Targeting RNA with Small Molecules to Drug the Undruggable: new report analyzes companies, investors, partnerships, technologies and targets from an industry perspective

Promotion of report with 10% discount

STUTTGART, Germany I November 6, 2019 I Until November 15, 2019, La Merie Publishing is promoting with a 10% discount its newest report “RNA-Targeted Small Molecules 2019: a landscape analysis of companies, technologies, targets, investors and partners from an industry perspective”.

RNA as a target holds the promise of dramatically increasing the pool of druggable targets as compared with conventional protein targets. RNA as a therapeutic drug has validated this concept, but has limitations such as delivery issues and toxicity. Thus, small molecules targeting RNA is an attractive concept for the pharmaceutical industry.

The report describes and analyzes from an industry perspective with a view on emerging technology companies regarding their founding background, financial history, technological capabilities, target and indication selection and most advanced lead compounds

The potential therapeutic value of targeting RNA is well recognized. So far, intentionally targeting RNA has required oligonucleotide drugs with challenging pharmaceutical properties. Nevertheless, there are precedents for RNA-targeted small molecules, including several approved drugs (e.g ribocil targeting bacterial mRNA). In contrast to oligonucleotide drugs, these molecules were all discovered through serendipity; only after their discovery was it learned they acted on RNA.

Technological advances mainly in the past decade have revolutionized the understanding of co-transcriptional and post-transcriptional control of gene expression. It has become clear that the different steps of pre-mRNA processing, from 5' capping to nuclear export, are tightly co-regulated and coordinated with transcription, translation and mRNA decay. Furthermore, recent discoveries and the functional characterization of several chemical modifications in mRNA such as N6-methyladenosine (m6A) and 5-methylcytosine (m5C), which together form the epitranscriptome, have revealed a new layer of regulation of mRNA processing and maturation.

While RNA translation regulation and splicing modification are older approaches characterized by advanced stages of drug development, direct and indirect RNA targeting are emerging technologies pursued by key players in the field which were founded only in recent years. Accordingly, these companies are focused on establishing their technologies at an industrial scale, start lead programs, discover lead molecules and use them for preclinical proof-of-concept followed ideally by clinical validation.

The key technology players for RNA translation regulation and splicing modification have attracted great interest from Big Pharma companies as evidenced by five drug discovery collaborations which make Skystar the best funded RNA-targeted small molecule company. Due to the earlier stage of technologies for direct and indirect RNA-targeting with small molecules, no collaborations between technology and major pharmaceutical companies have been closed so far. However, Big Pharma is supporting these technologies by participation in financing rounds.

The report can be acquired at La Merie Publishing’s online store at

About La Merie Publishing

La Merie Publishing is an independent business information provider for the biotechnology and pharmaceutical industry. La Merie Publishing prepares brief and full reports as well as competitor analysis reports and target pipeline lists, the latter two in a tabulated format with structured listings of industry-relevant data. La Merie Publishing products can be purchased in our new online store at and at selected Resellers.

The focus of our products is directed towards therapeutic biologics, such as antibodies, proteins, peptides, vaccines and gene & cell therapy. We pay special attention to biosimilar antibodies and proteins, antibody drug conjugates, bispecific antibodies, TCR & CAR engineered antibodies, mRNA vaccines & therapeutics, oncolytic viruses and immuno-oncology in general.

To leverage our expertise and knowledge accumulated in our in-house database, we offer custom report preparation services for corporate clients, including, but not limited to, pipeline analysis reports, drug profiles and any other competitive intelligence elaboration of interest for our clients.

SOURCE: La Merie Publishing

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