Neurodegeneration
Investigating the multicellular environment of neurodegenerative disease, with ALS as a principal disease context.
Dysregulated signaling can shape the neural environment, including glial responses and extracellular matrix remodeling. The NVP-13 hypothesis considers this wider tissue context, with ALS as a principal disease setting.
Independent Japanese and Korean studies found that different TGF-β pathway inhibitors prolonged survival in ALS mouse models. A 2026 CSF proteomics preprint reports stage-related TGFBR2 changes in ALS. Together, they highlight the importance of the TGF-β pathway, including TGFBR2, as a focus for ALS research. We are now investigating NVP-13 efficacy in ALS-relevant models in collaboration with Qilu Hospital.
Exposure, target modulation and functional benefit are different questions. Each requires disease-specific evidence.
Explore the NVP-13 approachNVP-13 / TGFBR2
Published NVP-13 studies show TGFBR2 reduction and lower pathway activity in human cell models, as well as neurogenic-niche changes in healthy non-human primates. Our current collaboration with Qilu Hospital is investigating NVP-13 efficacy in ALS-relevant models.
Public evidence and development statusNVP-13 · Preclinical research
In healthy non-human primates, intrathecal NVP-13 reduced CNS TGFBR2 expression and changed neurogenic-niche measures. This was not an ALS disease model or a test of patient benefit.
DOI: 10.1007/s13311-021-01045-2NVP-13 · Preclinical research
Reports reduced TGFBR2 RNA and protein and lower pSMAD2 in human neural precursor and lung-derived cell models. These cell findings do not establish clinical safety or efficacy.
DOI: 10.3390/ijms21061952NVP-13 · Non-GLP monkey study
Reports a phased non-GLP intrathecal NVP-13 toxicity and pharmacokinetic program in healthy cynomolgus monkeys. It does not test ALS efficacy or establish human safety.
DOI: 10.3390/app12030973NVP-13 · GLP monkey study
Reports a 13-week GLP repeated-dose intrathecal toxicity study in healthy cynomolgus monkeys. The paper title is retained verbatim; its findings do not establish clinical safety or therapeutic benefit.
DOI: 10.3390/pharmaceutics14010200Independent literature · ALS mouse study
In SOD1G93A ALS mice, a TGF-β signaling inhibitor given after disease onset extended survival. The intervention was not NVP-13 or a TGFBR2-targeting ASO.
DOI: 10.1016/j.celrep.2015.03.053Independent literature · ALS mouse study
In an ALS mouse model, early treatment with the TGF-β pathway inhibitor halofuginone delayed deterioration and prolonged survival. Halofuginone is not NVP-13 or a TGFBR2-selective ASO.
DOI: 10.1111/jnc.16102Independent literature · Unreviewed CSF preprint
CSF proteomics found stage-associated TGFBR2 changes and associations with poorer function and longer disease duration. This preprint does not, from the reported text alone, establish a general ALS-versus-control increase.
DOI: 10.64898/2026.04.13.26350753