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23rd April 2020

Ribomic is a Japanese clinical-stage pharmaceutical company founded in 2003, that develops aptamer therapeutics. The core drug 
On the April 10, 2020 - RIBOMIC, Inc., announced a press release that submitted an Investigational New Drug Application (IND) to the Medicines Agency (PMDA) in Japan to test its novel drug RBM-007, an anti-Fibroblast Growth Factor 2 (FGF2) aptamer to treat Achondroplasia.

The RBM-007 is currently under clinical trial in the USA for the treatment of ocular disease (age-related macular degeneration). RIBOMIC expects to begin phase 1 clinical trial until the end of June 2020 (Q1 Japanese fiscal year) at a single site (hospital) in Japan with a total of 24 healthy adult males to assess the safety, tolerability, and pharmacokinetics of RBM-007.

About RBM-007 and action mode

RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo.
In this post in Beyond Achondroplasia, you can read a comprehensive report about this innovative molecule. RBM-007 has been shown to have potent effects in limiting excessive interactions between fibroblast growth factors. In achondroplasia, the FGFR3 receptor to overly active when binding to growth factors such as FGF2, which leads to reduced growth of chondrocytes, resulting in short stature. By inhibiting the binding of FGF2 to FGFR3, RBM-007 has demonstrated therapeutic effects in studies using animal models of Achondroplasia and patient-derived iPS (induced pluripotent stem) cells.

1. Ribomic press release
2. Ribomic technology
3. Clinical research phase studies - FDA
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