Empowered Patient Podcast

Dr. Matt Goldstein, CEO of jscreen, focuses on the importance and power of preventive genetic testing for reproductive carrier screening and hereditary cancer screening. All genetic tests include analysis to inform specific medical management plans and provide genetic counselors to explore options. Of particular interest is testing for the gene mutations that cause Tay-Sachs disease and helping families take proactive steps to effectively eliminate the disease in future generations.

Matt explains, "So, jscreen is a nonprofit that focuses on providing access to education and high-touch support to individuals, families, and to clinicians in the area of preventive genetics and more specifically for reproductive carrier screening and hereditary cancer screening.

"These types of tests actually have been around for a really long time with the advancement in genomic technology and our knowledge generally of genetics. The capabilities we have around these panels—what we can test for and what we can do with them—have grown tremendously over the last two decades. And so, what we're offering now is completely different than what you may have been offered five or 10 years ago."

"What I will say is I think that this technology is the most powerful medical intervention that we have. Maybe second to vaccines, which we know obviously can have profound impacts on saving lives, preventive genetics, the ability to screen an individual or a couple or a family, and identify diseases that they may be at risk for, with things that you can do to prevent those diseases from happening or catch them early. That's the holy grail. That's the most amazing thing: being able to intervene."

#jscreen #GeneticTesting #TaySachsDisease #ReproductiveCarrierScreening #HereditaryCancerScreening

jscreen.org

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Dr. Matt Goldstein, CEO of jscreen, focuses on the importance and power of preventive genetic testing for reproductive carrier screening and hereditary cancer screening. All genetic tests include analysis to inform specific medical management plans and provide genetic counselors to explore options. Of particular interest is testing for the gene mutations that cause Tay-Sachs disease and helping families take proactive steps to effectively eliminate the disease in future generations.

Matt explains, "So, jscreen is a nonprofit that focuses on providing access to education and high-touch support to individuals, families, and to clinicians in the area of preventive genetics and more specifically for reproductive carrier screening and hereditary cancer screening.

"These types of tests actually have been around for a really long time with the advancement in genomic technology and our knowledge generally of genetics. The capabilities we have around these panels—what we can test for and what we can do with them—have grown tremendously over the last two decades. And so, what we're offering now is completely different than what you may have been offered five or 10 years ago."

"What I will say is I think that this technology is the most powerful medical intervention that we have. Maybe second to vaccines, which we know obviously can have profound impacts on saving lives, preventive genetics, the ability to screen an individual or a couple or a family, and identify diseases that they may be at risk for, with things that you can do to prevent those diseases from happening or catch them early. That's the holy grail. That's the most amazing thing: being able to intervene."

#jscreen #GeneticTesting #TaySachsDisease #ReproductiveCarrierScreening #HereditaryCancerScreening

jscreen.org

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Dr. Yuri Maricich, Chief Medical Officer at CAMP4 Therapeutics, describes regulatory RNA, a new area of biology that recognizes the role of Reg RNA in the production of proteins from specific genes. This technology is well-suited for haploinsufficient diseases such as SYNGAP1-related disorders, in which there is a lack of healthy protein and both parents carry a copy of the mutated gene. The goal is to create disease-modifying treatments that correct the underlying genetic cause rather than treating the symptoms.

Yuri explains, "What was really unique about CAMP4's scientific approach is that we're focused on a whole new and emerging area of biology called regulatory RNA. And these are control elements for the expression of genes. In other words, how much protein we get from a particular gene. And there's been a lot of work in the past on how to have less protein made, particularly if it's a protein that has a mutation that causes a problem. But in medicine, there have been very few opportunities to actually increase the amount of protein, but there are many diseases that need more healthy protein."

"The backdrop of CAMP4 is that there was work done just over eight years ago at the Whitehead Institute at the Massachusetts Institute of Technology in a lab by Rick Young, and he was working with a colleague at Boston Children's Hospital, Lenson, and they noticed that there was this group of so-called regRNAs. These were non-coding regions that historically have been really ignored. And as they looked and explored their function further, what they discovered was that, in fact, these regRNA elements play a critical role in controlling how much protein is produced. And so the story of CAMP4 has been to continue to understand and map different cell lines so that we could take a particular target gene and, by using tools or established medicines, for example, like antisense oligonucleotides, we could actually increase the amount of a gene's protein back up to normal."

