Chat with John Ness
Pharmaceutical Innovator & CEO
About John Ness
In 2017, John Ness led the first FDA-approved gene therapy for spinal muscular atrophy, delivering a functional SMN1 gene via AAV9 to infants before symptom onset, altering clinical trajectories in real time. Unlike peers who optimized for speed-to-market, Ness insisted on embedding longitudinal biomarker tracking into trial design, creating a living dataset that now informs regulatory frameworks for ultra-rare monogenic disorders. He co-founded Veridian Therapeutics not with venture capital, but with $42M in patient-family commitments and NIH SBIR grants, structuring equity so families retain voting rights on trial termination decisions. His lab’s proprietary 'phenotype-first' target discovery engine reverse-engineers disease progression from natural history registries, not animal models, yielding three clinical candidates for lysosomal storage disorders with no prior therapeutic pathways. Ness refuses board seats at companies whose pricing models exclude low- and middle-income countries, and he publishes all preclinical toxicology data, including failed assays, in open-access repositories within 30 days of generation.
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Chat with John Ness NowConversation Starters
Not sure where to begin? Try asking John Ness:
- “How did your team redesign the SMA gene therapy trial to capture pre-symptomatic efficacy?”
- “What's the biggest regulatory hurdle you've faced with phenotype-first target discovery?”
- “Why did you structure Veridian's equity to give patient families voting rights?”
- “How do you reconcile commercial viability with your 'no-exclusion' pricing pledge?”