Mimoun Azzouz is currently Professor of Translational Neuroscience and Director of Gene Therapy Innovation & Manufacturing Centre (GTIMC) at the University of Sheffield. His pioneering work, which has already produced major breakthroughs, led to real translational impact into major therapeutic advances for human neurodegenerative diseases. His team was the first to demonstrate spectacular success of SMN gene therapy in mouse models of spinal muscular atrophy. This gave confidence to develop this concept for human trials in SMA leading to new licensed therapy. The pioneering work in his team generated a body of dramatic therapeutic effects, supported by pharmocodynamic biomarkers, in pre-clinical models of both SOD1-ALS which have stimulated uptake of this concept into human trials. His work led to the first human clinical trials in Parkinson’s disease using direct brain delivery of a lentivector-mediated dopamine replacement. A successful translational story emerged from my team by generating compelling efficacy and safety data of a gene therapy for patients with HSP47. This approach received FDA clearance to enter clinical trials in patients.
He successfully attracted a unique combination of substantial funding from public, private, charity and local authority to establish the Gene Therapy Innovation and Manufacturing Centre (GTIMC), a centre which increase UK capacity to supply clinical vectors to treat human diseases, translational support and skills and training. He co-led (with Roche) the ‘Accelerating Research & Development for Advanced Therapies’ (ARDAT: www.ardat.org), a €25.5M EU IHI consortium, which includes 34 partners to accelerate the research and development of Advanced Therapy Medicinal Products (ATMPs). ARDAT is covering several medical science areas including neurodegeneration, cardiovascular, blood disorders, immunology, regulatory science.
His passion to shape and influence therapy development across the medical science community and generate impact for a wider society are demonstrated by: As scientific founder of BlackfinBio and co-founder of Crucible Therapeutics. He played important role in securing investments to progress promising therapeutics to benefit patients through commercialisation journey. Since 2006, He has supervised the research training of 81 individuals. It is widely recognised the need for different careers in ATMPs due to its rather lengthy timelines. He is making important contribution to evolve this in the UK Community.
His leadership has been recognized by several prestigious awards and prizes. He is acting as member of Panels/Boards of funding bodies, Scientific Advisory Board memberships, Evaluation Committees, keynote and plenary lectures at established international meetings/institutions. He is inventor on several patents and co-founder university start-ups companies.