00:00Well, first of all, how it was invented? Someone had this idea that said, if viruses are so smart getting inside a cell, getting inside the nutrients of the cell, and the cause of the disease, can we use that approach to get in the cell, get in the nutrients of the cell, and create a therapy that can create a positive impact in the life of a patient.
00:29And they started with this virus that is highly prevalent in society, because AAV is very, you know, it doesn't cause any particular disease, and basically they clean all the genetic material inside, and that was a bit of the ship that was used to say, we're going to use this to get inside the cell and the glucose of the cell, and try to heal the medicine.
00:55So then, once, if you do that, what is the next thing that you do? Well, you kind of have made the mechanic that once this is in the right place, it's going to solve the problem, and that's what's called the transgene. And basically, depending on the disease, you have to choose the right transgene that is specific to do what it's supposed to do.
01:15That many times is to do what is called gene augmentation in Parkinson's program, where you add well-functioning copies of a gene. You can do gene silencing when you want to stop the activity of a gene that is causing any disease. Or what is called, and this is probably the newer phase of the science, what is called gene editing, which is identifying your gene of interest, and a bit like a live
01:45and you would place that segment that is causing the problem for a healthy copy. These are the modernities. The part of what we call the cassette of a gene therapy that does that is the transgene. So now you have the ship to go where the program is, now you have the mechanic to fix the problem. What is missing? What is missing is the captain. And the captain is called the promoter, whose role is to say, okay, if we want the therapy for
02:15brain, it will be very effective. It just goes to the brain and we have all that goes to other places. Because again, probably you create a more ineffective therapy because you're dosing patients with more product that you should if the product goes to places where you shouldn't go. Sometimes you can be causing toxicity. For example, if it goes to organs for which the therapy was not designed for, that is the job of the promoter
02:44the promoter inside the promoter. So basically the sum of the capsule, the transgene and promoter. That is correct, as said, and that, as we see in the picture, is what has the mission to get in the cell, the influence of the cell, and provoke a positive therapeutic effect in the person.
03:06Then also, gene therapy being such a new field, and this is a little bit at the heart of the strategy of Aspiro, probably for, if you start today, a traditional pharmaceutical company, you have vendors that you don't need to do your CMC because you have a company in India that can do that for you and they can do it well.
03:31You can deconstruct a lot of what you do. In the case of gene therapy, because it's such a new field, the company realized at some point is that every step of the world is a new step.
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