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news.Takeda Pharmaceutical, a Japanese pharmaceutical company, will work with University College London (UCL) in a research aimed at tackling muscle disorders, in particular muscular dystrophy.
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The research group of Dr Francesco Saverio Tedesco from the UCL department of Cell and Developmental Biology is conducting the research, which is being supported through funding of $250,000 from Takeda’s New Frontier Sciences group.
Takeda’s New Frontier Sciences group aims to support research that could eventually lead to drug discovery and development.
Dr Tedesco’s team will focus on a study of muscular regeneration and the potential for stem cell therapies to treat muscular dystrophy, in particular induced pluripotent (iPS) stem cells. Potential for treating muscular dystrophy through developing novel gene and cell therapy strategies using artificial human chromosomes and novel biomaterials is also being investigated by Dr Tedesco’s team.
The team, using this approach, hopes to overcome a number of current limitations to develop effective treatments for muscular dystrophies. Through the use of the modified stem cells, it is hoped that large quantities of progenitor cells could be produced to be transplanted into a patient’s muscle following genetic correction or to be used for drug development platforms.
Dr Tedesco’s team will attempt to produce the modified stem cells, which can be applied more easily in a clinical context, 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 demonstrated the potential of pre-clinical gene replacement therapy using an artificial human chromosome.
In a separate study, Dr Tedesco’s team has demonstrated 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).
Dr Tedesco noted 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," Dr Tedesco added.