Ryan Clarke, Co-Founder, CEO, and CTO of Syntax Bio, is focused on solving manufacturing challenges in producing stem cell-derived therapies, specifically the process of stem cell differentiation. The Cellgorithm platform was designed to make differentiation an engineering problem by using a modified form of CRISPR to turn genes on and off in a specific, controlled sequence, reducing processing time from months to days and resulting in cost savings. One goal is to use AI and machine learning to build models capable of predicting optimal gene regulation sequences accelerating the discovery of new differentiation protocols and treatments for degenerative diseases.

Ryan explains, "The key problem that we are focused on applies to stem cell-derived cell therapies. And so, just for a little bit of context setting, we use induced pluripotent stem cells or IPS cells. And about 20 years ago, when these were first derived, everybody was very excited because this is the platform where you could then have an infinite supply of stem cells to then make any tissue-specific cell type at will, theoretically. Fast forward 20 years, and there are finally some cell therapies in phase three clinical trials, but none are approved yet. And that just tells you that the development cycle for this modality is actually slower than the other modalities, like biologics or small molecules. And the problem is manufacturing in particular, or how do you convert the stem cell into the target tissue cell type for the process of stem cell differentiation? And so we are solely focused on making stem cell differentiation an engineering problem rather than a kind of dark art."

"We have a program for type one diabetes that is making pancreatic beta cells from IPS cells. And we have also done some work in the musculoskeletal system. So these other cell types we focus on are more demonstrations of the platform technology, but we are interested in possibly co-developing those with other pharmaceutical partners, and that's the musculoskeletal cells of the hematopoietic system. And we've done some work on retinal cells as well, but we endeavor to make many other cell types. And our goal is to partner with the therapeutic experts in the area to develop these."

#SyntaxBio #CellTherapy #CRISPR #StemCells #Biotech #RegenerativeMedicine #Diabetes #Manufacturing #Innovation #GeneTherapy #LifeSciences #Bioengineering #SyntheticBiology #CellProgramming

syntax-bio.com

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Dr. Daniel Sodickson is Chief Medical Scientist at Function Health and author of the new book, The Future of Seeing: How Imaging Is Changing Our World. Building on the drive to improve medical imaging, AI and neural networks are now reshaping image interpretation and how and what data are collected. This approach produces high-quality results with minimal additional data and is inspiring innovative scanning techniques and equipment design. The future of medical imaging is the everywhere scanner, enhancing a single-shot session with a large hospital machine with continuous health monitoring through wearables and devices integrated into everyday life.

Daniel explains, "One of the things that imaging can do is peel away all of the obscuring layers of skin or skull or whatever else there is, without having to make a single cut, and show us the inner workings, show us inner space, what's inside. I think that means being able to detect tumors early enough that they can be cured, to guide surgeries, to try to understand what normal anatomy is, and exactly when it turns abnormal. So I think the ability to see what was once invisible has become so much a part of medicine that it's almost hard to imagine it without it."

"There are many analogies between inner space and outer space, and between the tools we have built as humans over the millennia to inspect them. I guess what I'd say, though, is that somehow the inspection of inner space, that sort of medical imaging for understanding our health, is a little bit more intimate. It causes us to ask very personal questions like, " Am I okay? Are my kids okay? Am I normal? What is normal? I think when we look at other types of imaging, imaging the world around us, imaging the cosmos a great distance from us, there are also existential questions, but it's really more, where do I fit in the big picture? So I think in some ways medical imaging picks up where, say, astronomical imaging leaves off and leaves us wondering who we are and how we're built."

#DanielSodickson #FutureofSeeing #FunctionHealth #AIHealthcare #MedicalImaging #HealthTech #FunctionHealth #DigitalHealth #MRI #HealthcareInnovation #PatientEmpowerment #FutureOfMedicine #HealthcareAccessibility

functionhealth.com

The Future of Seeing: How Imaging Is Changing Our World

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Dr. Howard Federoff, Scientific Co-Founder, Chief Medical Officer, and Executive Vice President of Corporate Medicine and Science at Kenai Therapeutics, is developing a cell therapy for Parkinson's disease that involves transplanting IPSCs into the brain to replace lost dopamine neurons. The goal is not to cure the underlying cause of the disease, but to restore motor function and reverse the disease's progression. Initial clinical focus was on patients with moderate to moderate-severe idiopathic Parkinson's, showing efficacy and safety, leading to expanding the study to include earlier-stage and familial forms of the disease.

Howard explains, "Kenai was formed about three and a half years ago, and the intention is to develop a cellular product, which means that what is manufactured will be eligible in the right patient groups who have a diagnosis of Parkinson's to be placed into the brain. And consequent to its placement, the cells will then form new circuits, dopamine circuits that are lost owing to the disease diagnosis that will have occurred years earlier."

