Significance of Regulatory RNA in Protein Production in Specific Genes with Dr. Yuri Maricich CAMP4 Therapeutics

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 Download the transcript 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 Download the transcript here

Dr. Sanjay Kakkar, CEO of Tensive, has developed an innovative bioresorbable polymeric scaffold for breast regeneration following a lumpectomy.  The scaffold was designed to address a significant unmet need for breast cancer patients who have limited options for reconstruction. A key feature of this technology is that it facilitates a natural wound-healing response, allowing the body's own vascular, connective, and fat tissues to regrow as the scaffold gradually dissolves. Sanjay explains, "What we've been developing at Tensive is at a late clinical stage of these advanced biomaterials, and we've spent the last decade or so pioneering this bioresorbable polymeric scaffold for natural tissue regeneration. So, coming directly to your question, we're on this mission to provide breast cancer survivors with the option of a natural breast reconstruction. What we developed is this resorbable biopolymer that can be implanted at the time of the lumpectomy procedure." "But what it does is it replaces volume immediately, and it facilitates new tissue growth without cells or growth factors. This sponge, which is made of a fine scaffold matrix, gradually degrades. So it immediately fills the cavity left by the tumor removal. It facilitates natural tissue regeneration, allowing the body's own cells to grow back into the 97% of empty space, which is formed by this sponge. And that basically means that initially vascular tissue regrows inside the porous architecture, then connective tissue, and then fat comes back."  #Tensive #BreastCancer #BreastCancerAwareness #ClinicalTrials #ReconstructiveSurgery #REGENERA #BreastHealth #BreastReconstruction #Lumpectomy #Innovation tensive.com Download the transcript here

Dr. Saria Saccocio, Chief Medical Officer at Essence Healthcare, is using AI to enhance care and services for patients who are enrolled in Medicare Advantage programs. One application is to analyze data related to health equity, care authorization, and to address biases. Their AI agent is being used to interact with patients, offering them the option to access a human. Integration with wearables, such as the Oura Ring, focuses on identifying clinically useful information to prevent physicians from being overwhelmed by data. Saria explains, "Medicare Advantage plans are absolutely that. It's an advantage because of the additional benefits that exist and the opportunity to connect with our members is rather unique. In fact, what I'll share with you and the audience is that AI has been a focus for us and a priority as we continue to expand the focus on quality and engagement with our members so that they have their best quality life possible to them. AI is a major piece of this work to make that happen. I can give you a couple of examples. In fact, when it comes to health equity, CMS has recently required Medicare plans to ensure that we are analyzing our data through a health equity lens. This year, in fact, we're focused on authorizations, authorizations of care. We want to make sure that it is critical to our members and AI. I see an opportunity to recognize any blind spots or implicit bias."   "I think that every human being has a capability for technology. And what's critical is that we meet them where they are, that we understand what they want, what they need out of technology. I think that works as well as providers. As you see all of these AI developers starting up companies and solving problems, the conversation that we're having every day at Lumeris and at Essence Healthcare is what does the patient want? What does that member need for quality of care? And then engage and include the providers in that conversation. What makes their job easier? Because one of the challenges with new technology is that we can also reach a point of technology exhaustion. So I'll give an example of our relationship with Oura ring, the wearable device that tracks for sleep, for health, for heart rate, and a number of different measures."    #EssenceHealthcare #MedicareAdvantage #HealthAI #OuraRing #DigitalHealth essencehealthcare.com Download the transcript here    

Kerry Mello-Parker, Director of Rare Diseases and REMS programs at Shields Health Solutions, addresses the complexities and challenges faced by patients with rare diseases including diagnostic delays and limited access to medication. Using an integrated specialty pharmacy model, Shields works with pharmacists to have direct access to the EHR, enabling better patient monitoring, shorter time to initiate medication, tracking outcomes, and providing patients with a direct line of communication to their care team. Pharmacists treating rare diseases are also supported with specialized information on medication administration, side effects and complex billing.  Kerry explains, "Shields Health Solutions is a specialty pharmacy accelerator. We partner with over 80 health systems across the United States. We support them in establishing and growing a specialty pharmacy. We help them to access critical medications. Some of them are not available at traditional pharmacies, and we support them in managing clinical quality care programs for their patients." "I'd like to step back a little and talk about what a rare disease is and how we define it. Currently, it is defined as a condition that affects fewer than 200,000 people. However, over 7,000 rare diseases exist. So when we do the math, we come to see that they are not so rare. And some estimates show that up to 10% of the US population may actually have a rare disease. And what I've seen is that there are really three main complexities of treating rare diseases and challenges that patients may face. One of them is that there is a lack of available treatment options when compared with more common diseases. So, for example, only about 10% of rare diseases have an FDA-approved treatment option, but that is changing. We have the Orphan Drug Act, which has actually changed the landscape. It has incentivized and facilitated the development of medications to treat rare diseases." #ShieldsHealthSolutions #IntegratedCareModel #SpecialtyPharmacy #RareDiseases #MedicationAdherence shieldshealthsolutions.com Download the transcript here

