Empowered Patient Podcast

Nolan Townsend, CEO of Lexeo Therapeutics, and Jen Farmer, CEO of FARA, the Friedreich's Ataxia Research Alliance, are working together to better understand and treat Friedreich's Ataxia. This rare genetic condition includes cardiomyopathy and scoliosis. Lexeo Therapeutics is studying gene therapy as a potential treatment for the cardiovascular component of the disease. The Friedreich's Ataxia Research Alliance (FARA) plays a role in funding research, understanding the natural history of the disease, and advocating for treatments. Nolan explains, "For most patients in Friedreich’s ataxia, cardiovascular disease becomes a very challenging component of the pathology, and it is what’s called a hypertrophic cardiomyopathy phenocopy. So, it mimics hypertrophic cardiomyopathy, which is a thickening of the heart wall. And that typically progresses as well, which, for many patients, ends up being the cause of mortality associated with the disease. This pathology does appear to be amenable to treatment approaches li

Dr. Craig Joseph, Chief Medical Officer at Nordic Global, highlights the concerns and challenges healthcare professionals face when using health IT and the clinician burnout related to the administrative burdens of electronic health records.  He addresses the obstacles to integrating IT into healthcare workflows, including cost, risk aversion, and resource limitations. He identifies areas where AI can have an immediate impact in reducing errors and biases. Craig elaborates, "In the past, we lived in a paper-based world, so it was pretty flexible. You could fill out a form or not fill out a form. You could give as much detail or as little detail. Often, especially physicians really couldn't read what they were writing anyway, so it was less important. All of that's changed now in the last 15 or 20 years since the advent of electronic health records." "What we've seen with large language models, like Chat GPT and other modern AIs, is the ability to do a lot of things with voice now, to be able to communicate di

Mike Kelly, CEO of NervGen Pharma, discusses the company's vision to build a startup biotech focused on repairing the central nervous system, helping patients regain function, not just slowing progression. Their first product, NVG-291, is currently in clinical trials for spinal cord injury. The drug candidate aims to promote natural repair by targeting molecules called CSPGs that inhibit nerve growth and repair. NerveGen is also developing NVG-300 for other indications such as stroke, ALS, MS, and Alzheimer's. He draws parallels between his previous experience developing the Narcan Nasal Spray for opioid overdose and the unmet medical need in spinal cord injury. Mike explains, "We are a company focusing on a group of products, more like a platform, that will enable the nervous system to repair itself. So the vision is to build a startup biotech focused in CNS and to get our first product over the line in spinal cord injury." "There are no drugs available. So this is something that happens in an instant and s

Matt Cronin, Founding Partner of House of Kaizen, discusses how patients' changing healthcare expectations are shaped by their experiences with products and services such as Netflix and Amazon Prime. Consumers now expect a longer-term relationship and personalized experiences with their healthcare providers. House of Kaizen emphasizes the importance of building better customer experiences in health and wellness to improve health outcomes and create more value for businesses. In the age of AI, the importance of trust and brand reputation is paramount. Healthcare providers, as the trusted source of information and a filter for web-based searches, play a crucial role in this landscape.  Matt elaborates, "We're a specialized consulting firm for recurring revenue products and services. We build loyalty and advocacy for better lifetime value through customer experience optimization. We do this for all kinds of businesses that operate on a recurring revenue model, which includes many products and services in the h

Dr. Mollie Leoni, Executive Vice President for Clinical Development of Kura Oncology, discusses the company's ziftomenib program, a menin inhibitor for acute myeloid leukemia (AML). The company initially allowed all AML patients to participate in the clinical study, but later found that specific subtypes, such as those with NPM1 mutation or KMT2A rearrangement, were more likely to respond to the menin inhibitor. They also discovered other subtypes that were responsive, expanding the potential patient population. Understanding the role of menin and menin inhibitors in addressing abnormal gene expression and promoting healthy cell development opens the door to potential combination therapy, where menin inhibitors could be layered onto existing treatments for various cancers related to menin independence. Mollie explains, "In oncology, we have gotten good at treating the end state, but cancer has many, many causes with a common final endpoint to the way the cells look. So, it's many, many different diseases al

