11.06.2025
Ion channel gene therapies
In this latest blog, Marc Rogers reviews the current status and future potential of ion channel gene therapies — from gene replacement and editing to RNA-based methods that modulate channel expression. These approaches are now being applied to a broad range of therapeutic areas including pain, epilepsy, cystic fibrosis, and retinal diseases.
After academic research as a neuroscientist and 20 years of commercial ion channel drug discovery, Marc Rogers now works as a freelance blogger, advisor and consultant for clients in the UK, EU and US where he shares his expertise and enthusiasm for all aspects of ion channel screening. He is particularly interested in automated patch clamp, and exploiting the potential of human iPS stem cell assays to facilitate the successful translation of new drugs into the clinic.
The blog highlights how these genetic techniques are being used to correct mutations, regulate gene expression, or reduce disease-driving ion channel activity, often with greater selectivity and duration than traditional small molecules.
Case studies include programs targeting Nav1.7, Nav1.1, Nav1.2, Kv1.1, CFTR, and Kir7.1, using tools such as AAV vectors, ASOs, RNAi, CRISPR, and epigenetic modifiers. The role of delivery vectors, safety challenges, and functional readouts (e.g. automated patch clamp, iPSC models) are also discussed.
This blog offers a detailed but accessible overview of how gene therapy is being integrated into ion channel drug discovery, and where the field may be headed next.