"The induced pluripotent stem cell approach is one we favor for several reasons. The nature of what then becomes the IPSC, as we like to call it, starts with a normal human volunteer whose medical history is very detailed and does not contain any familial personal history of neurodegeneration. That cell then undergoes a process called reprogramming. And in our case, it's done with a slightly different approach than many others. And the reprogramming effectively creates a cell that is pluripotent, meaning it can become any cell of the body. That's when it is designated as an induced pluripotent stem cell."

#KenaiTherapeutics #ParkinsonsDisease #CellTherapy #StemCells #Neuroscience #MedicalInnovation #ClinicalTrials #RegenerativeMedicine #Biotechnology #BioTech #AdvancedBiologics #NeurologicalDisorders #Neurology #DrugDevelopment

KenaiTx.com

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Brent Ness, CEO and President of Aclarion, highlights the challenges of diagnosing and treating chronic lower back pain, a leading driver of healthcare costs and opioid addiction. Traditional MRI and CT imaging do not reveal the biochemical source of pain within spinal discs, leading to misdiagnosis and unsuccessful treatment. The Aclarion technology uses MR spectroscopy to measure pain-causing biomarkers and, through a cloud-based, AI-powered SaaS model, analyzes the raw data and sends the physician a report within minutes.

Brent explains, "The diagnosis and accurate treatment planning of back pain are incredibly complex. And when you think about pain management physicians, rehab, all the way up to spine surgery identifying the source of pain accurately leads to better treatment and then obviously better outcomes.There are 266 million people around the world who suffer from chronic low back pain. So I'm not talking about the kind that you had a rough weekend skiing, golfing, or hiking, and you're a little sore. I'm talking about the kind that keeps people from participating in a meaningful life. "

"When you think about the joints and the sources of blood flow, the nerves that are all around your spinal cord, the vertebral columns, and there's just a lot of moving parts and a lot of really, let's just call it high-value real estate that can actually be the source of pain. And really, our superpower is to help physicians see the invisible. Meaning that normally when you go to the doctor, and they do a workup on you, they'll use an MRI or a CT scanner. And those modalities are really good at pinpointing anatomical issues that might be causing pain. What we do is we use MR spectroscopy, not to make a picture of your back, but rather to measure the biomarker content inside the discs that are invisible on a normal MRI. And as it turns out, what's inside your disc can actually be the source of pain."

#ACON #CLARITYtrial #lowbackpain #spinesurgery #MRSpectroscopy #Biomarkers #AugmentedIntelligence #innovation #ChronicPain #BackPain #MedicalTechnology #AI #HealthcareInnovation #SpineCare #MRI #PainManagement #DigitalHealth #Diagnostics #HealthTech #PatientCare

aclarion.com

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Dr. Alanna Morris-Simon, Senior Medical Director for US Medical Affairs at Bayer, describes the symptoms and diagnostics used to classify heart failure and the key at-risk populations for this condition. The rapidly evolving landscape of heart failure treatments now includes the Bayer drug KERENDIA, a non-steroidal MRA approved to reduce cardiovascular death and heart failure in adults with an ejection fraction of 40% or more. This drug is part of an emerging trend to treat multiple related conditions simultaneously and could prevent the onset of heart failure and treat established heart failure.

Alanna explains, "At a basic level, heart failure is a clinical syndrome, and that's important. I'm actually a heart failure cardiologist as well. And so this is important because patients have to have signs and symptoms. And those signs and symptoms really result from the heart being unable to either fill with blood properly or squeeze that blood out in a way that meets the body's demands. Either way, patients experience the same symptoms, and those include symptoms like swelling and weight gain, shortness of breath, either at rest or with activity, fatigue, abdominal swelling and bloating, loss of appetite, as well as other symptoms."

"If a doctor or a clinician suspects a diagnosis of heart failure, 99.99% of the time, they'll start by ordering an echocardiogram or a heart ultrasound. Of course, the guidelines tell us to get a chest X-ray, get labs, those sorts of things. But really, we make the diagnosis for the most part based on the results of an echocardiogram because that echocardiogram allows us to visualize how the heart is pumping. It allows us to classify the type of heart failure so that if we see that the squeeze of the heart is impaired, we call that heart failure with reduced ejection fraction. And that's when the ejection fraction or EF is 40% or less. If the EF is in the 41 to 49% range, we classify that as heart failure with mildly reduced ejection fraction. And if patients have an ejection fraction of 50% or greater, we call that heart failure with preserved ejection fraction or HFpEF."

"And we were excited that the FDA actually granted a priority review for KERENDIA because this really only occurs when the FDA recognizes that a treatment can fill a significant unmet need for a disease or a population of patients. And lo and behold, in July of 2025, finerenone was approved by the FDA under the trade name KERENDIA to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visits in adults with an ejection fraction of 40% or more."