Ram Sahasranam, President and Co-Founder of Fold Health, has created a platform that integrates with the EHR to create an AI-driven interface that streamlines workflows, improves communication across the care team, and helps better manage patient care. Traditional EHR systems, while effective for documentation, were not built for proactive care and data sharing. With a focus on the patient and providers, Fold Health is integrating data sources and automating redundant administrative tasks helping to reduce clinician burnout and improve patient communication for scheduling and reminders. Ram explains, "The way to look at it is that empowering providers and patients need to go hand in hand. That is the fundamental reason why we called ourselves Fold Health, which is that healthcare needs to fold around the providers and the team supporting the providers and the patients and the families of the patients who are involved in the care. We have seen healthcare go from $1.6 trillion in spending to $5.3 trillion in spending this year. But in those 15 years, we haven't yet seen it fold around the two most important people, the provider or the patient, in terms of better outcomes or improving the burnout rates that clinicians and their teams face today." "So the fundamental challenge that we noticed was that multiple people were living in different systems and hence different silos from a data standpoint. So the clinician did not know what was happening with their care teams. The patient was living in a different system. If the patient goes to a specialist, the data comes in a compressed format to the physician in an easily digestible manner, which was a challenge. So what Fold does as a result of getting control of all these different nodes is that data can be used to summarize and provide things for the different teams in a quick, easily digestible manner."  #Foldhealth #CareCoordination #CareDelivery #ConnectedCare #HealthTech #AI #HealthAI Fold.Health Download the transcript here

Ralph Keiser, CEO of ArcheHealth, highlights the severe financial pressures that hospitals are confronting as a result of rising costs and declining reimbursements. Reductions in reimbursements are threatening hospitals that serve underinsured populations. Costs are increasing due to tariffs on medical device components and the general price inflation on medical supplies and drugs.  ArcheHealth is focused on helping hospitals improve operational efficiency to lower the expense of delivering care. Ralph explains, "The tariff index is going to come mostly around medical devices and device components. So plastics, metals, packaging, things that the device manufacturers use to deliver their products to hospitals and health systems, those will likely get passed through. It won't be huge incremental costs, so there will be additional costs due to tariffs. It depends on a couple of things. Do you have a strong contract that sort of embargoes your current price? Then maybe in the next contract term you'll see that pass through or the contract so the supplier to pass through, you'll see them. We're monitoring month by month the current or last month's spend was on these items, and do we see a chart impact by manufacturer by line." "One thing that I want to make sure we talk about, too, isn't just shared impact. So the cost to a hospital for devices and drugs averaged 8.9% more in 2024. So just by nature of the market raising its prices for goods that hospitals use to cover patient care, those moved up without tariffs 8.9% last year. We think we're tracking increases that are happening despite the tariff overlay or on top of the tariffs. We still think that the pressure on hospitals is due to the price of therapeutics, and is going to continue to go up, tariff or not. And so I want to mention that because this is the ongoing impact of the cost of MedTech and drugs that gets passed through to the consumer." #ArcheHealth #AIinHealthcare #HealthTech #HealthcareOperations archehealth.ai Download the transcript here

Dr. Marc Salzberg, President, CEO, and Chief Medical Officer at Airway Therapeutics, is developing a drug AT-100 to prevent bronchopulmonary dysplasia (BPD) in preterm infants, a severe condition with no existing treatment. These infants are at high risk for BPD because of a low level of a protective protein that reduces inflammation and clears infections from their underdeveloped lungs. The drug's mechanism of action suggests it has potential for treating other inflammatory conditions such as asthma, COPD, and severe pneumonia. Marc explains, "We're focusing on developing drugs or interventions for severe respiratory conditions for prevention or treatment of severe respiratory conditions. And our first project is focusing on the respiratory condition that preterm neonates have, which is called bronchopulmonary dysplasia, and cannot be treated or prevented yet. It's a very severe condition. And that really triggered my interest in getting engaged because it's going to be a paradigm shift if we manage to bring this drug to market." "The protein that we're developing is an endogenous protein that we all have. In the past years or decade, it has been discovered that this protein has a very essential function in fighting inflammation and infection on a daily basis. Now, there are certain conditions or instances where the protein is low-functioning. For instance, in preterm-born babies, but also in adults with severe respiratory inflammation, with pneumonias, with asthma, or COPD. In all those cases where protein is low, those are the instances where you actually need it most. So, replacing it in those instances is a kind of logical rationale for the concept for the treatment that we're developing." #AirwayTherapeutics #BPD #InflammatoryDiseases #Zelpultidealfa #PrematureBabies #PretermBabies airwaytherapeutics.com Download the transcript here