Since the groundbreaking results from IONIS-HTTRx (RG6042) for Huntington’s disease, genetic therapies for neurodegenerative diseases have become a remarkable avenue for potentially slowing and preventing disease. As more genetic therapies are surfacing in the field, we wanted to find out what the challenges are surrounding clinical trials for these therapies and what technological advancements could help bring them to the clinic sooner.
At the Wolfson Closing Symposium (19 June 2019, University College London, UK), we spoke with Sarah Tabrizi, who led the pioneering study of IONIS-HTTRx, to find out the answers to these questions. In addition to this, Sarah also provided her thoughts on where the field might be progressing and what we might hope to see in 10 years’ time.
Interview questions on genetic therapies for neurodegenerative diseases:
01:01 – How far has the development of genetic therapies come in the last few years?
01:55 – What are the challenges surrounding clinical trial design for genetic therapies when it comes to neurodegenerative diseases?
03:32 – In your opinion, how might these challenges be overcome?
04:35 – What technological advancements would you most like to see brought into the field to help bring these therapies to the clinic more quickly?
05:14 – Looking at IONIS-HTTRx, the Phase I/IIa trial has demonstrated safety and tolerability in patients with early-stage Huntington’s disease. Can you tell us about the next steps for this?
07:35 – Lastly, where do you see the field progressing in the next 10 years?
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