Interview with Dan Tardiff - CSO of CAMP4 Therapeutics

Welcome to the Biotech Voyager. It's the show that covers what's next in biotech, mapping the early-stage signals before their big news. Hosted by Benjamin McLeod and Jeff Martin, PhD. Dan Tardiff, Chief Scientific Officer of CAMP4 Therapeutics, joins the show to explain how his company is using antisense oligonucleotides to upregulate gene expression by targeting regulatory RNAs, why haploinsufficient disorders represent the ideal therapeutic window for their platform, and why a modest twofold increase in transcription could transform treatment for rare neurodevelopmental diseases. CAMP4 has developed a novel approach to gene upregulation that targets non-coding regulatory RNAs transcribed from promoters and enhancers rather than the genes themselves. Dan walks through the biology of these regulatory elements, explaining how nearly universal transcription from promoters and enhancers creates a kinetic trap for transcription factors that can be modulated with antisense oligonucleotides. The platform uses genome-wide mapping techniques including chromatin accessibility, histone modifications, and Hi-C sequencing to identify regulatory regions, then confirms RNA expression through long-read sequencing before screening for therapeutic oligonucleotides. The conversation explores why ASOs offer advantages over small molecules and gene therapy for certain indications, how CAMP4's lead program in SYNGAP1-related disorder compares to Stoke Therapeutics' splice-switching approach in Dravet syndrome, the technical challenges of intrathecal delivery versus emerging blood-brain barrier shuttle technologies, and why the company expects to initiate Phase 1/2 clinical trials by the end of the year following regulatory approval in Australia and Argentina. New episodes every Tuesday and Thursday at 11:00 AM. #biotech #CAMP4therapeutics #ASO #genetherapy #CNS #raredisease