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RIBOMIC, Inc. is a leading company in the world developing innovative aptamer therapeutics.

In-House Drug Discovery Programs

Overviews of our major in-house programs are as follows.

PAIN (RBM004 : anti-NGF aptamer)

Pain is an important signal to indicate the abnormality of the body, and sometimes impairs the quality of life (QOL), causing various problems in life. Hence, many painkillers have been developed and launched.
   We aim to generate an innovative painkiller, which should meet the unmet medical needs, by raising therapeutic aptamer against nerve growth factor (NGF).
   NGF is the first-discovered growth factor and is known to cause pain in the periphery. It is likely that inactivation of NGF could weaken or remove pain. Therefore, several global pharmaceutical companies like Pfizer have been engaged in developing anti-NGF antibodies as novel effective painkillers.

<Transmission of pain by NGF>

Transmission of pain caused by NGF

Our home-made anti-NGF aptamer strongly blocks NGF function as does Pfizer’s anti-NGF antibody, having strong painkilling efficacy. Unlike the Pfizer’s antibody, which is a long-lived painkiller upon administration, RIBOMIC’s anti-NGF aptamer is a relatively short-lived painkiller, whose efficacy continues 2-7 days (at longest 2 weeks) upon administration. This is a superior characteristic compared with certain antibodies, whose long-lived efficacy happened to cause unwanted side effects and could not be ceased in the body.
   In April 2014, we entered into a world-wide exclusive license agreement for the anti-NGF aptamer (RBM004) with Fujimoto Pharmaceutical Corporation. We do our best for its development together with Fujimoto to achieve POC, which in turn expands our experience and knowledge of clinical development.

SEPSIS (RBM005: anti-HMGB1 aptamer)

Sepsis is a fatal disease caused by bacterial, viral and fungal infection that triggers uncontrollable host immune response (inflammation) and severe damages in tissues and organs leading to the multiple organ failure and fatal shock. To meet the unmet medical needs for sepsis, we are developing therapeutic aptamers against HMGB1 (high mobility group box 1), which is known to be secreted from dying cells upon progression of sepsis.

LUNG FIBROSIS, AND OTHER ORGAN FIBROSIS (RBM006: anti-ATX aptamer, RBM005, RBM007, and RBM008)

Fibrosis is a severe disease that occurs in several tissues and organs including lung, liver, kidney and muscle, leading to fatal dysfunction. Among these, our R&D primarily focuses on idiopathic pulmonary fibrosis (IPF), which has limited cure in the treatment and often progresses to lung cancer.
   We have been studying several targets in the discovery program to respond for the unmet medical needs in tissue and organ fibrosis. Of those, our primary target is autotaxin (ATX), the enzyme responsible for the production of lysophosphatidic acid (LPA) from lysophosphatidyl choline (LPC). ATX is known to be up-regulated in many inflammatory conditions, including but not limited to cancer, arthritis, multiple sclerosis, and lung fibrosis.

BONE DISEASES (RBM007: anti-FGF2 aptamer)

Unmet medical needs remain in bone diseases such as joint destruction from arthritis, and bone metastasis from cancer, because of limited current treatment.
   Fibroblast growth factor 2 (FGF2) is involved in homeostasis (wealthy balance) of bone growth, absorption and regeneration, and its up-regulated imbalance is known to cause bone destruction diseases often associated with severe pain. Although there are several drugs available to prevent or cure inflammation associated with bone diseases, there exist a limited number of drugs primarily for bone destruction disorders.
Our developing anti-FGF2 aptamer exhibits a dual therapeutic impact in animal models on bone diseases such as rheumatoid arthritis, osteoporosis and achondroplasia (ACH), as well as on severe pain associated with bone cancer pain. These perspectives remain to be addressed in clinical studies.

EYE DISEASES AND INFLAMMATORY DISORDERS ( RBM008: anti-periostin aptamer, RBM007 )

Diabetic retinopathy (DR) is the second leading cause of blindness in Japan (2013), accounting for more than 3,000 cases every year. The third-leading cause of blindness in Japan is age-related macular degeneration (AMD), while AMD is the first-leading cause of blindness in Europe and the United States.
   We aim to solve these unmet medical needs in eye diseases by generating therapeutic RNA aptamers against periostin (for DR) and FGF2 (for AMD). Anti-periostin aptamer should be useful as well to cure severe atopic dermatitis since periostin is known to be involved in inflammation-triggered chronic atopic dermatitis and its worsening.