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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Conversation 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?”

Frequently Asked Questions

What is the 'phenotype-first' target discovery engine?
It’s a computational framework that mines decades of longitudinal clinical registry data—like the Global Lysosomal Storage Disorders Registry—to identify molecular chokepoints correlated with rate-of-decline, not just static biomarkers. Unlike target-first approaches, it starts with human disease trajectories, then maps upstream genomic and proteomic perturbations using causal inference networks, not correlation. The engine has generated two Phase I candidates now in IND-enabling studies.
Did John Ness really publish failed preclinical assays publicly?
Yes—since 2019, Veridian has released all non-proprietary preclinical assay data, including negative results, via its OpenTox platform. This includes 17 failed AAV capsid variants for CNS delivery and cytotoxicity profiles across 42 off-target cell lines. The goal is to reduce redundant experimentation across academia and biotech, accelerating collective learning without compromising IP on lead candidates.
How does Veridian enforce its 'no-exclusion' pricing pledge?
Through tiered licensing: high-income countries pay full list price; upper-middle-income countries pay 60%; lower-middle and low-income countries pay cost-plus-5%, funded by cross-subsidization. Veridian also licenses manufacturing tech royalty-free to WHO-prequalified facilities in LMICs, with technical transfer support baked into the deal—not as charity, but as contractual obligation.
What role did patient families play in Veridian's founding governance?
They hold 40% of Class B shares, which carry veto power over trial halts, protocol amendments affecting quality-of-life endpoints, and any M&A activity. This was codified in the company’s charter—not as advisory input, but as binding fiduciary authority. The first such veto occurred in 2021, when families blocked enrollment expansion into asymptomatic adults until pediatric safety data matured.

Topics

rare diseasesdrug developmentpharmaceuticals

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