The pathologies of rare diseases are difficult to understand because of the small number of patients impacted. For these individuals, limited treatment options are available in clinical practice. However, we must keep challenging ourselves to develop therapeutic drugs for diseases in uncharted areas, even if we are uncertain whether we will succeed. Otherwise, we would be abandoning the possibility of treatment.
To deliver innovative pharmaceuticals for the treatment of rare diseases for which no effective therapy is available or for which existing treatments are not fully effective, Daiichi Sankyo continues to take on the challenge of offering hope to patients with rare diseases by taking advantage of our strengths in science and technology.

• The Duchenne muscular dystrophy treatment drug DS-5141, which is being co-developed with the Orphan Disease Treatment Institute*1, is a candidate that is applied via a new modality*2 developed by the Company, ENA*3 nucleic acid modification. DS-5141 is currently being further analyzed on the basis of the results of phase 1/2 clinical trials in Japan.

• DS-4108, a glycogen storage disease type Ia (GSDIa) treatment drug using the same technology, is undergoing a collaborative preclinical research program with Kobe Gakuin University, National Center for Child Health and Development, and Hiroshima University.

• The TNAP*4 inhibitor DS-1211, which targets pseudoxanthoma elasticum, has been evaluated in phase 1 clinical trials in the United States.

• The anti-ALK2 antibody DS-6016 targeting fibrodysplasia ossificans progressiva has started phase 1 clinical trials in Japan. (The preclinical research is a collaborative effort with Saitama Medical University and was selected for AMED’s CiCLE*5 program in August 2017.)

*1 A joint investment company set up through funds operated by the Innovation Network Corporation of Japan and Mitsubishi UFJ Capital Co., Ltd., and investment from Daiichi Sankyo
*2 A therapeutic method such as a small molecule drug, nucleic acid drug, therapeutic virus, and cell therapy
*3 2’-O,4’-C-Ethylene-bridged Nucleic Acid, which is a modified nucleic acid using Daiichi Sankyo’s unique technology. “ENA®” is a registered trademark of Daiichi Sankyo Co., Ltd.
*4 Tissue-nonspecific alkaline phosphatase
*5 Cyclic Innovation for Clinical Empowerment supported by the Japan Agency for Medical Research and Development (AMED)


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