PBSS Symposium Highlights & Key Takeaways
July 21 – 22, 2025
In July 2025, I had the privilege of co-organizing and moderating the PBSS Frontiers in Neuroscience: Emerging Biology and Therapeutics Symposium, a two-day virtual meeting bringing together leaders across academia, biotech, and pharma. The event marked the launch of PBSS’s new neuroscience-focused symposium series, beginning with neurodegeneration and expanding into broader neurological disorders in future years.
As organizer and moderator—and as the opening speaker—I had the opportunity to frame the scientific landscape, highlight key unmet needs, and set the stage for an outstanding lineup of experts who shared their latest discoveries and translational advances.
Why Neuroscience, Why Now?
My opening remarks emphasized a central reality: we are facing a global, accelerating crisis in neurological disease, with staggering human, societal, and financial costs.
Over 50–60 million people are currently living with neurodegenerative conditions, a number expected to double every 20 years.
Dementias—primarily Alzheimer’s and vascular dementia—represent the majority of cases, but Parkinson’s disease, ALS, Huntington’s disease, and frontotemporal dementia (FTD) collectively impose enormous burden.
The economic impact is projected to exceed $9 trillion annually by 2050.
Despite this urgency, neurology consistently shows the lowest clinical development success rates across therapeutic areas. The reasons are complex: limited CNS penetration, insufficient predictive models, biomarker scarcity, patient heterogeneity, and long development timelines. Yet, as I highlighted in the keynote, we are also witnessing a true inflection point—with advances across biologics, small molecules, RNA-targeting therapies, gene therapy, and stem-cell based approaches.
Key Themes Across the Two-Day Program
1. Biomarkers & Patient Stratification
Dr. Kalpana Merchant opened Day 1 with a deep dive into advances in Parkinson’s disease biomarker discovery, a foundational challenge for improving trial design and accelerating therapeutic development.
From CSF markers to imaging tools and genetic risk stratification, the message was clear: validated biomarkers remain essential to de-risk CNS drug development, especially in late-stage trials.
2. Crossing the Blood–Brain Barrier (BBB)
From Denali’s Transport Vehicle (TV) platform, Dr. Joe Lewcock shared promising data enabling biologics to cross the BBB—addressing one of the most persistent obstacles in neuroscience therapeutic delivery.
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This technology represents a meaningful shift in what modalities can realistically be explored for CNS indications.
3. Small Molecules: Novel Targets & Modalities
Speakers from Nura Bio, Verge Genomics, Neuron23, and BNDC showcased diverse approaches, including:
SARM1 inhibitors to block axonal degeneration
A PIKfyve inhibitor (VRG50635) advancing toward ALS indications
TYK2 inhibition for MS and neuroinflammatory diseases
APOE4-targeted therapies for neurodegeneration
Collectively, they illustrated how small molecules continue to provide tractable, scalable options across neurodegenerative disorders.
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4. Human iPSC Models & Organoids
Day 2 began with compelling presentations from Rumi Scientific and NeuCyte, demonstrating how:
Human iPSC-derived organoids
3D neuroimmune co-culture systems
are reshaping disease modeling and early-stage discovery. These platforms directly address the translational gap highlighted in my keynote: over 80% of animal model findings fail to translate to humans.
Frontiers in Neuroscience_talk_…
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5. Autologous Stem Cell Transplantation
Dr. Andres Bratt-Leal (Aspen Neuroscience) presented encouraging early clinical data on autologous iPSC-derived dopaminergic cell transplantation for Parkinson’s disease—a long-awaited step toward personalized cell therapies.
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6. AI/ML-Enabled Drug Discovery
Insitro’s Aj Kaykas highlighted machine learning approaches for ALS target discovery, demonstrating how large-scale multi-omic integration can accelerate precision target identification.
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7. RNAi and Gene-Modulation Therapies
Day 2 concluded with compelling innovations in gene-regulatory therapeutics:
AcuraStem’s UNC13A-targeted ASO (AS-241) for ALS and FTD
Alnylam’s expansion of RNAi-based therapeutics into CNS indications
These modalities underscore a paradigm shift: RNA-based therapies are maturing into viable, disease-modifying interventions for complex CNS disorders.
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A Renewed Landscape of Innovation
Across both days, a consistent theme emerged:
Neuroscience is no longer “too hard.” It is finally becoming tractable—scientifically, technologically, and clinically.
Multiple speakers presented breakthroughs that would have been improbable just a decade ago: reliable BBB shuttles, patient-specific organoid models, precision genetic targeting, and maturing RNAi and ASO pipelines.
This convergence of innovation is beginning to translate into real impact, as reflected in the recent FDA approvals I highlighted during my opening talk.
