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RMB-007

What is RMB-007 and how does it work?

RMB-007 is a potential drug under development for achondroplasia by Ribomic Inc. and it's an aptamer. This company deals only in these small nucleic acid molecules and is testing several of them for a few different diseases (RMB-007 is currently being tested for 3 groups of diseases, among them achondroplasia).

Being an aptamer, RMB-007 is highly specific for a molecule, Fibroblast Growth Factor 2 (FGF2), one of the signaling molecules that activate Fibroblast Growth Factor Receptor 3 (FGFR3), the cell receptor that's constitutively activated in achondroplasia. FGF2 has been shown to have an important role in bone homeostasis [1].

It acts in a similar fashion to Therachon's TA-46 by targeting molecules that activate FGFR3. The main difference is that RMB-007 doesn't mimic the receptor and, instead, targets a specific signaling molecule. Specifically: where TA-46 targets all FGFs capable of binding to FGFR3 (but not necessarily of activating it), RMB-007 binds to FGF2 in particular, blocking the signals this molecule would provide for FGFR3 [1].

What stage if development is it in?

This drug is still in pre-clinical studies, in a Toxicologic Good Laboratory Practices study [2].

Sources

 

  1. Jin L, Nonaka Y, Miyakawa S, Fujiwara M, Nakamura Y. Dual Therapeutic Action of a Neutralizing Anti-FGF2 Aptamer in Bone Disease and Bone Cancer Pain. Molecular Therapy. 2016;24(11):1974-1986. doi:10.1038/mt.2016.158.
  2. Ribomic Inc. Pipeline of Drug Discovery. Our Business 2017 [cited 2017 28/11].
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