Daiichi Sankyo signals gene therapy intent with Ultragenyx deal

Daiichi Sankyo is some way from putting a gene therapy into the clinic, but it apparently has big ambitions: a tech transfer deal with Ultragenyx gives it broad access to the US biotech’s AAV-based technologies. The Japanese pharma giant said it wanted to secure commercially scalable manufacturing capabilities at the very start of its push into this space. This is understandable – available capacity to make these hugely complex products is a major issue, and one that is only likely to become more pressing as more products reach the market. Ultragenyx benefits from a $125m up-front payment and a $75m equity investment at $60 a share – a healthy 36% premium to yesterday’s close – and can opt in to rare disease projects. And, as the tie-up is not exclusive, similar deals are possible in the future. Except for Astellas, which has a big gene therapy presence thanks to its $3bn Audentes acquisition, and a few legacy Baxter and Shire projects that Takeda is continuing, big Japanese drug makers have not signalled a huge interest in this technology. Daiichi said it wanted to start the manufacturing of research projects by mid-2020, adding another name to this list.

Japanese big pharmas and their gene therapy work
Company and project Details
Astellas Pharma
JVS-100 (Option with Juventas) SDF1 stimulant/cardiovascular diseases
AT132 (resamirigene bilparvovec) MTM1 gene transference/Muscular dystrophy
AT342 (volrubigene ralaparvovec) UGT1A1 gene transference/Crigler-Najjar syndrome
AT845 AAV8-delivered gene therapy/Pompe disease
AT982 GAA gene transference/Pompe disease
(Plus at least six preclinical candidates)
TAK-754 (phase II)  NAV AAV8 vector/Haemophilia A 
(Plus at least five preclinical candidates)
Mitsubishi Tanabe Pharma
Haemophilia B research project  Coagulation factor IX AAV gene therapy
Santen Pharmaceutical
Retinal disease research projects Lentivector therapies under Oxford Biomedica deal
Daiichi Sankyo
Undisclosed existing research projects, and now a deal with Ultragenyx
Source: EvaluatePharma. 

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