Dr. Steffen-Sebastian Bolz, Chief Scientific Officer of Swiss-based Aphaia Pharma, shines a light on the excitement around weight loss drugs due to the discovery that GLP-1 agonists and points out the shift in the perception of obesity as a root cause of various health issues. Aphaia Pharma's approach involves stimulating hormone production in the small intestine by reinvigorating the mechanism that has become dormant due to the ingestion of highly refined foods. The company is also exploring the effects of the circadian rhythm on weight loss and maintaining a healthy weight.   Steffen elaborates, "What is understood now is obesity is the root cause of many sequel pathologies like type 2 diabetes, diabetes, fatty liver disease, and a whole lot of cardiovascular disease. So, the idea is that if we get obesity under control and the obesity pandemic under control, we might also lower the incidence of all the other diseases. There’s a massive rush into this field because there are already blockbuster drugs in th

Rick Vanzura, CEO of Freight Farms, and Dr. Amber Orman, Chief Wellness Officer at Advent Health, discuss their partnership in bringing hyperlocal, sustainable food to healthcare facilities. Freight Farms uses modified shipping containers to grow crops, allowing for year-round production with minimal land and water usage. Advent Health recognized the importance of nutrition in whole-person care and saw the opportunity to provide their team members, physicians, patients, and the public with fresh, nutritious food and donate to local food pantries. The partnership has demonstrated the power of food as medicine and the potential for sustainable farming practices in healthcare settings.   Rick explains, "We grow about two and a half to three acres worth of crops out of an 8 by 40-foot shipping container. It looks like any shipping container you would see on a dock or a boat, but it's highly modified to grow food using LED lights, airflow, nutrients delivered through the farm, and controlled through software. It

Guang Qu, Co-Founder of NGGT Corporation is focused on developing gene therapy products using dual functional vector strategies. Guang highlights the importance of distinguishing between recessive and dominant mutations in rare diseases and explains how their approach differs from other gene therapy approaches. The two leading indications caused by a gene mutation that NGGT is working on are Bietti's Crystalline Dystrophy (BCD), a rare ocular disease, and phenylketonuria (PKU), a metabolic disease.   Guang explains, "For our strategies and our product development strategies, I think we are leveraging our experience in the gene therapy field. Later, I will talk more about myself, Dr. Lixin Jiang, and our team. The other thing we are leveraging is our fully integrated team in gene therapy product development, which involves R&D, research and development, CGMP manufacturers, from tox development and clinical regulatory functions and the medical teams. So with all of the teams we built up in the last couple o

Jennifer Jones-McMeans, divisional vice president of global clinical affairs at Abbott’s vascular business, and Dr. Brian DeRubertis, a doctor at the New York-Presbyterian Weill Cornell Medical Center, shine a light on peripheral artery disease and critical limb-threatening ischemia where artery blockage restricts blood flow to the lower legs. Abbott has developed the Esprit, a dissolving scaffold that opens up the artery in the blocked area and elutes a drug that slows disease progression. Their LIFE-BTK trial demonstrated success in a diverse patient population and is a significant new treatment option for CLTI.  Brian explains, "One of the things that I’ll say is our group is starting to explore doing additional efforts at patient education with our own patients, patients who have actually been diagnosed with this disease, and educating them a little bit more about what it means to have this disease, how they can be involved in the process of trying to prevent their disease from getting worse. And the rea

Marcio Souza, President and CEO of Praxis Precision Medicines, discusses the challenges in treating central nervous system disorders and is focused on developing life-altering treatments for patients with essential tremor.  Current treatments for epilepsy are often broad-based and not specific to the underlying cause of the disease. