NVP-13
NVP-13 is an LNA-gapmer antisense oligonucleotide designed to reduce TGFBR2 expression.
Can reducing TGFBR2 expression modify pathological tissue responses driven by dysregulated TGF-β signaling? This question connects the neurodegeneration and fibrosis branches of NVP-13.
The rationale is disease- and cell-context dependent. Target engagement alone does not establish therapeutic benefit.
The antisense approach
01
NVP-13 binds its complementary sequence in TGFBR2 RNA.
02
RNA degradation reduces the template available for receptor production.
03
Reduced receptor expression can alter TGF-β signaling responses.
Development rationale
The molecule and target provide the common thread. Disease-specific exposure, cell biology and functional outcomes must be established separately.
01 / NVP-13 · CNS
In human neural precursor cells, NVP-13 reduced TGFBR2 and pSMAD2, a marker of TGF-β signaling. We are investigating its potential in ALS with Qilu Hospital.
Explore the CNS branch
02 / NVP-13 · Tissue biology
Exploring the relationship between persistent TGF-β signaling, matrix accumulation and maladaptive tissue repair. The therapeutic hypothesis requires validation in each disease setting.
Explore the fibrosis branch| Program / target | Disease branch | Public evidence |
|---|---|---|
| NVP-13TGFBR2 · Antisense | Neurodegeneration | Published preclinical research Current development-stage wording awaits company confirmation. |
| NVP-13TGFBR2 · Antisense | Fibrosis | Therapeutic hypothesis Indication-specific development status awaits company confirmation. |
The cited publications do not establish the current IND, clinical-trial or regulatory status.
NVP-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/pharmaceutics14010200