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RNA-Targeting Small Molecules Pass Their First Pharma Test

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Wayfinder Biosciences advances Daiichi Sankyo collaboration on RNA-targeting small molecules to lead optimization.

Here’s the thing about “undruggable” targets. They’re usually only undruggable if you’re trying to hit the protein. Go after the RNA that makes the protein, and the whole equation changes.

Wayfinder Biosciences builds small molecules that modulate targets at the RNA level, and Daiichi Sankyo liked the initial results enough to advance their collaboration into a second phase. The first discovery phase, focused on an undisclosed neurodegeneration target, produced RNA-targeting drug candidates. The second phase moves into lead optimization, chasing potency, selectivity, and oral bioavailability.

No financial terms were disclosed, which is standard for these multi-phase collaborations. But the signal here isn’t in the dollars. It’s in the fact that Daiichi Sankyo, a major pharma with plenty of internal R&D resources, looked at Wayfinder’s candidates and decided to keep going.

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RNA-targeting small molecules are an emerging category that sits between traditional small molecule drugs and the siRNA/ASO world. The idea is to get the target selectivity of RNA-based therapeutics with the oral bioavailability and manufacturing simplicity of a pill. Several companies are working in this space, but partnered programs with major pharma that clear their first milestone and advance? Those are rarer.

Wayfinder is Seattle-based and preclinical-stage, so this is still early. But neurodegeneration is a space full of targets that have resisted conventional approaches for decades. If RNA-level intervention can crack even one of those targets, the implications go well beyond this particular collaboration.

The target remains undisclosed, which is typical for discovery-stage pharma partnerships. But given Daiichi Sankyo’s oncology strength and Wayfinder’s stated focus areas, a neurodegeneration target suggests the pharma partner is specifically seeking capabilities outside its comfort zone. That’s when these collaborations tend to be most meaningful.

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