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Small molecule drugs for previously untreatable diseases

Protein synthesis

16 August

Scientists have discovered a way to make safer, more versatile small molecule drugs that could pave the way to treatments for many currently hard-to-treat or untreatable diseases.

Traditionally, efforts in the drug discovery space have been geared towards finding small molecules that bind directly to proteins. However, approximately 80% of proteins are considered difficult to target or even ‘undruggable’ by these approaches, according to Yochi Slonim, CEO of NJ-headquartered Anima Biotech. Anima’s strategy focuses instead on discovering small molecules that have an input earlier on in the process – by binding to various other regulatory proteins that control protein production or translation in cells.

The company announced a collaboration back in 2018 with Eli Lilly that was worth 1bn in milestone payments. The collaboration was to discover and develop translation inhibitors for several undisclosed target proteins using Anima’s Translation Control Therapeutics platform.

‘Our Translation Control Therapeutics technology is the first platform for the discovery of small molecule drugs that specifically control mRNA translation as a new strategy against many diseases,’ says Slonim. ‘The technology not only allows us to reduce production, but also to ramp up production of the targeted protein – something that has never previously been possible,’ he explains.

For further details visit this month’s issue of Chemistry and Industry .


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