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Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
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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].



  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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