#CAMP4 #CAMP4Therapeutics #SYNGAP1 #CuresSYNGAP1 #regRNA #RegulatoryRNA

camp4tx.com

Listen to the podcast here

Dr. Yuri Maricich, Chief Medical Officer at CAMP4 Therapeutics, describes regulatory RNA, a new area of biology that recognizes the role of Reg RNA in the production of proteins from specific genes. This technology is well-suited for haploinsufficient diseases such as SYNGAP1-related disorders, in which there is a lack of healthy protein and both parents carry a copy of the mutated gene. The goal is to create disease-modifying treatments that correct the underlying genetic cause rather than treating the symptoms.

Yuri explains, "What was really unique about CAMP4's scientific approach is that we're focused on a whole new and emerging area of biology called regulatory RNA. And these are control elements for the expression of genes. In other words, how much protein we get from a particular gene. And there's been a lot of work in the past on how to have less protein made, particularly if it's a protein that has a mutation that causes a problem. But in medicine, there have been very few opportunities to actually increase the amount of protein, but there are many diseases that need more healthy protein."

"The backdrop of CAMP4 is that there was work done just over eight years ago at the Whitehead Institute at the Massachusetts Institute of Technology in a lab by Rick Young, and he was working with a colleague at Boston Children's Hospital, Lenson, and they noticed that there was this group of so-called regRNAs. These were non-coding regions that historically have been really ignored. And as they looked and explored their function further, what they discovered was that, in fact, these regRNA elements play a critical role in controlling how much protein is produced. And so the story of CAMP4 has been to continue to understand and map different cell lines so that we could take a particular target gene and, by using tools or established medicines, for example, like antisense oligonucleotides, we could actually increase the amount of a gene's protein back up to normal."

#CAMP4 #CAMP4Therapeutics #SYNGAP1 #CuresSYNGAP1 #regRNA #RegulatoryRNA

camp4tx.com

Download the transcript here

Phil L'Huillier, CEO of Scancell, has developed an off-the-shelf DNA immunotherapy designed to generate a potent and durable immune response against advanced melanoma in patients who are unresponsive to current therapies. Their lead candidate showed significant benefit when added to standard of care checkpoint inhibitors, improving duration of response without adding side effects or toxicity. Patient selection in future trials will use a blood test to identify the immune types that can be expected to respond best to the therapy.

Phil explains, "Perhaps first and foremost, Scancell is a clinical-stage company developing novel active immunotherapies for patients. And our objective for patients is to pick up the patients that are unresponsive to current therapies or that respond for a short period of time to improve the overall survival through developing therapies that give a good duration of response, potent immune responses, but are also safe for patients."

"Perhaps before I share results from the studies, I should step back a little bit and just tell you about the platform and the product that the data has arisen for. At Scancell, we're developing these off-the-shelf. The data that we're about to talk about comes from our lead program, which is an off-the-shelf DNA immunotherapy called Immunobody, that's the name that we use for it. And it's different from the personalized therapies, the personalized vaccines that require an individual to give a tumor sample. And then there's sequencing and manufacturing for the individual there. This is off the shelf."

#Scancell #Immunobody #Immunotherapy #Cancer #Oncology #Melanoma #Biotech

scancell.co.uk

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Phil L'Huillier, CEO of Scancell, has developed an off-the-shelf DNA immunotherapy designed to generate a potent and durable immune response against advanced melanoma in patients who are unresponsive to current therapies. Their lead candidate showed significant benefit when added to standard of care checkpoint inhibitors, improving duration of response without adding side effects or toxicity. Patient selection in future trials will use a blood test to identify the immune types that can be expected to respond best to the therapy.

Phil explains, "Perhaps first and foremost, Scancell is a clinical-stage company developing novel active immunotherapies for patients. And our objective for patients is to pick up the patients that are unresponsive to current therapies or that respond for a short period of time to improve the overall survival through developing therapies that give a good duration of response, potent immune responses, but are also safe for patients."

