5 Top Pharma Startups Developing RNA-Targeting Drugs

We analyzed 65 ribonucleic acid (RNA)-targeting drug startups in the pharma sector. Arrakis Therapeutics, Reborna Biosciences, Secarna, EPICS & Locanabio develop 5 top solutions you should watch out for. Learn more in our Global Startup Heat Map!

Our Innovation Analysts recently looked into emerging technologies and up-and-coming startups working on innovative solutions for the pharma sector. As there is a large number of startups working on a wide variety of solutions, we want to share our insights with you. This time, you will discover 5 promising RNA-targeting drug startups.

Heat Map: 5 Top RNA-Targeting Drug Startups

Using our StartUs Insights Discovery Platform, covering 1.379.000+ startups & scaleups globally, we looked at innovation in the field of pharmaceuticals. For this research, we identified 65 relevant solutions and picked 5 to showcase below. These companies were chosen based on a data-driven startup scouting approach, taking into account factors such as location, founding year, and relevance of technology, among others. Depending on your specific criteria, the top picks might look entirely different.

The Global Startup Heat Map below highlights 5 startups & scaleups developing innovative RNA-targeting drugs. Moreover, the Heat Map reveals regions that observe a high startup activity and illustrates the geographic distribution of all 65 companies we analyzed for this specific topic.


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Which startups develop the other 60 solutions?


Arrakis Therapeutics – Small Molecule Drugs

In recent years, research has revealed the role of RNA in different diseases such as muscular dystrophy and cancers. Inspired by these discoveries, pharma startups are developing drugs that target RNA molecules. Small molecule drugs bind to secondary or tertiary RNA structures and affect translation or localization, or cause RNA to degrade. The size of small molecule drugs makes the entry into cells easier, where they target RNA sequences.

Arrakis Therapeutics is a US-based startup developing RNA-targeting biopharmaceuticals. The startup’s small molecules drugs bind to targets previously thought undruggable, as well as those validated with genetic and pharmacological methods. These drugs inhibit either translation or splicing of RNA or incorporate multiple mechanisms. The startup’s pipeline includes drugs for oncology, dyslipidemia, and rare diseases.

Reborna Biosciences – Structure-Based Drug Design

Structure-based drug design employs the knowledge of the three-dimensional structure of a target to design drugs against it. Since RNA assumes a unique structure based on its sequence, pharma startups use bioinformatics to identify RNAs that form targetable structures. This informs the design of drugs that bind and degrade RNA implicated in the pathogenesis of different diseases.

Japanese startup Reborna Biosciences works on drugs for rare genetic diseases. The startup uses a proprietary screening method that uses the three-dimensional structure of RNA in vivo to discover small molecule drugs. Their pipeline includes drugs against neuromuscular disease, dementia, and lysosomal storage disease.

Secarna – Antisense Oligonucleotides (ASOs)

ASOs are synthetic RNA or deoxyribonucleic acid (DNA) sequences that hybridize target RNA to inhibit gene expression, modulate messenger RNA (mRNA), or inactivate micro RNAs. Since the therapeutic ASO in RNA interference (RNAi) binds the disease-causing mRNA, it is no longer available for expression. To facilitate the entry of ASOs inside the cell, pharma startups use delivery by ligand-based targeting molecules, viral vectors, and delivery by lipids or polymers.

Secarna is a German startup that offers antisense therapies. The startup’s proprietary LNAplus drug discovery platform identifies antisense oligonucleotides for preclinical and clinical development. Oligofyer, Secarna’s bioinformatics platform, enables the rational design of ASOs and complements LNAplus. The startup is also working on therapies for thrombocytopenia, liver toxicity, and nephrotoxicity.

EPICS – RNA Epigenetics

Several studies highlight the role of epigenetic modulation of RNA in cancer progression. Encouraged by these findings, pharma and MedTech startups are developing epigenomics-based solutions as potential therapies. Startups also are developing inhibitors of methyltransferase and histone deacetylases, two enzymes key to epigenetic modifications.

Belgian startup EPICS develops drugs that target unmet medical needs in oncology. The startup’s pipeline includes small molecule drugs that bind to RNA targets. These drugs then target the epigenetic mechanisms underlying cancer development. The startup’s lead candidate is an RNA methyltransferase inhibitor that has also been validated in animal models.

Locanabio – RNA Targeting Gene Therapies

A major challenge of RNA-targeting drugs is the intake into cells where the particular RNA is present. Gene therapies address this issue by delivering the therapeutic DNA inside cells. Since advancements in precision editing technologies such as CRISPR now make it easier to develop gene therapies. This leads pharma startups to explore gene therapies that target specific RNA.

Locanabio is a US-based startup that develops RNA targeting gene therapies. The startup combines gene therapy and RNA modification to precisely target disease-causing RNA. The solution uses an adeno-associated viral (AAV) vector to deliver RNA binding proteins into the cells. Locanabio’s pipeline includes therapeutics against muscular dystrophy, as well as neurodegenerative diseases like Huntington’s, spinocerebellar ataxia, and amyotrophic lateral sclerosis (ALS).

What about the other 60 solutions?

While we believe data is key to creating insights it can be easy to be overwhelmed by it. Our ambition is to create a comprehensive overview and provide actionable innovation intelligence and enable you to achieve your goals faster. The 5 RNA-targeting drug startups showcased above are promising examples out of the 65 we analyzed for this article. To identify the most relevant solutions based on your specific criteria, get in touch.

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