Empowered Patient Podcast

Paula Ragan is the CEO and President of X4 Pharmaceuticals, which focuses on the rare disease WHIM Syndrome. Patients have a deficiency in their immune system, and as a result, this diminishes their ability to fight chronic infections, and they have a higher risk for severe cancers.

Paula explains, "If one thinks about doing the right test, it's easy to diagnose, and the right test is doing genetic sequencing. There are commercially available panels, so it exists for the patient and physician communities. The current challenge is getting the physicians to recognize the test is needed. And then to also enable payment. X4 has done a great job in providing free testing through our PATH4WARD program."

"Our lead candidate is called mavorixafor, and it's an oral once-daily capsule. And the amazing thing is its mechanism. What it does is binds to a receptor called CXCR4. And it basically shuts it down a little bit. It's kind of blunting the signaling that normally exists in the cell. And for these patients with WHIM syndrome, their disease is literally caused by too much signaling, by profound over signaling of CXCR4. So we can give them a once-daily capsule that blunts that effect and really then restores the more normalized function of the immune system so that they can combat infections and hopefully minimize their cancer risk."

@X4Pharma #WHIMSyndrome #ChronicNeutropenia #CXCR4 #Waldenstrom #RareDisease

X4pharma.com

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Paula Ragan is the CEO and President of X4 Pharmaceuticals, which focuses on the rare disease WHIM Syndrome. Patients have a deficiency in their immune system, and as a result, this diminishes their ability to fight chronic infections, and they have a higher risk for severe cancers.

Paula explains, "If one thinks about doing the right test, it's easy to diagnose, and the right test is doing genetic sequencing. There are commercially available panels, so it exists for the patient and physician communities. The current challenge is getting the physicians to recognize the test is needed. And then to also enable payment. X4 has done a great job in providing free testing through our PATH4WARD program."

"Our lead candidate is called mavorixafor, and it's an oral once-daily capsule. And the amazing thing is its mechanism. What it does is binds to a receptor called CXCR4. And it basically shuts it down a little bit. It's kind of blunting the signaling that normally exists in the cell. And for these patients with WHIM syndrome, their disease is literally caused by too much signaling, by profound over signaling of CXCR4. So we can give them a once-daily capsule that blunts that effect and really then restores the more normalized function of the immune system so that they can combat infections and hopefully minimize their cancer risk."

@X4Pharma #WHIMSyndrome #ChronicNeutropenia #CXCR4 #Waldenstrom #RareDisease

X4pharma.com

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Dr. Luca Benatti is the CEO of EryDel, a biotech company developing and commercializing cell-therapy treatments for rare diseases using autologous red blood cell encapsulation technology to deliver drugs. With a focus on the rare genetic disease Ataxia Telangiectasia (AT), Erydel has just completed a multi-center, multi-national phase III trial for the disease. Their symptomatic treatment has been shown to delay the progression of the disease and could be part of a regular immunoglobulin therapy session for these patients.

Luca explains, "The first characteristic of our technology since there are similar technologies that utilize red blood cells to deliver therapeutics, is that we are the only one that has developed a technology that can work at the bedside. So, it's really the only one that can use the patient's blood, and we don't need to have a centralized lab. The beauty of this technology is that it is the only one that can use a sustained release formulations technology - that is, the one that is actually being developed for Ataxia Telangiectasia - on which we have completed phase III study results."

"So, from the time of blood withdrawal from the patients and for our lead product, we take 50 milliliters of blood from the patient, a small amount, and within a couple of hours, patients can go home. They are only required to stay for the blood withdrawal and the blood infusions. The rest of the process is done by a small machine that is fully automated and operated by trained personnel."

#EryDel #AtaxiaTelangiectasia #EryDex #RareDisease #AT

EryDel.com

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Dr. Luca Benatti is the CEO of EryDel, a biotech company developing and commercializing cell-therapy treatments for rare diseases using autologous red blood cell encapsulation technology to deliver drugs. With a focus on the rare genetic disease Ataxia Telangiectasia (AT), Erydel has just completed a multi-center, multi-national phase III trial for the disease. Their symptomatic treatment has been shown to delay the progression of the disease and could be part of a regular immunoglobulin therapy session for these patients.