"Perhaps before I share results from the studies, I should step back a little bit and just tell you about the platform and the product that the data has arisen for. At Scancell, we're developing these off-the-shelf. The data that we're about to talk about comes from our lead program, which is an off-the-shelf DNA immunotherapy called Immunobody, that's the name that we use for it. And it's different from the personalized therapies, the personalized vaccines that require an individual to give a tumor sample. And then there's sequencing and manufacturing for the individual there. This is off the shelf."

#Scancell #Immunobody #Immunotherapy #Cancer #Oncology #Melanoma #Biotech

scancell.co.uk

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Dr. Costas Karatzas, CEO, Director, and Co-Founder of Acesis BioMed, is focused on developing a novel treatment for low testosterone in men, an oral peptide that stimulates the body to restore its own natural testosterone production. The therapy's mechanism differs from current testosterone replacement therapies and avoids the side effect of infertility seen in younger men using traditional TRT, which shuts down natural hormone and sperm production. This low T condition is not just a function of aging but is increasingly affecting younger men due to lifestyle, environmental factors, and co-morbidities like diabetes and obesity.

Costas explains, "Our mission is to revolutionize the treatment of low T, or male hypogonadism, and related comorbidities. Although we remain laser-focused on going in phase one with a low T application, because low testosterone is involved in many other diseases like type 2 diabetes, fatty liver disease, obesity, etc., we're also going to be co-developing those with partnerships initially with academia and later on with other pharma companies. So this is, in a nutshell, the short story about Acesis. The differentiation being everything else marketed provides the body with exogenous testosterone, whereas in our case, we teach the body to go back to its old memory and generate or restore its own testosterone in a natural way."

"Men can develop low testosterone because of the aging process, but this is a wrong idea. Low testosterone is not just an aging problem. It's true that we as men lose testosterone from the age of 30, about 1-1.5%, which is circulating in our blood. By the time we reach sixties or seventies, we could have probably 50% to 60% of the testosterone levels we had while we were at the age of 30 years old. Globally, it's estimated that about 40% of adult men may have suboptimal testosterone levels, especially if you include men with other health issues like diabetes, and we mentioned obesity. So, for older men, this is a big demographic, and as the global male population ages, the number of men with low T is increasing. Interestingly enough, the FDA has only approved the current marketed products of testosterone for men who are between the ages of 30 and 65. Anything over 65 is considered a normal process of aging according to the regulators."

#AcesisBioMed #HormoneHealth #TestosteroneReplacementTherapy #TRT #MensHealthAwareness #BiotechInnovation #EndocrinologyResearch #LowT #MensHealth #Testosterone #HormoneResearch #Menswellbeing #Andropause

acesisbio.com

Listen to the podcast here

Dr. Costas Karatzas, CEO, Director, and Co-Founder of Acesis BioMed, is focused on developing a novel treatment for low testosterone in men, an oral peptide that stimulates the body to restore its own natural testosterone production. The therapy's mechanism differs from current testosterone replacement therapies and avoids the side effect of infertility seen in younger men using traditional TRT, which shuts down natural hormone and sperm production. This low T condition is not just a function of aging but is increasingly affecting younger men due to lifestyle, environmental factors, and co-morbidities like diabetes and obesity.

Costas explains, "Our mission is to revolutionize the treatment of low T, or male hypogonadism, and related comorbidities. Although we remain laser-focused on going in phase one with a low T application, because low testosterone is involved in many other diseases like type 2 diabetes, fatty liver disease, obesity, etc., we're also going to be co-developing those with partnerships initially with academia and later on with other pharma companies. So this is, in a nutshell, the short story about Acesis. The differentiation being everything else marketed provides the body with exogenous testosterone, whereas in our case, we teach the body to go back to its old memory and generate or restore its own testosterone in a natural way."

