“CRISPR (clustered regularly interspaced short palindromic repeats) and the technology may be the key to curing diseases, especially genetic disease such as Sickle Cell, Down syndrome, Familial Alzheimer's and Cancer among others,” said, Trevor Martin, co-founder and CEO of Mammoth Bioscience at the recently concluded Web Summit Qatar 2025.
These are diseases where the root cause is in the DNA, Martin stated. “We are excited to commercialise this technology that we call CRISPR to change that DNA and the code of life so that we can fundamentally cure these diseases rather than treating the symptoms of them.”
Martin said there is an approved therapy on the market in the US and other countries using CRISPR to treat sickle cell disease. “CRISPR is a kind of biology's search and replace engine. If you think of your genome, your DNA, that's in every single one of your cells, you can think of it as a giant word document with two billion letters in it. And in some ways, it's a very simple word document,” he noted.
“CRISPR allows you to do a search anywhere in that document and find any specific, what we call sequence, or any specific set of letters, and then change it to anything you want. So if you think about it as this word document, maybe a disease is caused by the misspelling of a word, or maybe there's a whole paragraph missing from a chapter in your genome.
“And basically what we can do with CRISPR is we can fix that. We can insert a paragraph, we can spell check a word, or we can even delete a sentence. And that's a very powerful concept for genetic disease, he said.
According to the expert, CRISPR is something that originally evolved in nature. “Similar to how we all have immune systems, we get a virus and our body fights it off, actually microorganisms like bacteria have to fight off things like viruses as well. And one of the immune systems that they built is this technology called CRISPR.”
Martin said that CRISPR works when a virus invades a bacteria as CRISPR systems constantly monitor the cell against the viruses. “We have basically ripped that technology out of these microorganisms. This is a really great technology where we can co-opt all that machinery and use it as a technology that we can insert into human cells and use that to chop a very specific section of the DNA and make a very specific change using a guide RNA.”
He said that the guide RNA is the only thing to be changed when making CRISPR go from targeting Alzheimer's disease to targeting sickle cell disease, a very powerful concept.
“The beauty of CRISPR is that across all these thousands of genetic diseases, the core technology, the core CRISPR system stays the same, and you're just kind of switching out that search function. So you can think of it as you are always using the Google search engine, but you're just typing in a different search string into it. And that means the first therapy to build with a CRISPR technology is the hardest, and then the second one's easier, the third one's easier, the fourth one's easier,” he elaborated.
It could be the start of the move beyond curing specific diseases into prevention of disease, which is a very exciting position to be in, Martin said.
“ Over the next five or 10 years, you're going to be shocked at how many diseases start to be cured. And this is something that's been promised for a long time, but with the first approved therapy now for sickle cell using CRISPR in the United States, I think that that's the first of a dam that's about to truly break,” he added.
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