Genetic therapies are becoming a remarkable avenue for potentially slowing and preventing neurodegenerative diseases. Since the groundbreaking results from IONIS-HTTRx (RG6042) for Huntington’s disease were announced, researchers across the globe have been working to translate gene-based approaches to other neurodegenerative diseases such as Alzheimer’s. In particular, the Neurogenetic Therapies Programme at University College London (UK) aims to facilitate drug discovery right from pre-clinical work in genetic therapies to first-in-man clinical trials.
We recently spoke with Catherine Mummery, who is a Consultant Neurologist at the National Hospital for Neurology and Neurosurgery (London, UK), at the Wolfson Closing Symposium (19 June 2019, University College London) to find out more about the program. In addition to this, Catherine also spoke about how she led a first-in-human, gene-silencing treatment for Alzheimer’s disease and what the core challenges are in bringing gene-silencing treatments to the clinic.
00:05 – Please could you introduce yourself and tell us about your current research focuses?
00:23 – Can you tell us a little about the Neurogenetic Therapies Programme for neurodegenerative diseases? What promising new and emerging therapies are being developed?
01:08 – You led a first-in-human study on Alzheimer’s disease using a gene-silencing treatment to reduce the levels of tau protein – can you tell us more about this?
02:33 – What would you say are the core challenges in bringing gene-silencing treatments to the clinic for Alzheimer’s disease? How can these challenges be addressed?
03:38 – Lastly, where do you see the field progressing in the next 10 years?
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