Dr. Charlotte McKee, Chief Medical Officer at Sionna, describes the nature of cystic fibrosis (CF, a genetic disease caused by a mutation in the single CFTR gene. While current CFTR modulator therapies do not address the most common mutation, Sionna's novel oral medicine is designed to target the previously undruggable NBDI domain of the CFTR protein. This new therapy aims to lead to better lung function and prevent the accumulation of permanent damage to other organs like the pancreas, gut, and liver.

Charlotte explains, "Cystic fibrosis is a genetic disease. The gene was actually discovered in 1989 for cystic fibrosis. It's considered a rare disease, but it's a relatively large rare disease. And one of those rare diseases that is potentially fatal, as you mentioned, it's thought of as a lung disease. And most patients, if their life is shortened, it's typically because of lung disease, because the lungs can be very severely affected. But the protein is caused by a genetic mutation in a gene called CFTR, and the protein is made from that gene. The CFTR protein is present on every epithelial cell of the body."

"Sionna is focused on a novel target in the CFTR protein. So you may know that, actually, there are approved medicines that have been developed over the last couple of decades that improve the function of the CFTR protein. And they've really advanced the clinical field, and there have been tremendous advances for people with CF. But this protein, this CFTR protein that goes wrong in CF, is a big, complicated, multi-part channel."

"Another unusual thing about CF is that there's one mutation that's so common around the world, and the part of CFTR that goes most wrong with F508del. This mutation is in a part of CFTR that was previously considered undruggable. It's that part that is called NBD1, and Sionna has been working for over a decade and a half of research, actually starting with Genzyme and then continuing through the company, Sanofi, has actually figured out how to develop potential medicines against this part of the CFTR protein that goes most wrong. And so we are working on these, they're called modulators, CFTR modulators, or we are working on NBD1-focused potential medicines that can directly bind to and stabilize this specific part of the CFTR protein."

#Sionna #CysticFibrosis #NBD1Stabilizers #CFTRModulators #RareDisease #Biotechnology #MedicalInnovation #PrecisionMedicine #GeneticDisease #PulmonaryHealth

sionnatx.com

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Mark Bertagnolli, Chief Operating Officer at ViroMissile, has developed a modified vaccinia virus that can be delivered intravenously to seek out and destroy solid tumors throughout the body and is not dependent on a specific genetic driver of a cancer. The virus has been engineered to be resistant to the body's complement system, allowing it to survive in the bloodstream to infect the tumor. This action also turns the tumor microenvironment from cold to hot, signaling the immune system to attack the tumor and potentially working in combination with other treatments like PD-1 inhibitors to make them more effective.

Mark explains, "ViroMissile has a modified vaccinia virus, and viruses tend to like to infect tumor cells. So the trick has been how to harness this. And we're on our third generation of virus, and we have a virus that you can inject intravenously that searches for tumors throughout the body, infects them, the body clears the rest of the virus, and the virus embeds itself in the tumors, replicates, spreads, activates the immune system, and destroys the tumors."

"So, the vaccinia virus itself, nobody knows really where it came from, but it ended up being the foundation of the smallpox vaccine, and we get the word vaccine from the vaccinia virus. Our founder was the first to sequence the genome of the vaccinia virus over 30 years ago. For the last 20 years, he's been working on it as an oncolytic agent. And in this particular case, within vaccinia, there are different strains. And he was able to isolate a very specific, never before isolated strain of virus that is resistant to complement attack, and also replicates very quickly in infected cells. So it has two embedded features within it, naturally, that we're able to capitalize on."

#ViroMissile #CancerResearch #Immunotherapy #OncolyticVirus #Biotechnology #MedicalInnovation #CancerTreatment #PrecisionMedicine #HealthcareInnovation #CancerCare #VacciniaVirus

viromissile.com

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Dr. Ronald Lane, Founder of HOPE-Neuron Therapeutx, is developing an approach to treating ALS by rebalancing the body's immune system, which becomes dysfunctional in neurodegenerative diseases. The mechanism involves treating a patient's blood outside the body to activate the immune system and then infusing it back into the patient. The theory is that traditional drugs often fail because they target static genetic mutations and symptoms, while the source of the disease lies in the ongoing immune imbalance.

Ronald explains, "Well, in a broad sense, the goal of HOPE Neuron is based on the fact that our challenge here is to bring hope. And we use a neuronal basis to do that. We're not about drugs. There are no chemicals involved. We don't go that direction. But basically, it cuts across. We deal with memory, we deal with dementia and Alzheimer's, but our target initially is ALS."

"As I mentioned a moment ago, we're not doing drugs, we're not using chemicals. So what are you doing? Well, we're using the cell's immune system, and the problem with disease is that the immune system gets out of balance. When it's out of balance, you become ill. It can be in many different diseases. And there are certain indicators, and we know what to look for and what compounds the body generates."

