RNA therapeutics at a glance
For many years the focus of therapeutic developments and scientific research was directed at the genome and DNA-related proteins. Yet, there is a completely different level of cell regulation – transcriptome level, presented by RNAs. RNA molecules are not just intermediates between DNA and proteins, in fact, they carry enzymatic functions, control gene expression, and build up proteins from amino acids.
Major breakthroughs in transcriptomics have been made since the beginning of the century: the discovery of RNA interference in 2001 blew up the industry and was awarded the Nobel Prize in 2006. 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.
Orna therapeutics is a Cambridge-based biotech startup founded in 2019 whose main focus lies in the novel field of circular RNAs (oRNAs) which is the next step in RNA-based therapies. The company’s innovation relies on the groundbreaking results of Prof. Daniel Anderssons’s group (MIT) that investigated oRNAs and found its superiority over linear analogs in terms of stability, protein expression, and synthetic availability. The potential applications of this technology are far-reaching, including Orna’s initial focus on developing in situ CAR-T delivery system which avoids costly and complicated procedures of the current immunotherapies. The company already managed to close the Series A round in February 2021 raising $80 million from a group of prominent healthcare investors including F2 Ventures, Taiho Ventures, and MPM Capital.
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