We do have some preliminary results, so it’s not really super preliminary because we validated this technology in vitro, what we call in vitro, meaning on cells, even on the organoids and 2D systems, which is cultured neurons, expressing specifically APOE4. So we tested this in this setting. So, for example, we have an organoid system and 2D system, neuronal system, which expresses specifically APOE4, and we have other neuronal systems expressing APOE2 and APOE3 as a control...
We do have some preliminary results, so it’s not really super preliminary because we validated this technology in vitro, what we call in vitro, meaning on cells, even on the organoids and 2D systems, which is cultured neurons, expressing specifically APOE4. So we tested this in this setting. So, for example, we have an organoid system and 2D system, neuronal system, which expresses specifically APOE4, and we have other neuronal systems expressing APOE2 and APOE3 as a control. So we were able to demonstrate very specific, accurate, and efficient downregulation only in the culture, which genotype carrying APOE4 allele only. And we didn’t see any effect on APOE2 culture or organoids expressing APOE3. So next step, we went ahead and we tested the system in animal settings. So we injected a virus which is based on what is called an adeno-associated virus, which is considered to be the gold standard platform for gene therapy applications. Most recently, the AAV system has been approved by the FDA for the treatment of hearing loss, for example. The drug named Otarmeni has been approved in 2026, several months ago. But this is just one out of nine systems, nine drugs approved by the FDA and other regulatory agencies. So it’s become a reality. It’s really gene therapy that works. And this is based on this virus called, again, an adeno-associated vector. So this virus, if we take this virus and we package the content of, as I mentioned, a Cas9, MECP2 crab repressor system inside this virus, alongside other components needed for CRISPR-Cas to act. And we can do that. We proved that we can take this virus and inject it into a rodent, which we did. So we saw a really nice level of downregulation of APOE4 in this system using this animal system. So this is not really preliminary, so this is still considered to be sort of pre-clinical studies, but we’re very confident, we’re very confident that we can take this next step, which will be a clinical study, because we have very exciting results, which, by the way, were published in a very good journal called Nature Communications in 2024, and all our systems which we develop are available for research communities through distribution in we deposited into a repository called Addgene. So all these tools are available, they are in high demand. People really get them specifically from us or from Addgene, and it’s sort of indirect proof validation in different labs, which is very encouraging for us to see our tools being used by other investigators and they’ve been proven to be very efficient. Unfortunately, it’s not only science. I wish it would be only science, which is good science, would lead to translational research and application related to clinical trials. We need money, we need funds, that’s how science works and works, we work very hard through our affiliation with Florida State University Institute of Pediatric Rare Disease, my primary appointment, and my second appointment at Duke University School of Medicine. So, utilizing this federal grant, trying to get federal grants, we got some federal grants to move these programs for clinical trials, and also outside of academia, as we patented this technology and we licensed this technology out to our company called CLAIRIgene. CLAIRIgene is a small biotech company which tries also to move this technology to clinical trial, and we’re trying just to get funds and resources, and very hopefully, we will be able to, you know, take this exciting preliminary results and translate it to clinical trials to patient technology is ready, technology is polished, just need, you know, figure out logistics and mostly funding to move this technology and make it available to patients.
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