Luca explains, "The first characteristic of our technology since there are similar technologies that utilize red blood cells to deliver therapeutics, is that we are the only one that has developed a technology that can work at the bedside. So, it's really the only one that can use the patient's blood, and we don't need to have a centralized lab. The beauty of this technology is that it is the only one that can use a sustained release formulations technology - that is, the one that is actually being developed for Ataxia Telangiectasia - on which we have completed phase III study results."

"So, from the time of blood withdrawal from the patients and for our lead product, we take 50 milliliters of blood from the patient, a small amount, and within a couple of hours, patients can go home. They are only required to stay for the blood withdrawal and the blood infusions. The rest of the process is done by a small machine that is fully automated and operated by trained personnel."

#EryDel #AtaxiaTelangiectasia #EryDex #RareDisease #AT

EryDel.com

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Eliav Shaked, CEO, and Catherine Bornbaum, Head of Clinical Operations and Partnerships at RetiSpec are using hyperspectral retinal imaging to find more accurate and earlier biomarkers of neurodegenerative diseases. As part of a regular eye exam, this non-invasive test can be administered by an ophthalmologist or optometrist using existing retinal cameras with the data being analyzed using AI to identify unique molecule signatures.

Eliav explains, "What's unique about hyperspectral imaging or specifically hyperspectral retinal imaging is the fact that it's a tool that measures different coloring of elements in a much more detailed way. So, while we humans are limited to seeing and interpreting only three colors, hyperspectral retinal imaging can cover more than a hundred different colors in a very precise way. And it's really our way to get much more content from the back of the eye. And what RetiSpec is doing with that data is we're integrating our very own artificial intelligence that allows us to recognize patterns that no human eye can see."

Catherine elaborates, "We are focused right now on identifying the markers for Alzheimer's disease through this hyperspectral retinal imaging. But because of the close connection between the retina and the brain, we believe that there are also a number of other indications and specifically neurologic conditions that can be detected using this approach for things like ALS, MS, Parkinson's disease, CTE, and a number of conditions."

@RetiSpec @EliavShaked @CathBornbaum #RetiSpec #EarlyAlzheimersDetection #AlzheimersDisease #RetinalScans #HyperspectralImaging

RetiSpec.com

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Eliav Shaked, CEO, and Catherine Bornbaum, Head of Clinical Operations and Partnerships at RetiSpec are using hyperspectral retinal imaging to find more accurate and earlier biomarkers of neurodegenerative diseases. As part of a regular eye exam, this non-invasive test can be administered by an ophthalmologist or optometrist using existing retinal cameras with the data being analyzed using AI to identify unique molecule signatures.

Eliav explains, "What's unique about hyperspectral imaging or specifically hyperspectral retinal imaging is the fact that it's a tool that measures different coloring of elements in a much more detailed way. So, while we humans are limited to seeing and interpreting only three colors, hyperspectral retinal imaging can cover more than a hundred different colors in a very precise way. And it's really our way to get much more content from the back of the eye. And what RetiSpec is doing with that data is we're integrating our very own artificial intelligence that allows us to recognize patterns that no human eye can see."

Catherine elaborates, "We are focused right now on identifying the markers for Alzheimer's disease through this hyperspectral retinal imaging. But because of the close connection between the retina and the brain, we believe that there are also a number of other indications and specifically neurologic conditions that can be detected using this approach for things like ALS, MS, Parkinson's disease, CTE, and a number of conditions."

@RetiSpec @EliavShaked @CathBornbaum #RetiSpec #EarlyAlzheimersDetection #AlzheimersDisease #RetinalScans #HyperspectralImaging

RetiSpec.com

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Dr. Sam Asgarian is the Chief Medical Officer of Scipher Medicine which is predicting drug response with precision immunology by building on the work of the Human Genome Project. Now that the human genome has been mapped, the next challenge is how to turn that information into use cases that are applicable in treating specific diseases.

Sam explains, "The goal for us is to figure out which part, very specifically, of the autoimmune system is overacting and analyze that through a simple blood sample. We can take the blood in just a simple lab draw, just like other blood work, and analyze areas of perturbation or areas where the immune system specifically is overacting. That gives the physician enough to then treat it with a prescription that focuses only on that specific area which, of course, in the immune system is critically important because it's still serving a very important goal of protecting that person against disease. You don't want to shut down the overall immune system. You just want to quiet the area that's leading to that rheumatoid arthritis diagnosis."

