphavaccine technology for specific disease applications for commercialization.
Recent Developments:
The company claims to have partnered with the US government agencies in order to out-license its technology for vaccine treatments against certain viruses. Further, the company has collaborated with two companies - Merck's Intervet subsidiary and Novartis Vaccines and Diagnostics for its vaccine development initiatives. Further information related to partnerships and funding instances is available in the full report.
MTL-CEBPA + Sorafenib is a self-activating RNA therapy, being evaluated in phase II clinical trials in the US and Singapore for the treatment of hepatocellular carcinoma arising from Hepatitis B and Hepatitis C. The drug is aimed at mediating the regulation of myeloid cells that are known to be associated with solid tumors. The phase II clinical trial of the drug initiated in January 2022 and the study is likely to be completed by May 2025. The vaccine is currently being evaluated as a first and second-line treatment for advanced stage hepatocellular carcinoma. Considering that the drug has received orphan drug designation from the US Food and Drug Administration, the anticipated approval year for the drug in the US is 2029, followed by Singapore (2032). Further, the safety and efficacy profiles of the drug can be validated from its phase Ib clinical trial study results in combination with Sorafenib demonstrating increased effectiveness and an overall response rate in 27% of the target patient population.
Developer Details:
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Established in 2008, MiNA Therapeutics is a small, UK-based biotechnology company pioneering the development of small activating RNA therapeutics, primarily targeting hematological, cardiovascular and immune-oncological disease indications. The company has an in-house pipeline of seven next generation RNA therapeutic programs, evaluating their potential across discovery, preclinical and clinical stages of development. Further, the company possess a proprietary saRNA technology platform for research and discovery, design and development of its next generation RNA therapeutics. that since 2019, the company has raised a sum of USD 45 million, across two funding instances in order to advance its internal pipeline of its new class of RNA therapeutics focused on cancer, immune-oncology and genetic disorders.
Recent Developments:
In April 2023, the company entered into research collaboration with BioMarin Pharmaceuticals to advance the saRNA technology in rare genetic diseases.3
In December 2021, the company entered into a clinical trial agreement with The National University Cancer Institute, Singapore (NCIS) for the clinical development of MTL-CEBPA. Further, the company has entered into multiple research and development agreements with Eli Lilly, Servier and AstraZeneca for advancing its saRNA therapeutics pipeline. Further information related to partnerships and funding instances is available in the full report.
SLATE is a self-amplifying RNA vector based vaccine, being evaluated in phase I / II clinical trials in the US for the treatment of KRAS-driven non-small lung cancer and microsatellite stable colorectal cancer. The phase I / II clinical trial of the drug initiated in July 2019 and the study was completed in March 2023. The anticipated approval year for the drug in the US is 2029. Further, the safety and efficacy profiles of the drug can be validated from its phase I / II clinical trial study results suggesting its implications in early-line of treatment.
Developer Details:
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Established in 2015, Gritstone bio is a mid-sized, US-based biotechnology company engaged in the development of next generation vaccines, primarily targeting cancer and infectious diseases. The company has an in-house pipeline of five vaccine programs, evaluating their potential in the treatment for microsatellite-stable colorectal cancer, KRAS-mutated solid tumors, COVID-19 and Human Immunodeficiency Virus (HIV). Further, the company possess a proprietary vaccine platform for the drug design, development and delivery of its next generation RNA vaccines. It is worth highlighting that since 2019, the company has raised a sum of USD 430 million, across seven funding instances in order to fund its ongoing research and clinical development initiatives of its oncology and infectious disease programs.
Recent Developments:
In December 2021, Gritstone bio entered into an agreement with The Coalition for Epidemic Preparedness Innovations (CEPI) in order to support the development of its vaccine targeting Omicron COVID-19 variant. Further, in February 2021, the company entered into a technology utilization agreement with Gilead Sciences to advance the research and discovery of its vaccine program targeting Human Immunodeficiency Virus (HIV). Further information related to partnerships and funding instances is available in the full report.
The above presentation features five promising RNA therapeutics / RNA vaccines selected from a pool of over 100 candidates that we have compiled. If you are interested, you can download the Sample Report on “RNA Therapeutics and RNA Vaccines Market” by Roots Analysis. For personalized assistance in identifying the most relevant solutions based on your specific criteria, please don't hesitate to reach out to us at sales@rootsanalysis.com.
Dhriti is a Senior Business Analyst, working as an integral part of the Roots Analysis team since 2021. Passionate about the intersection of science and industry, she holds a Bachelor’s degree in biotechnology from a renowned university. Possessing keen interest in the pharma domain, Dhriti initiated her professional journey with a strong academic foundation. Her passion to learn more about the emerging trends and technological advancements has enabled her to provide comprehensive insights through syndicate reports and consulting. The unique blend of scientific knowledge and business acumen has enabled her to provide insightful analysis in the ever-evolving life-sciences domain. Till date, she has earnestly contributed to various subjects on trending topics, including women health, oligonucleotide synthesis, RNAi therapeutics and technologies, cell therapy manufacturing and deep learning in drug discovery and diagnostics.
Sources 1: https://indianexpress.com/article/cities/pune/cepi-pune-firm-to-fund-development-of-mrna-vaccine-tech-8883082/
Sources 2: https://www.nature.com/articles/s41467-023-39053-9
Sources 2: https://www.bloomberg.com/press-releases/2023-04-25/mina-therapeutics-enters-research-collaboration-with-biomarin-pharmaceutical-to-advance-rnaa-platform-research-in-rare-genetic
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