#HOPENeuron #ALS #Neurology #ImmuneTherapy #MedicalInnovation #ALSResearch #Biotechnology #NeurodegenerativeDisease #MedicalBreakthrough #ClinicalTrials #Healthcare #Innovation #Neurodegeneration #Neuroplasticity #MedicalDevices #Immunotherapy #NeuroscienceInnovation #FutureOfMedicine #NonPharmaTherapies

hopeneuron.com

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Dr. Galia Schwarz, CEO and Co-Founder of C8 Health, has developed a platform that addresses the gap between hospital best practices and their implementation across all departments. This approach consolidates medical knowledge from numerous disconnected systems and uses AI to proactively deliver relevant information to a clinician. C8 Health does not create best practices; it ensures that a hospital's vetted standards are communicated consistently to provide optimal patient care.

Galia explains, "Hospitals are struggling to implement their best practices effectively, or in other words, control the quality and cost of care delivery. And let me explain this. There is tons of medical knowledge out there, and now with AI coming into our lives, there is even more. And hospitals create this knowledge through clinical trials, or they license this knowledge through platforms like Open Evidence or UpToDate. And every hospital takes these global best practices and turns them into local best practices, things like protocols, guidelines, procedures, and educational content. Any type of knowledge whose purpose is to ensure that standards of care are met. But I haven't met one hospital that knows how to take these best practices and turn them into actual practice, and this is what we allow them to do."

"So first of all, on a conceptual level, our platform consolidates fragmented knowledge across departments or sites into a single source and uses AI to surface the right guidance at the right moment based on the clinician's role, schedule, and patient context. So, for example, we can create an experience where we say, "Hi, Dr. X. Tomorrow you are scheduled for OR number eight. You have these four procedures. At 8:00 AM, you have a CABG procedure. These are the best practices of your hospital for this procedure. This is what changed since the last time you did this procedure. These are the people who are going to be with you in the OR."

#C8Health #HealthTech #AIinHealthcare #MedicalInnovation #PatientSafety #DigitalHealth #HealthcareTransformation #MedicalErrors #BestPractices #ClinicalExcellence #HealthcareAI #ArtificialIntelligence #AI #OpenAI

C8health.com

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David Klein, a longtime healthcare innovator and Founder and CEO of Click Therapeutics, is a pioneer in the field of prescription digital therapeutics, PDT, which are defined as software as medicine. Click has received FDA authorization for an app to treat depression and migraine and had a successful pivotal trial for an app treating negative symptoms of schizophrenia. These digital therapeutics are designed to fill significant gaps in current care and there are opportunities to combine software with drugs and biometric data from wearables to determine personalized treatment plans.

David explains, "Click's approach towards digital therapeutics is, I would say, probably the most rigorous from a clinical evidence perspective in the industry. So we really take a drug-like clinical approach to these programs where we discover and then validate these programs in multiple randomized control trials, all culminating usually in a pivotal trial. And on top of that, you'll often see in some other areas of the world or even in this country, to some degree, folks might say randomized control trial, but they're really using standard of care as the control or wait list control, and so on and so forth. We actually test our interventions against sham active control, so against other apps that control for time on task, expectation of benefit, and so on and so forth."

"Just a few months ago, we announced with Behringer Ingelheim, our partner in schizophrenia, that we hit our primary endpoint in our pivotal trial for our app that's designed to be a treatment of negative symptoms of schizophrenia. So, the holy grail, in serious mental illness altogether, but certainly in schizophrenia, is trying to mitigate negative symptoms of schizophrenia. This is such a huge patient issue and frankly an economic issue that none of the drugs have been successful at actually hitting it. So that's a unique one because we showed that an app can actually effectively and safely treat a disease that even a drug can't."

#ClickTherapeutics #DigitalTherapeutics #DigitalHealth #FDA #MedicalDevices #SoftwareAsMedicine #HealthTech #Innovation #Migraine #Depression #Schizophrenia #PrescriptionApps #PDT #PrescriptionDigitalTherapeutics #ClinicalTrials #HealthcareInnovation

clicktherapeutics.com

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Tony Baldassarre, President and CEO of UniDoc Health, has a mission to provide accessible healthcare through remote technology and a Health Cube that can be deployed in a wide range of settings. This approach integrates into existing EMR/EHR platforms and includes a healthcare professional who works with the patient in the Cube to interact with medical devices and consult remotely with a doctor. There have been successful deployments in Ukraine, Italy, and Alaska, showing a significant reduction in emergency room visits and demonstrating capabilities for specialized care and management of chronic conditions.

Tony explains, "With the UniDoc solution, what we do is not just enable a conversation between a doctor and patient - it is not just a phone or video call. We actually provide real diagnostic medical devices for doctors to conduct a comprehensive medical visit remotely, just as if the patient were in their office. The important thing is that number one, we only use real medical devices. These are medical devices that are approved by Health Canada, the FDA, and the European Union. They are the exact same medical devices that you find at a doctor's office or a hospital. So the doctors are actually conducting the visit, and the data that they're receiving is exactly the same as it would be if they were together in the office."

