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.
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