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7 Promising RNA Biotech Companies To Watch in 2024

For many years the focus of therapeutic developments and scientific research was directed at the genome and proteins. Yet, there is a completely different level of cell regulation – transcriptome level, presented by Ribonucleic acids (RNAs). RNA molecules are not just intermediates between DNA and proteins, in fact, they also carry enzymatic functions, control gene expression, and perform other functions in cells.

Major breakthroughs in transcriptomics have been made since the beginning of the century: the discovery of RNA interference blew up the industry and the Nobel Prize in Physiology or Medicine 2006 was awarded jointly to Andrew Z. Fire and Craig C. Mello “for their discovery of RNA interference – gene silencing by double-stranded RNA.” 

RNA interference is a gene silencing mechanism promoted by small siRNA molecules that are also exploited in patisiran and inotersen – first-in-class RNA therapeutics. Both drugs target transthyretin, a protein involved in the pathogenesis of transthyretin-mediated amyloidosis, and reduce the expression of its mutant version. 

Today, RNA therapies can be categorized into 3 broad groups: RNAs that modulate DNA, RNAs that target proteins, RNAs that encode proteins, and hybrids of three listed approaches. Among existing modalities – ASOs, single-stranded antisense oligonucleotides that block mRNA translation, siRNAs and microRNAs that degrade protein-encoding mRNAs, RNA aptamers that tightly bind specific spots on proteins and regulate their functioning. In addition to that, last year showed a real boom in mRNA vaccines, with coronavirus vaccines and cancer vaccines being the most prominent representatives.

While there are numerous companies developing RNA-based therapeutics and vaccines, including major corporations like Pfizer, BioNtech, Moderna, etc, below we decided to list several biotech startups to keep an eye on. 

Atomic AI, founded in May 2021, is headquartered in the San Francisco Bay Area and is focused on leveraging a fusion of machine learning and structural biology to advance RNA drug discovery. The company has developed a proprietary platform that utilizes deep learning foundation models to explore and design RNA-targeted small molecules, RNA-based medicines, and RNA tools.

Atomic AI raised a total of $42 million in funding over two investment rounds, with the latest funding being secured on January 2023, from a Series A round. The company has attracted investments from 14 investors, with Nat Friedman and Neal Khosla being the most recent backers.

In a notable personnel update, Atomic AI announced the appointment of Dr. Manjunath “Manju” Ramarao, a veteran from Bristol Myers Squibb with over 20 years of experience in the pharmaceutical industry, as its Chief Scientific Officer, showcasing the company’s commitment to advancing its RNA drug discovery platform.

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