"So, simply what happens first and foremost is that the patient, either through the browser, the UniDoc website, or through our app, chooses a doctor, be it male, female, different races, languages, etc. Once they have the appointment confirmed by the doctor, the patient just shows up at the Health Cube at the time that they requested the appointment, and they walk in. Once inside the Cube, there's a chair and a nurse, I'll call them a nurse for generality. There's a nurse on site, and the doctor is the only person who is remote. At that point, the nurse connects the doctor to the visit, and the medical visit starts."

"We have two Cubes in Ukraine right now, actually in Kyiv and at the border of Romania and Ukraine. That's been set up there since April of this year. We've already seen over 3000 patients, and a lot of it is actually being used by people in the town and for chronic diseases. On top of that, by law in Ukraine, we had to place these Cubes by the hospitals. That's a requirement. And the hospital is actually seeing a 35% decline in people going to the emergency room."

#UniDocHealth #HealthTech #RemoteHealthcare #MedicalInnovation #Telehealth #HealthcareAccess #Telemedicine #MedicalDevices #HealthEquity #DigitalHealth #RuralHealthcare #HealthcareInnovation

UniDocHealth.com

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Dr. Jennifer Perusini, CEO and Founder of Neurovation Labs, is developing a precision medicine platform to create targeted therapeutics and diagnostic tools that treat specific brain regions to address central nervous system diseases. For the treatment of PTSD, the approach focuses on the overactive amygdala due to dysregulated glutamate signaling. This objective, brain-based approach of treating a discrete part of the brain rather than the whole brain moves mental health treatment beyond the current one-size-fits-all approach, reducing side effects and improving outcomes.

Jennifer explains, "What we are doing is really taking an objective, precision approach to treating mental health disorders, something like an oncology model. So what we really care about is that mental health disorders and psychiatric disorders are rooted in brain dysfunction, yes, but critically that dysfunction occurs in very discreet brain regions and circuits, not necessarily uniformly across the entire brain. So we have really developed a platform designed to identify compounds with dual potential. Precision therapeutics that act on specific brain regions, as well as diagnostic imaging agents that can reveal receptor-level dysfunction and circuit activity in the brain. And so our lead asset and main indication right now is for PTSD, post-traumatic stress disorder."

"I think that academics like to focus on this research in very specific brain regions, but that therapeutic angle really hasn't caught up yet. And so, really more specifically, what we're doing is focusing on a very particular signal, an excitatory neurotransmitter in the brain called glutamate, which is a key regulator of neural activity and cell firing. And so there are many disorders, including PTSD, that are affected by dysregulated glutamate signaling. However, the glutamate drugs that are on the market today really bind to the whole brain. So that's just something that hasn't caught up yet, and that's what we're trying to do."

#NeurovationLabs #PrecisionMedicine #PTSD #Neuroscience #MentalHealthInnovation #Biomarkers #DrugDiscovery #Glutamate #BrainHealth #DigitalHealth #HealthTech

NeurovationLabs.com

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Dr. Paul Testa, Chief Health Informatics Officer who helped create the About Me Initiative at NYU Langone Health, and Dr. Katherine Hochman, Director of Hospital Medicine at NYU Langone Health, who envisioned the initiative, discuss the benefits of encouraging patients to provide non-medical information. The goal is to help care teams see patients as whole people beyond their diagnoses and to give patients an opportunity to share interests, hobbies, and fun facts that can help build trust and meaningful human connections. This About Me Initiative is being expanded from inpatient care to outpatient settings, such as infusion centers.

Paul explains, "The About Me section in our EHR Epic is something that NYU Langone developed with the inspiration of Dr. Hochman, and it's essentially a dedicated discreet space in the medical record to allow patients to declare themselves, allowing them to give them a space that is transparent to all the users of the electronic health record, which are many and vast in their rules. So let them articulate something beyond their disease process or their health. So often, we reduce records to pneumonia, a broken hip, or an infection. The patient is so much more than that, and patients want to share that with us. We know that from practicing for years, Kathy and I both know that the way you connect with patients is through a narrative. Our currency is narrative. This is a location where either a patient or, if they choose to defer, can have one of their nurses or care team members provide a small sample of who they are outside of the care environment."

Katherine elaborates, "So I'm a hospitalist. Every Tuesday, I come into 16 to 18 new patients. And at NYU Langone, our length of stay keeps getting shorter and shorter, which is fantastic for patients because we want to get them in and out of the hospital as quickly and as safely as possible. But by definition, if patients stay shorter and shorter, we don't have as much time to connect. So the About Me Initiative really kicks off that connection that Paul was talking about a second ago. So long ago, and this is how it started, I would tell my teams, slow down to speed up, get to know who this patient is before getting into the heart failure, the pneumonia, the COPD, and really try to establish a little bit of a connection and build trust."

#NYULangone #PatientVoices #PatientCare #HealthcareInnovation #PatientExperience #HealthIT #EHR #EpicEHR #HumanConnection #HealthcareTransformation #PatientCentered #MedicalTechnology #DigitalHealth #EmpathyInMedicine

nyulangone.org

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