#Bayer #Finerenone #Pharma #HeartFailure #HFpEF #HFmrEF #MRA #UnmetNeed #Cardiology #KERENDIA #FDA #CardiovascularHealth #MedicalBreakthrough #PatientCare #Innovation

Bayer.com

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Steve Brown, Founder and CEO and Lisa Booth, Vice President of Operations of CureWise, are both cancer survivors, which led them to develop an AI-powered tumor board platform to advance cancer diagnoses and treatment. This approach to precision medicine provides patients with the opportunity to better understand their specific condition, educating them about possible courses of action that may be more appropriate than the standard of care. The platform also helps patients find relevant clinical trials and manage the side effects of their treatments.

Steve explains, "So the mission really is to harness AI to advance cancer care. And ultimately, for that reason, anybody who's touched by cancer is hopeful that we will be making more progress toward cures for cancer. And we believe that that's going to happen through precision medicine, which really means recognition that everybody's cancer is unique. And at some point, we're going to treat cancer that way because we're going to know enough about how it all works. We're going to be able to individualize care. So our mission is to advance that cause."

Lisa elaborates, "When Steve and I started working together, one of the things I said was that I needed a way as a patient to be able to access different perspectives of a radiologist, an interventional radiologist, a pathologist, a geneticist, an oncologist, etc. And I can get that in Seattle, but most of the people I work with can't, meaning the patients and my friends in the cancer community don't have access to that because they don't exist in their communities."

#CancerCare #AI #PrecisionMedicine #PatientEmpowerment #HealthTech #CancerSurvivors #DigitalHealth #Oncology #TumorBoard #ClinicalTrials #HealthcareInnovation #PatientAdvocacy #PatientLedInnovation #CancerAndAI #TumorBoardTech #AIForPatients #SteveBrownAI #LisaBoothSurvivor #MedicalAutonomy #HealthTechWithHeart #NOfOneMedicine

curewise.com

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Nayla Chaijale, Medical Strategy Lead for Rare Syndromes at UCB, describes Dravet syndrome, a rare, medication-resistant epileptic encephalopathy that involves seizures and significant co-morbidities like cognitive and developmental delays. Their approved drug FINTEPLA has a unique mechanism of action that modulates pathways in the brain and has demonstrated a significant reduction in the frequency of convulsive seizures in those with this condition. Nayla notes that the good news is that most patients with Dravet syndrome have a mutation in the SCN1A gene, a critical biomarker that enables accurate diagnosis.

Nayla explains, "So, Dravet syndrome is a rare condition in epilepsy, and what we call it is neurodevelopmental and epileptic encephalopathy. I'm just going to call it DEEs, to say that it's a rare epileptic syndrome. Even though it's very rare, it's also very burdensome for the patients and their families. So, people living with these conditions start having the symptoms at a very early age, between two and 15 months of life. And it usually appears after a fever or a high temperature. And the symptoms are seizures that usually are long, prolonged seizures. It's not just about the seizures, it's also the other symptoms, such as potential cognitive delays and other developmental delays, problems with sleep, problems with behavior over time."

"The good news about Dravet syndrome is that there is a very well-established biomarker. So, there is a genetic test for it, for the gene code that is called SCN1A. And also, in science, we love to have acronyms, but really, it's related to a specific type of receptors or proteins that are in charge of neuronal transmission and communication, like in the neurons. And these are called sodium channel neurotransmitters. And there is a mutation of that gene that is very characteristic, around 90% of these patients will have that mutation of the gene. So, having that biomarker will give a very good indication to the healthcare provider that the person living with the conditions may have Dravet syndrome."

#UCB #DravetSyndrome #RareEpilepsy #FINTEPLA #Neuroscience #RareDiseases #EpilepsyAwareness #MedicalInnovation #PatientCare #NeurodevelopmentalDisorders #ClinicalTrials #RareDiseases

ucb.com

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Vanessa Lemarié, Chief Operations Officer at myTomorrows, which is a platform for connecting patients, physicians, and clinical trial sponsors using a Trial Search AI tool designed to improve the accuracy of matching patients with suitable trials. The technology aims to speed up trial recruitment, increase diversity by reaching underserved populations, and ultimately shorten the drug development timeline. There is a particular focus on patients with no treatment options or who have not responded to current therapies.

Vanessa explains, "We are all about patients. First and foremost, we want to help patients know their options. Patients who turn to us either have no treatment options for their conditions or have exhausted the treatment options. They are looking for alternatives and insights into whether we can help them find, for example, a clinical trial. Physicians who treat those patients turn to us."

"And last but not least, we work with biopharma companies that plan and conduct clinical trials and that sponsor expanded access programs. And those are our clients. So we connect all of those stakeholders. In addition, we work very closely with representative groups of patients and patient advocacy groups to make sure that we are known and to make sure that we understand what specific patient groups might need from companies like ours. So we kind of connect the dots, I guess, and an otherwise somewhat siloed system sometimes, and consider ourselves a platform player. Think of us a little bit like, let's say, booking.com for clinical trials."

#myTomorrows #ClinicalTrials #HealthTech #PatientAccess #RareDiseases #DigitalHealth #Healthcare #Innovation #AI #PatientEmpowerment #Biotechnology #Pharma #MedicalResearch #HealthcareEquity #Technology

mytomorrows.com

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