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The Future of Rare Disease Therapies


ICT: How do you see the landscape of gene therapy trials for rare diseases looking in the future?

Dr Matthew Klein: Ever since the human genome was cloned, there has been the potential for using gene therapy to target diseases, particularly rare diseases, many of which result from missing or inactive genes. Over the past few years, we have finally caught up in terms of having the necessary understanding, technology, and experience to not only discover potential gene therapies, but develop them. What we’re seeing right now, not surprisingly, is an explosive growth in the development of gene therapies targeting rare diseases because there’s always been a lot of understanding that rare diseases typically result from biological defects related to missing or inactive genes. The landscape now is chock full of efforts looking at gene therapies for rare diseases, and I think that’s going to be increasingly the case. It's really an exciting time for gene therapy, and gene therapy for rare diseases.

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Dr Matthew B Klein, MD, MS, FACS is Chief Development Officer at PTC Therapeutics. Prior to joining PTC, Matthew was CEO and Chief Medical Officer of BioElectron Technology Corporation, a biotechnology company focused on developing redox active small molecules for mitochondrial disease and related disorders of oxidative stress. Prior to joining BioElectron, he was the Auth-Washington Research Foundation Chair of Restorative Burn Surgery at the University of Washington. Dr Klein completed his undergraduate degree at the University of Pennsylvania, US, where he graduated summa cum laude and Phi Beta Kappa, and received his MD degree with honours from Yale University, US.
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