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KDDF-201304-03 Development of therapeutic agent for duchenne muscular dystrophy by targeting NMD(Others, Chemical) [07.23.2013]


Development and Market Objectives

To develop a medicine for hereditary conditions including duchenne muscular dystrophy (DMD), a rare disease arising from nonsense mutations, by developing novel compounds through cellular-based screening and optimization.

Unmet Medical Need & Target Patients

DMD is a rare disease, but affects more than 30,000 people in America and Europe. No lasting treatment for DMD currently exists on the market. 

The development of treatments that can improve dystrophin protein expression, a primary factor in DMD, is critically needed.

Other hereditary diseases derived from nonsense mutations can potentially be diagnosed by genomic analysis, but current medicinal methods are very limited. 

Hereditary diseases that arise from nonsense mutations cause more serious symptoms than those of other genetic disorders. Therefore, radical treatments targeting these diseases are urgently needed. 

Prolonged treatment with a low molecular weight compound is ideal.


(i) NMD / Researching and discovering inhibitor nonsense mutations
(ii) Lead materialization of identified lead candidates
(iii) Currently in progress with research to explore the possibility of clinical trials with DMD patients.

Intellectual Property

The pathological cause of rare diseases derived from nonsense mutations and DMD is relatively simple. For the case of DMD, the function of dystrophin is downregulated, and therefore, the conduct of preclinical-clinical translation for radical treatments is expected to be easier than for other diseases. This allows project planning in the near future for the discovery of new compounds without patent infringement. 

Extent of Patent: 
1. New compounds that inhibit NMD specifically, and their derivatives. 
2. The new compounds in # 1, and protein translation inhibitors and their derivatives
3. The new compounds in # 1 and hereditary disease medicines and their derivatives

Competitive Advantages

Our research team is currently in the process of discovering NMD inhibitors by targeting all stages of NMD using an NMD-relevant cell line optimized for inhibitor screening. Inhibitors discovered from this process will have a big advantage in that we can apply it to DMD which arises from NMD, and many other hereditary diseases.

This research will be undertaken through cooperation with the Green Cross Corp., a leading pharmaceutical company in Korea that has successful experience in developing new drugs, and Professor Chae Jong Hee’s team from Seoul National University Hospital, whose primary research focus is in DMD clinical trials.



Research Period

Jul. 01. 2013 ~ Apr. 30. 2014


Korea University

Developmental Stage

Lead Generation

Additional Information

Contact Information

Address Company Name: Korea University
WebSite Homepage: Contact Person: Kim, Yoon Ki
E-mail: Contact: +82-2-3290-3410

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