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14 Cutting-Edge Startups Focusing On RNA Research

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: the transcriptome level, presented by ribonucleic acids (RNAs).

RNA molecules are not just intermediates between DNA and proteins, in fact, they also carry out 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, efforts by companies in the transcriptomics space are typically directed in one of the following ways:

  • RNAs that modulate DNA,
  • RNAs that target proteins,
  • RNAs that encode proteins,
  • modalities, like small molecules, that modulate RNA or related transciption machinery (e.g. splicing)
  • delivery of RNA-based therapies
  • other strategies, like non-coding RNAs

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, small molecules targeting RNAs or transcriptome-related proteins, and so on.

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 I am listing several biotech startups to keep an eye on. 

Some of them are platform companies powered by artificial intelligence (AI):

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.

HAYA Therapeutics

HAYA Therapeutics is developing precision medicines targeting the dark genome, focusing on long non-coding RNAs (lncRNAs) to reprogram disease-driving cell states.

They utilize proprietary tools to identify disease-associated tissues and cells, then apply RNA-targeting modalities like modified antisense oligonucleotides (ASOs) to inhibit specific lncRNAs.

This approach aims to modulate the epigenome and reverse disease processes. Their technology addresses rare and chronic diseases by leveraging the regulatory potential of dark genome-derived RNAs. This innovative strategy represents a next-generation therapeutic paradigm in biopharmaceuticals.

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