Robert Benkowski Presents Opsin Biotherapeutics at LSI USA '24

Robert Benkowski  0:02  
Bob, hello. My name is Bob Benkowski, CEO of opsin Biotherapeutics, where we're lighting the path to pain relief with optogenetics. So do you know somebody who's in chronic pain? Let's see a show of hands. If you know anybody that's in chronic pain, it's quite a debilitating disease, and it's truly an epidemic. In the United States, you can see the economic impact, and there's more than 30% of people in America that have some form of chronic pain or severe pain. So what do these patients do that have chronic pain? There's really two pathways. There's pharmaceuticals, opioids, and you all know the side effects of opioids and over the counter medication, those all have a systemic response. There's also devices, specifically spinal cord stimulators, used for chronic pain. The problem with spinal cord stimulators is the electrical pulses affect the neurons that both block pain and transmit pain. One of the patients that had an electrical spinal cord stimulator, she said, every time it turns on, I feel like I'm getting shocked. And the truth is, she is getting shocked. That's how they work, and they modulate frequency, voltage, current, pulse pattern, so they try to get the neurons that block pain to react more than the neurons that transmit pain. Here's a few quotes. With patients that have chronic pain, it totally affects their life. They can lose their job, they lose their ability to sleep. Well, they can lose their appetite. It's truly a debilitating disease. And of course, there must be a much better solution, which brings us to optogenetics. This is our solution, optogenetic neuromodulation. Let me break that down for you. We use a multi characteristic opsin, which is a photoreceptor. We take those photoreceptors and we put them on your spine, and we can modulate those photoreceptors using light. And it sounds like science fiction, but that's how your eyes work. So it's basically taking the same principles in your eyes and putting it in your spine. How do we do this? We use functionalized gold nanorods mixed with the plasma injected in your spine, and we use low power laser light to heat those functionalized gold nanorods which allow the DNA to go into the neurons. The plasmid and the multi characteristic opsin has a promoter, so the opsins are only expressed on the neurons that block pain. They're not expressed on the neurons that transmit pain. Transmit pain, which gives us incredible specificity for controlling only the neurons that can block pain. We don't edit the human genome. And if you think about it, these opsins that are on the surface of the neuron, they're like little tiny photocells, little tiny solar cells. And when enough of them pop up after about 24 to 48 hours, then we can use a low powered LED light to turn those neurons off. And again, like I said, because of the promoter, they only affect the neurons that block pain. We've done a whole series of preclinical and vivo studies. On the left, in the purple, you can see this is a normal mouse their pain tolerance. The middle bar is a mouse that's in chronic pain. And in the right is a mouse. Is the series of mice in chronic pain, but with our therapy applied, and there's no statistical difference between a pain, a mouse in chronic pain, with our therapy and a normal mouse. We've now started our preclinical translational work to go to a larger species and develop the tools for the surgical approach for humans. We also looked at the negative signal. Safety is a big issue with any type of invasive therapy in the spine. We looked at toxicity of the cells. We looked at the immune response, and we also looked at the inflammatory response, and saw no negative safety signals. Of course, the market opportunity for chronic pain is huge. Our initial indication is going to be targeting those patients that would be indicated for electrical spinal cord stimulation. We believe this is a great path, because you can see it's already a $2 billion market, already being spent on the therapy, and our approach is going to replicate exactly what's being done for electrical spinal cord stimulators. So we believe adoption will be quite, quite easy. Myself and Dr Mohanty are co founders of. The company. My background is in classroom medical devices. Dr mohanty's background is in gene therapy. So this is a combination product. Dr Narcisse, he's our principal investigator, and Mike Patterson is my former colleague. We worked on a biologic company had a nice exit about 12 years ago. If you're in the electrical spinal cord stimulation market, or if you're in the gene therapy market, you probably recognize some of these people. They're absolutely experts in their fields, and they're on our advisory board. Of course, we have competitors. We have all of the big device competitors that do electrical spinal cord stimulation. We also have competitors on the pharmaceutical side. We believe we take the best of both of those type of therapies with our optogenetic neuromodulation and our and are going to provide a superior efficacy for our patients with reduced side effects. Here's our timeline. Our sister company, nanoscope technologies, they also have a company that uses the same multi characteristic opsin for blindness. Remember, I said this therapy is how your eyes work. It was natural for them to use it for blindness. Of course, our indication that we've licensed this for the spinal column, which is why we have to have a device to put the light in. We don't have the light that comes in naturally. We've been funding this ourselves for several years. We've raised a family and friends round. We received the NIH phase one grant, and we'll be raising a Series A round later this year, we have strong intellectual property. Of the four patents we've licensed to have already been granted. We've also applied for two additional patents to strengthen our field, specifically for pain, as well as the delivery tools to deliver this gene therapy in the spine. So in conclusion, we are very promising and vivo results the multi characteristic ops and efficacy and safety has been demonstrated by our sister company in their indication for blindness. They'll be filing a bla later this year. We don't require opioid or other types of pharmaceuticals, and it provides a solution where all other therapies have failed, specifically a solution against opioids and the electrical spinal cord stimulators. And like I said, we're going to use a well established surgical technique to apply this therapy. And there's my contact information on the bottom. Please reach out to me by email or LinkedIn, and thank you for your time. Applause.