Takeda and UCL launch muscle dystrophy alliance

Takeda and University College London (UCL) are to collaborate on research into tackling muscle disorders, in particular muscular dystrophy.

The research will be led by the university’s Dr Francesco Saverio Tedesco and will be supported through funding of $250,000 from Takeda’s New Frontier Sciences group.

The Japanese pharma firm’s NFS aims to support innovative research which could eventually lead to drug discovery and development.

Dr Tedesco’s team will focus on the study of muscular regeneration and the potential for stem cell therapies to treat muscular dystrophy, in particular induced pluripotent (iPS) stem cells.

The team will also investigate novel gene and cell therapy based on artificial human chromosomes and novel biomaterials.

Dr Tedesco says he hopes to overcome a number of current limitations to developing effective treatments for muscular dystrophies. By using these modified stem cells, large quantities of progenitor cells could be produced to be transplanted into a patient’s muscle following genetic correction or used for drug development platforms.

Importantly, the team will attempt to produce these cells which can be applied more easily in a clinical context, in order to reduce the hurdles that might limit their possible future use in clinical studies.

Through previous work using a mouse model of Duchenne muscular dystrophy, the team has already demonstrated the potential of pre-clinical gene replacement therapy using an artificial human chromosome.

Dr Tedesco and his team have also shown the potential of genetically corrected iPS cells which had been transplanted into another mouse model of a genetic muscle disorder (limb-girdle muscular dystrophy 2D).

Commenting on the new funding for his research, Dr Tedesco, from the UCL Department of Cell and Developmental Biology, said: “This funding from Takeda comes at a crucial time for building on our research to develop new and innovative approaches to developing potentially life-changing treatments for muscular dystrophies.

“With Takeda’s backing, my team and I will be building on the processes we’ve already shown to be promising and which could pave the way for the development of novel strategies for both cell therapies and drug discovery in muscle disorders.”

Gordon Wong, D.Phil., Head of New Frontier Science at Takeda, added: “We are delighted to support Dr Tedesco and his team because their ground breaking work has the potential for significant patient benefit.”

He concluded:”That several different strands of their research have already borne fruit was strong evidence for us of the translational potential of Dr Tedesco’s research for muscular dystrophies.”

Links

Summit enters biomarker collaboration for rare disease, Duchenne Muscular Dystrophy

 

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