So based on this nature's observation, we're simply recapitulating that same observation using CRISPR, reuse the CRISPR tool to make and edit that essentially switches the fetal hemoglobin back on. And it made up for the deficiency or defectiveness of adult hemoglobin, which are the root cause of the diseases of thalassemia or sickle cell.
Now in these populations that were studied, the switch didn't work so the fetal hemoglobin was never turned off. Within six months of being born, the body turns off the fetal hemoglobin with a switch and that is replaced by adult hemoglobin.
Fetal hemoglobin is an alternative form of hemoglobin to adult hemoglobin that we're all born with. And it turned out that through a naturally occurring mutation they had high levels of fetal hemoglobin. And the question was why are those folks asymptomatic. Over the last 40 years, leading scientists were doing population studies in the Middle East and Asia, where they found families of people where they had the genetics of sickle cell or thalassemia but were asymptomatic and normal. The approach we're using to provide a potential cure for sickle cell and beta thalassemia is based on observations in nature. So maybe let's start off with CTX001, describe how this works and basically, how you're using CRISPR, or this molecular scissors, as you mentioned, to help cure sickle cell and beta thalassemia? Yes, very powerful technology, really cool. And we have a number of indications that we pursue with in vivo approaches behind that as well. We’ve started with sickle cell and thalassaemia as our lead program and then we've expanded that into immuno oncology where we have three different CAR-Ts in the clinic and now we're expanding that into regenerative medicine in the area of diabetes. And in a very short period of time, since inception of the company, we've been able to apply this technology to develop transformative medicines across a whole host of indications. And once you make a cut in the genome, you can use the body's own mechanisms, repair mechanisms to either knock-out genes, knock-in genes, regulate genes and many different things they could do to control the genetic machinery of the body. The barcode is a genetic sequence that if it matches anywhere within the genome, the scissors will go to that location and cut the genome. But most fundamentally, the CRISPR Cas9 technology is facile, versatile and easy to apply that makes it so powerful.Īt a very high level, what we have with the CRISPR technology are pair of molecular scissors and they are attached to a barcode. And the reason for that are many folds, there's been a lot more we know about the human genome from the human genome sequencing project, there's a lot more we have in terms of tools at our disposal like delivery technologies. What's different about this revolution CRISPR Cas9 is from other discontinuities we've seen in biotech before is that the technology cycle has been much faster than what we've seen with antibodies or some other platforms. And right then there was a lot of speculation about what its impact would be in health, and what we're seeing now is that that promise is becoming a reality. Emmanuelle Charpentier and Jennifer Doudna in the 2010-2011 timeframe. Just at a 20,000 foot level, the CRISPR Cas9 technology was elucidated by Dr. But I think what you'll appreciate is, I think the first one we did too now the technology has been moving at a rapid, rapid pace. And I've always enjoyed these fireside chats. This is our fifth year doing this conference with you. Thanks, Ted again for having us at this conference. So pretty incredible Nobel Prize was already awarded to this technology, perhaps you can start off by telling us how CRISPR Cas9 works and how it can be applied therapeutically? With us from CRISPR today is my good friend.
Additionally, CRISPR has advanced a pipeline of three gene edited CAR-Ts into the clinic, and has a very rich and exciting preclinical pipeline. CRISPR has demonstrated the curative potential of CTX001 in sickle cell disease and beta thalassemia. CRISPR is the leading developer of CRISPR Cas9 therapies, building on the discovery of Emmanuelle Charpentier and Jennifer Doudna, who were both awarded the Nobel Prize in Chemistry this year. I'm a Senior Biotechnology Analyst at Piper Sandler, and welcome to our Virtual Healthcare Conference. CRISPR Therapeutics AG ( NASDAQ: CRSP) Piper Sandler 32nd Annual Virtual Healthcare Conference Decem9:30 AM ET
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