Our published work.

Clinical and Translational Science · DOI 10.1111/cts.70489

Gontier G, Kim G, Wang C, Zhu K, Gau R, Zhang N, Naphade S, Stewart A, Schmidt MJ, Galemmo R, Shook B, Tarachandani A, Choi I, Raines S, Scannevin RH, Grievink HW, Smits LMG, Kremer PHC, Cadavid D

Verge identified GPNMB, a protein measurable in blood and brain, as a clinical biomarker confirming VRG50635 engages its target (PIKfyve). Demonstrated across cells, animals, and ALS patients dosed in the Phase 1 trial.

Annals of Neurology (submitted Dec 2025)

Cadavid D, et al. (Verge VRG50635 team)

Top-line results from Verge’s proof-of-concept Phase 1b ALS trial of VRG50635 — safety, pharmacokinetics, biomarker effects, and digital endpoints across familial and sporadic ALS patients.

Frontiers in Immunology (submitted Oct 2025)

Heinrich L, Choi I, Scannevin RH, Dhara M

Verge showed that inhibiting CD38, the target of Verge’s obesity candidate VRG201, reshapes T cell metabolism and reduces inflammation, supporting CD38 as an immunometabolic drug target.

Molecular Neurodegeneration · DOI 10.1186/s13024-023-00699-0

Martirosyan A, Ansari R, Pestana F, Hebestreit K, Ghosh H, Poovathingal S, Hadzhiev Y, Hadziahmetovic A, Cisterna A, Hardy J, Mighdoll M, Scannevin R, Kottick A, Hanson-Smith V, Guelfi S, et al.

Single-nucleus RNA sequencing of post-mortem brain tissue from Parkinson’s patients mapped how every cell type responds to disease, identifying vulnerable dopaminergic neuron populations and new molecular leads for PD drug discovery.

bioRxiv (preprint) · DOI 10.1101/2022.12.13.514863

Gómez-Pascual A, Martirosyan A, Hebestreit K, Mameffe C, Poovathingal S, Belgard TG, Altar CA, Kottick A, Holt M, Hanson-Smith V, Cisterna A, Mighdoll M, Scannevin R, Guelfi S

Computational analysis of single-nucleus PD data suggests iron accumulation in dopaminergic neurons drives their selective vulnerability in Parkinson’s, pointing to iron homeostasis as a target axis.

Scientific Reports (Nature Portfolio) · DOI 10.1038/s41598-021-04583-z

Naphade S, Bhatnagar R, Hanson-Smith V, Choi I, Zhang A

Verge benchmarked low-input RNA-seq library prep kits, providing the methods foundation for high-throughput transcriptomics on small biological samples used across the CONVERGE platform.

PLOS Computational Biology · DOI 10.1371/journal.pcbi.1007770

Lamparter D, Bhatnagar R, Hebestreit K, Belgard TG, Zhang A, Hanson-Smith V

Verge published BAGEA, a Bayesian computational method that combines genomic annotations with eQTL data to predict how genetic variants change gene expression. A core building block of the CONVERGE target discovery platform.

Nature Medicine · DOI 10.1038/nm.4490

Shi Y, Lin S, Staats KA, ..., Chen JA, Hanson-Smith V, Belgard TG, Zhang A, et al.

Foundational paper (Verge co-authored) showing that loss of the C9ORF72 gene drives motor neuron death in C9-ALS and frontotemporal dementia through disrupted cellular waste clearance and excitotoxicity. Underpins Verge’s ALS scientific strategy.