Agilis forms joint venture to advance gene therapy vectors

Japanese flag
Agilis will work on AAVs for use in gene therapies against AADC deficiency and Parkinson's. (Grantuking/CC BY 2.0)

Agilis Biotherapeutics has formed a joint venture with Japan’s Gene Therapy Research Institution (GTRI). The alliance gives Agilis a base in Japan and a partnership with a fellow CNS specialist to support its development of adeno-associated virus (AAV) vectors and gene therapies.

Cambridge, Massachusetts-based Agilis set up the joint venture using a grant from the Japanese government. The agreement will establish an AAV manufacturing facility in Japan, from where Agilis and GTRI will work on vectors using Sf9 baculovirus and HEK293 mammalian cell systems. Agilis and GTRI plan to develop and manufacture AAV gene therapy vectors through the joint venture.

Agilis and GTRI also plan is to collaborate on the development and commercialization of certain CNS gene therapies. 


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GTRI’s background suggests it is well-equipped to contribute to the project. The Japanese company grew out of the work of Shin-ichi Muramatsu, M.D., a scientist who sequenced AAV3 in the 1990s before going on to create AAVs designed to cross the blood-brain barrier. GTRI is working on gene therapies against diseases including Alzheimer’s, amyotrophic lateral sclerosis and Parkinson’s that build on this research into AAVs.

Both biotechs are developing gene therapies to treat aromatic l-amino acid decarboxylase (AADC) deficiency. GTRI aims to get its candidate into the clinic in 2019. Agilis picked up its candidate from a university in Taiwan, which enrolled 18 patients in two clinical trials of the gene therapy. Those trials have taken the candidate toward a pivotal trial. 

These programs may benefit from the joint venture. Working out of the Life Science Innovation Center of Kawasaki City, the joint venture intends to develop and produce AAVs for use in gene therapies against AADC deficiency and Parkinson's.   

The joint venture marks the second time Agilis has looked outside of its walls for help with AAV vectors. Late in 2013, Agilis struck a deal with Intrexon that gave it access to the latter’s vector platform. Agilis is using the vectors to develop a treatment for Friedreich’s ataxia.

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