1:40 PM
 | 
Jan 03, 2018
 |  BC Innovations  |  Product R&D

Expanding small molecule horizons

How 5AM newco Expansion Therapeutics is hitting RNA targets with small molecules

Editor's Note: This article was updated on Jan 04, 2018 at 2:22 PM PST

With a platform boasting more than a decade of preclinical rigor and $55.3 million in series A funding, Expansion Therapeutics Inc. is the latest company to step into the RNA-binding small molecule arena. The newco is setting its sights first on pathogenic RNA repeat expansions responsible for a range of rare genetic disorders.

As the most recent graduate of 5AM Ventures’ 4:59 Initiative incubator, Expansion emerged from stealth mode yesterday, after twelve months’ work with scientific founder Matthew Disney, a pioneer in the field who is professor of chemistry at The Scripps Research Institute.

5AM was joined in the series A by Sanofi Ventures, Novartis Venture Fund, Kleiner Perkins, RA Capital Management and Alexandria Venture Investments. Expansion President and CEO Kevin Forrest said that the involvement of corporate VCs reflects emerging interest in the field and that Sanofi Ventures, which joined in the seed round, makes “a great strategic fit” because it is one of the industry leaders in orphan diseases.

The announcement marks the third newco formed in as many years that aims to expand the scope of disease targets druggable by small molecules by developing compounds that bind RNA rather than protein.

Last March, Arrakis Therapeutics Inc. announced a $38 million series A round to use its bioinformatics and chemical biology platform to identify RNA-binding compounds for cancer, CNS diseases and rare genetic conditions. In 2015, Ribometrix Inc. was formed around a pair of high throughput platforms for screening RNA-binding molecules that were developed by Kevin Weeks, a professor at the University of North Carolina at Chapel Hill. Funding was not disclosed.

The budding field is part of the larger trend towards drug companies paying more attention to RNA.

In addition to the lengthening list of known types of RNA molecules that can serve as therapeutic agents or targets, such as siRNA, microRNA (miRNA), long non-coding RNA (lncRNA) and circular RNA, the field has identified epigenetic sites on RNA that form the platform of at least one newco, Storm Therapeutics Ltd., which launched in 2016.

Moreover, some companies and academic groups have stumbled upon small molecules that bind RNA or its translational machinery without a systematic screening method, such as an inhibitor of PCSK9 translation discovered last year by Pfizer Inc.

Traditionally, small molecules are designed to bind highly defined and stable pockets formed by the tertiary structure of protein targets. While the modality has several stability and PK advantages over biologics and nucleic acids, it is limited by...

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