Date: 2014-03-10
Type of information: R&D agreement
Compound: gene therapy, cell therapy, stem cell therapy
Company: Takeda Pharmaceutical (Japan) University College London (UCL) (UK)
Therapeutic area: Rare diseases - Neuromuscular diseases
Type agreement: R&D
Action mechanism: gene therapy, cell therapy
Disease: muscular distrophy, other muscle diseases
Details: * On March 10, 2014, Takeda Pharmaceutical and University College London (UCL) have announced that they will work together to drive research into tackling muscle disorders, in particular muscular dystrophy. The research is being conducted by the research group of Dr Francesco Saverio Tedesco. This work is being supported through funding of $250,000 from Takeda’s New Frontier Sciences group. Takeda’s NFS aims to support innovative, cutting-edge 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 is also investigating the potential for treating muscular dystrophy through developing novel gene and cell therapy strategies using artificial human chromosomes and novel biomaterials. Using this approach, Dr Tedesco hopes to overcome a number of current limitations to developing effective treatments for muscular dystrophies. It is hoped that through the use of these modified stem cells, 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.
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. Moreover, in a separate study, Dr Tedesco and his team also 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).
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