"I think in this early onset of what we have out there, our goal is leading with the diagnostics, giving the physician a treatment selection or therapy selection tests to get them out of trial and error and into this world of precision immunology."

@ScipherMedicine #ScipherMedicine #PrecisionImmunology #PrismRA #Diagnostics #RheumatoidArthritis #AutoimmuneDiseases #AutoimmuneDisorders #IrritableBowelDiseases #HumanGenomeProject

ScipherMedicine.com

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Dr. Sam Asgarian is the Chief Medical Officer of Scipher Medicine which is predicting drug response with precision immunology by building on the work of the Human Genome Project. Now that the human genome has been mapped, the next challenge is how to turn that information into use cases that are applicable in treating specific diseases.

Sam explains, "The goal for us is to figure out which part, very specifically, of the autoimmune system is overacting and analyze that through a simple blood sample. We can take the blood in just a simple lab draw, just like other blood work, and analyze areas of perturbation or areas where the immune system specifically is overacting. That gives the physician enough to then treat it with a prescription that focuses only on that specific area which, of course, in the immune system is critically important because it's still serving a very important goal of protecting that person against disease. You don't want to shut down the overall immune system. You just want to quiet the area that's leading to that rheumatoid arthritis diagnosis."

"I think in this early onset of what we have out there, our goal is leading with the diagnostics, giving the physician a treatment selection or therapy selection tests to get them out of trial and error and into this world of precision immunology."

@ScipherMedicine #ScipherMedicine #PrecisionImmunology #PrismRA #Diagnostics #RheumatoidArthritis #AutoimmuneDiseases #AutoimmuneDisorders #IrritableBowelDiseases #HumanGenomeProject

ScipherMedicine.com

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Sunny Jain is the Founder and CEO of Floré by Sun Genomics and is driven to better understand the microbe cells that live inside of our intestinal tract and all around our body. With a history of working at Illumina, Sunny is studying the DNA from those cells, the microbiome, to understand their function and how they contribute to our overall health.

Sunny explains, "Sun Genomics has created the world's only precision probiotic, And it utilizes AI, DNA sequencing, biome chromatics, and e-health technology. Our technology helps us build the largest database of microbiomes that are sequenced via metagenomics, or whole genome sequencing, to understand the modulatory effects or the changing effects that food ingredients and probiotics can have for 80 plus million Americans that suffer from gut health issues every year."

"So, you truly are unique, from a gut microbiome perspective. Our human DNA is 99.9 percent similar. So, we're very identical from a human DNA perspective. But the DNA from microbes of the gut, you're maybe 10 percent similar, and you are 90 percent unique from the gut microbiome."

"And this is really where I think precision medicine can enable a lot of advancement to our human health. It is really within that large, vast difference where we will be able to find much more customized solutions."

@SunGenomics #SunGenomics #GutHealth #Probiotics #Microflora #CustomizedProbiotics #Microbiome #SanDiego

Flore.com

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Sunny Jain is the Founder and CEO of Floré by Sun Genomics and is driven to better understand the microbe cells that live inside of our intestinal tract and all around our body. With a history of working at Illumina, Sunny is studying the DNA from those cells, the microbiome, to understand their function and how they contribute to our overall health.

Sunny explains, "Sun Genomics has created the world's only precision probiotic, And it utilizes AI, DNA sequencing, biome chromatics, and e-health technology. Our technology helps us build the largest database of microbiomes that are sequenced via metagenomics, or whole genome sequencing, to understand the modulatory effects or the changing effects that food ingredients and probiotics can have for 80 plus million Americans that suffer from gut health issues every year."

"So, you truly are unique, from a gut microbiome perspective. Our human DNA is 99.9 percent similar. So, we're very identical from a human DNA perspective. But the DNA from microbes of the gut, you're maybe 10 percent similar, and you are 90 percent unique from the gut microbiome."

"And this is really where I think precision medicine can enable a lot of advancement to our human health. It is really within that large, vast difference where we will be able to find much more customized solutions."

@SunGenomics #SunGenomics #GutHealth #Probiotics #Microflora #CustomizedProbiotics #Microbiome #SanDiego

Flore.com

Download the transcript here