The Vanderbilt Industry Collaborations team recently welcomed Gondola Bio, a privately held biopharmaceutical company spun out of BridgeBio Pharma and focused on developing therapeutics for genetically defined diseases, for a two-day campus visit coordinated by Swapneeta Date, PhD, Assistant Director of Life Sciences Collaborations. Gondola Bio's strategy centers on identifying rare diseases with well-characterized genetic drivers and high unmet clinical need, then partnering with academic researchers to develop targeted therapies that address the disease at its genetic source.
The event was focused on accelerating translational research, fostering scientific exchange, and exploring new opportunities in genetic disease therapeutics. It highlighted the growing importance of collaboration between academic researchers and industry partners in moving scientific discoveries toward clinical impact.
Scientific Scope of the Visit
The program showcased Vanderbilt's depth of expertise across genetics, rare disease, neuroscience, drug discovery, drug delivery, and translational medicine — drawing researchers from across VU and VUMC whose work collectively spans biomedical engineering, nephrology, matrix biology, cancer drug discovery, neuroscience, and neurodevelopmental disorders. Vanderbilt's therapeutic assets and discovery platforms in rare genetic disease were a particular focus, reflecting the institution's experience across multiple drug modalities including small molecules, antibody therapeutics, siRNA conjugates, and antisense oligonucleotides (ASOs).
Discussions highlighted several programs with first-in-class and best-in-class potential, including:
- Next-generation siRNA conjugates for CNS disorders: engineered oligonucleotide platforms designed to silence disease-driving genes in the central nervous system
- Rett Syndrome — research addressing the synaptic and circuit-level consequences of MECP2 loss-of-function mutations in this severe X-linked neurodevelopmental disorder
- Alport Syndrome and Goodpasture Syndrome: programs grounded in Vanderbilt's decades of expertise in type IV collagen biology and glomerular basement membrane disease
- Primary Hyperoxaluria: research addressing this rare inborn error of metabolism characterized by excessive endogenous oxalate production, leading to progressive renal oxalate accumulation and end-stage kidney disease
- Pulmonary Arterial Hypertension and Systemic Sclerosis: programs exploring fibrotic and vascular remodeling pathways with a focus on serotonin receptor biology. Vanderbilt has developed selective 5-HT2B antagonists with demonstrated activity in preclinical models of fibrosis, with potential applications extending beyond pulmonary vascular disease to oncology and as safer adjuncts to psychoplastogen therapies
Featured Presenters
Researchers from across VU and VUMC contributed scientific presentations spanning the breadth of Vanderbilt's translational research enterprise:
- Alexander G. Bick, MD, PhD, Associate Professor of Medicine, Director of the Division of Genetic Medicine and Clinical Pharmacology, and Edward Claiborne Stahlman Chair
- Jennifer (Piper) Below, PhD, Professor of Medicine and Director of the Vanderbilt Genetics Institute
- Jill Pulley, MBA, Executive Director of the Vanderbilt Institute for Clinical and Translational Research (VICTR)
- Cheryl Lynn Gatto, PhD, Research Associate Professor of Medicine at Vanderbilt University Medical Center
- W. David Merryman, PhD, Walters Family Professor and Associate Chair of Biomedical Engineering
- Alex G. Waterson, PhD, Research Professor of Pharmacology and Chemistry and Director of the Vanderbilt Center for Cancer Drug Discovery
- Gordon Stott, PhD, National Institutes of Health
- Matthew H. Wilson, MD, PhD, Professor of Medicine in the Division of Nephrology and Hypertension
- Billy G. Hudson, PhD, Director of the Vanderbilt Center for Matrix Biology and Elliott V. Newman Professor of Medicine
- Sergey Budko, PhD, Research Assistant Professor of Medicine
- Ethan Lippmann, PhD, Associate Professor of Chemical and Biomolecular Engineering, Biomedical Engineering, and Neurology
- Colleen Niswender, PhD, Associate Professor of Pharmacology and Director of Molecular Pharmacology for the Warren Center for Neuroscience Drug Discovery
- Lisa Monteggia, PhD, Barlow Family Director of the Vanderbilt Brain Institute and Professor of Pharmacology
- Jeffrey L. Neul, MD, PhD, Director of the Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, and Professor of Pediatrics
About the Industry Collaborations Team
The Industry Collaborations team identifies, supports, and stewards corporate research and development partnerships across Vanderbilt University and Vanderbilt University Medical Center. Focusing on Life Sciences and Physical Sciences, the team cultivates collaborative research partnerships in a variety of industries — from automotive to pharmaceuticals to defense technology. The team's focus is to grow Vanderbilt's industry-sponsored research portfolio with partners who seek to further develop and commercialize Vanderbilt technologies. Connect with the team.