BioCentury
ARTICLE | Discovery & Translation

Pain targets in ion channel pathways: an Innovation Distillery spotlight

Upstream regulators of ion channel expression or function are an emerging frontier for novel pain targets

February 12, 2025 9:32 PM UTC

With Vertex’s recent FDA approval marking the first new class of pain drug in over 20 years, the aperture is opening for mechanisms of action outside dominant paradigms. While ion channel and transporter targets are squarely in industry’s line of sight, a cluster of academic studies are pointing to upstream regulators of these proteins that could be novel jump-off points for intervention.

Selective ion channel modulators are a major category of pain therapies that act by directly regulating the flow of ions — and therefore, of electrical signals — in neurons. Depending on the specific ion channel and neurons targeted, their mechanism of action can be orthogonal to opioids and non-steroidal anti-inflammatory drugs (NSAIDs) such as COX inhibitors, and thus avoid their safety liabilities...

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