"Men can develop low testosterone because of the aging process, but this is a wrong idea. Low testosterone is not just an aging problem. It's true that we as men lose testosterone from the age of 30, about 1-1.5%, which is circulating in our blood. By the time we reach sixties or seventies, we could have probably 50% to 60% of the testosterone levels we had while we were at the age of 30 years old. Globally, it's estimated that about 40% of adult men may have suboptimal testosterone levels, especially if you include men with other health issues like diabetes, and we mentioned obesity. So, for older men, this is a big demographic, and as the global male population ages, the number of men with low T is increasing. Interestingly enough, the FDA has only approved the current marketed products of testosterone for men who are between the ages of 30 and 65. Anything over 65 is considered a normal process of aging according to the regulators."

#AcesisBioMed #HormoneHealth #TestosteroneReplacementTherapy #TRT #MensHealthAwareness #BiotechInnovation #EndocrinologyResearch #LowT #MensHealth #Testosterone #HormoneResearch #Menswellbeing #Andropause

acesisbio.com

Download the transcript here

Dr. Adam Rogers, CEO of NervGen Pharma, highlights the significant unmet medical need for treating spinal cord injuries (SCI), as there are currently no approved pharmacological treatments to promote repair. The science behind this neurotrauma company's drug is designed to interfere with the inhibitory molecules that prevent neurons from regenerating after injury, allowing the nervous system to repair itself. Recent trials have shown success in patients with chronic SCI, indicating that it is possible to promote recovery long after initial damage occurs..

Adam explains, "Our mission at NervGen is to improve the lives of individuals with spinal cord injury. And there are roughly 310,000 individuals with spinal cord injury in the United States, and about 18,000, roughly 18,500 new cases per year. And it's such a difficult injury to treat because, other than largely exercise and physical therapy, there are absolutely no approved pharmacologic treatments that promote functional repair for individuals who have suffered a spinal cord injury."

"I'm a physician by training. I practiced for nearly 20 years, and in the medical world, we have always been taught that when a neuron is damaged, as occurs in spinal cord injury, the neuron becomes dystrophic and cannot regenerate. So the medical community has always believed, and I was taught this and even in my practice, that once you have a damaged neuron, that neuron is dead. The problem that we have to solve here is really twofold. Number one, are neurons still viable after a central nervous system (CNS) injury? And number two, if the neuron is viable, how can we get that neuron to sprout, remyelinate, and regrow so that we can gain some function in the end organ that that neuron is supposed to innervate? NervGen's scientific founder, the late Dr. Jerry Silver, dedicated his entire career to understanding what prevented the central nervous system from repairing itself."

#NervGenPharma #SpinalCordInjury #RegenerativeMedicine #NeuroTrauma

nervgen.com

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Dr. Adam Rogers, CEO of NervGen Pharma, highlights the significant unmet medical need for treating spinal cord injuries (SCI), as there are currently no approved pharmacological treatments to promote repair. The science behind this neurotrauma company's drug is designed to interfere with the inhibitory molecules that prevent neurons from regenerating after injury, allowing the nervous system to repair itself. Recent trials have shown success in patients with chronic SCI, indicating that it is possible to promote recovery long after initial damage occurs..

Adam explains, "Our mission at NervGen is to improve the lives of individuals with spinal cord injury. And there are roughly 310,000 individuals with spinal cord injury in the United States, and about 18,000, roughly 18,500 new cases per year. And it's such a difficult injury to treat because, other than largely exercise and physical therapy, there are absolutely no approved pharmacologic treatments that promote functional repair for individuals who have suffered a spinal cord injury."

"I'm a physician by training. I practiced for nearly 20 years, and in the medical world, we have always been taught that when a neuron is damaged, as occurs in spinal cord injury, the neuron becomes dystrophic and cannot regenerate. So the medical community has always believed, and I was taught this and even in my practice, that once you have a damaged neuron, that neuron is dead. The problem that we have to solve here is really twofold. Number one, are neurons still viable after a central nervous system (CNS) injury? And number two, if the neuron is viable, how can we get that neuron to sprout, remyelinate, and regrow so that we can gain some function in the end organ that that neuron is supposed to innervate? NervGen's scientific founder, the late Dr. Jerry Silver, dedicated his entire career to understanding what prevented the central nervous system from repairing itself."

#NervGenPharma #SpinalCordInjury #RegenerativeMedicine #NeuroTrauma

nervgen.com

Download the transcript here