The Princess Royal visited the Gene Therapy Innovation and Manufacturing Centre at the University of Sheffield in October for the official opening of the site which will help so many families.
The Star spoke with Sheffield University's Holly Garlick and gene therapy panelist Dharmisha Stezaly about the centre and the Royal visit.
The Star spoke with Sheffield University's Holly Garlick and gene therapy panelist Dharmisha Stezaly about the centre and the Royal visit.
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00:00We've got a new manufacturing centre here, specifically for gene therapies and then specifically beyond that AAB.
00:07That is designed that we can take the wonderful research that the academic groups do
00:13and translate that into a clinical product for patients.
00:16That's the ultimate aim at the end of the day.
00:19There's a few stages to get there.
00:21We've got pre-clinical work and testing and proving that that process works and that it's safe for a patient.
00:27I've been in one of the panels today that has received a gene therapy.
00:32My son was diagnosed with spinal muscle atrophy, type 1.
00:38He received gene therapy at about four and a half, five months.
00:43It's changed our lives forever, really, because the natural history of the disease is bleak.
00:52Without any treatment, he dies before the age of two.
00:55Every single year, he lives past that natural history.
01:02With the gene therapy as well, we don't really know what he's going to be able to do,
01:08but based on what he's doing now, he's eating independently, he's breathing independently,
01:12he's chatty, he's happy, he's going to a mainstream nursery.
01:18He's just living life like an average three and a half year old.
01:24You go from taking each day at a time, and living life like that is hard,
01:31because if you can't plan, it's just challenging.
01:35There are lots of families that do have to take each day in turn.
01:38We're able to make plans, and I know that sounds a bit vague,
01:44but being able to plan a future, and being able to say,
01:50based on what Sebastian's doing now, we can plan to do this or that,
01:55and not thinking about him regressing.
02:01It was really exciting today.
02:03We've got a full team now, we've got 25 people on site,
02:08and it's taken quite a long time for us to get here.
02:11There's a lot of hard work, lots of late nights,
02:14and stress and strain on people to get to this point,
02:17so it's really nice to show off how complete we are now,
02:21and also to celebrate with the funders that we've had on site today,
02:25the patients, hopefully we'll be treating more patients like that in the future.
02:29It was lovely. You don't know what to expect,
02:32but she's very down to earth, very light-friendly,
02:37she seems to be really interested in this.
02:40She doesn't care about my son receiving this or Jennifer,
02:44so it's nice, very nice, yeah.