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Dr Martin Donnelley from the Robinson Research Institute attended the American Society of Gene and Cell Therapy Conference and Gene Editing workshop in Chicago, USA in May 2018.

This is what Martin had to say about his experience in the USA.

What was a highlight of the travel?

Meeting a range of people in the gene therapy field that I have been wanting to for a very long time. Also some of the technology presented at the Gene Editing workshop is particularly exciting, despite being many years from clinical application.

Did you meet any researchers or collaborators of significance? Why are they important to your work?

I met a range of people. Some examples include:

• Uta Griesenbach,ÌýSteve Hyde,ÌýDeborah Gill (UK CF Gene Therapy Consortium): Discussed the progress on the UK CF GT human trial. Further delays make it likely that the first treatment will not be until 2021.
• Steven Howe (UCL and GSK): Discussed lentiviral gene vector production methods, including bioreactor production (LV-MAX, etc). We will arrange further talks via Zoom to link my PhD student and other lab members to Steve.
• Maria Limberis (UPenn): Discussed her gene therapy program, developments in the flu vaccine field, and others
• Patrick SinnÌý²¹²Ô»åÌýAshley Cooney (University of Iowa): Had lunch and discussions with Ashley about CF pig breeding, and studies in Iowa. Ashley has suggested coming to Iowa next time I’m in the USA to see their facilities and meet their team.
• Sven Kili (Head of GSK Rare diseases unit): Had lunch with Sven. GSK have closed the rare diseases unit, but Sven was able to give insight into what is required to get a product to clinical trials. He verified that IP protection of everything is important.
• Oxford Biomedica: Met a group from Oxford Biomedica about LV production and animal studies. They are particularly interested in our animal model (i.e. CF rat) capabilities, and depending on the direction that the company heads in they could be interested in collaboration.

How will the experience support you and your research going forward?

New collaboration, new connections, new knowledge about what we need to do to translate our research towards clinical trials.

What was the most exciting thing you learned/experienced whilst traveling?

The excitement about gene therapy. After 20+ years the promise has turned into reality – for eye disease there is a prescription drug called Luxturna now being used in 2018 that is a gene therapy. Another one for Spinal Muscular Atrophy (floppy baby syndrome has 14 out of 15 babies cured (they normally die in about 2 years). Another genetic cell therapy for paediatric cancer. And several more in the pipeline. Investment in the USA has taken off, there has been a leap in startups and acquisitions in the field. In reality, gene therapy is now a medicine, and that means the field is ripe for development to boost development in CF as well. The field does still acknowledge that CF is going to be one of the harder genetic diseases to treat, but slow and continuing progress is being made by us and others.