Discover 5 Top Pharma Startups developing RNA Therapeutics

Curious about new technological advancements in the pharma industry? Explore our analysis of 135 global startups & scaleups and learn how their RNA therapeutics impact your business!

Staying ahead of the technology curve means strengthening your competitive advantage. That is why we give you data-driven innovation insights into the pharma industry. This time, you get to discover 5 hand-picked startups developing RNA Therapeutics.

Global Startup Heat Map highlights 5 Top RNA Therapeutics Solutions out of 135

The insights of this data-driven analysis are derived from the Big Data & Artificial Intelligence-powered StartUs Insights Discovery Platform, covering 2.093.000+ startups & scaleups globally. The platform gives you an exhaustive overview of emerging technologies & relevant startups within a specific field in just a few clicks.

The Global Startup Heat Map below reveals the distribution of the 135 exemplary startups & scaleups we analyzed for this research. Further, it highlights 5 pharma startups that we hand-picked based on criteria such as founding year, location, funding raised, and more. You get to explore the solutions of these 5 startups & scaleups in this report. For insights on the other 130 RNA Therapeutics solutions, get in touch.

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ARTHEx Biotech develops Antisense RNA Therapeutics

The aberrant or depleted expression of certain genes is implicated in the development of many diseases. However, microRNAs (miRNA) act as modulators of gene expression by binding to messenger RNA (mRNA), thereby inhibiting their translation. Pharma startups are developing microRNAs to use as novel targets for antisense therapeutics. These are single-stranded RNA molecules that bind complementary sequences on the miRNA, thus inhibiting it.

Spanish startup ARTHEx Biotech develops antisense RNA therapeutics for genetic diseases. The startup’s lead drug candidate is an anti-microRNA that treats myotonic dystrophy type 1 (DM1). In muscle biopsies of patients, the startup’s anti-miRs are demonstrated to inhibit microRNAs that repress MBNL1/2 genes in DM1.

Vico Therapeutics offers RNA Modulating Therapeutics

Different kinds of RNAs regulate different cellular processes, as well as play a role in the development of different diseases. Drugs that bind to specific RNA targets modulate their structures and, subsequently, upregulate or downregulate the expression of particular genes. This is why startups create a range of RNA-targeting drugs, ranging from small molecule drugs to antisense oligonucleotides (ASOs) and gene therapies.

Dutch startup Vico Therapeutics builds an oligonucleotide platform technology. The platform develops antisense oligonucleotides into RNA modulating therapeutics targeting central nervous system (CNS) disorders. Its RNA modulating therapeutics act via various mechanisms. Some of these include inhibiting mRNA translation, targeting the pre-mRNA splicing machinery, editing point mutation, and reducing mutant mRNA levels. Further, the startup’s preclinical stage pipeline includes drugs for spinocerebellar ataxias and Huntington’s disease.

Pantherna Therapeutics provides mRNA Therapeutics

mRNA therapeutics leverage the cell’s ability to translate any mRNA into proteins. Thus, injecting therapeutic mRNA into cells essentially turns them into medicine-making machines. However, this process generates an immune response that shuts down the cell machinery required to translate it and is hard to deliver safely into cells. To advance mRNA therapeutics, pharma startups are working on solutions that address these challenges.

Pantherna Therapeutics is a German startup working on mRNA therapeutics. The startup’s lead drug, PAN004, is packaged in an optimized cationic lipid nanoparticle (cLNP) formulation. The nanoparticle specifically targets the pulmonary microvascular endothelial cells. Once inside the cells, the synthetic mRNA activates the Tie2pathway, restoring vascular barrier function in these cells.

RNAimmune develops mRNA Vaccines

Thanks to Pfizer/BioNTech’s and Moderna’s COVID-19 vaccines, mRNA vaccines have come into the limelight. mRNA vaccine platforms speed up vaccine development as once the platform is developed, it is rapidly amenable to deliver any synthetic mRNA. Owing to this flexibility, pharma startups are leveraging mRNA technology to develop vaccines against viruses, other infectious diseases, malaria, and cancers.

RNAimmune is a US-based startup developing its proprietary platform that combines Artificial Intelligence and synthetic biology to design mRNAs that elicit an immune response. Because the mRNAs are self-amplifying in nature, they provide stronger and longer immune protection. RNAimmune focuses on prophylactic vaccines against infectious diseases, as well as personalized cancer vaccines.

TransCode Therapeutics works on RNA Oncology Therapeutics

RNA therapeutics open up a range of mechanisms to alter cellular behavior. For example, they enable targeting previously undruggable proteins, as well as rational design of therapeutic proteins. Consequently, pharma startups and scaleups alike are exploring RNA therapeutics for unmet clinical needs, including different forms of cancers. Moreover, they also hold the possibility for personalized treatment of cancers by targeting individual-specific drug resistance mutations.

US-based startup TransCode Therapeutics creates RNAi therapeutics for oncology. The startup’s TTX platform utilizes an iron oxide nanoparticle as a delivery vehicle for RNA therapeutics. The iron oxide allows guided delivery by imaging techniques, making the delivery safe and efficient. TTX-MC138, the startup’s lead drug candidate, is undergoing preclinical trials targeting metastatic cancer and glioblastoma.

Discover more Pharma Startups

Pharma startups such as the examples highlighted in this report focus on additive manufacturing, precision medicine, digital therapeutics as well as artificial intelligence. While all of these technologies play a major role in advancing the pharma industry, they only represent the tip of the iceberg. To explore more pharmaceutical technologies, simply get in touch to let us look into your areas of interest. For a more general overview, you can download our free Pharma Innovation Report to save your time and improve strategic decision-making.

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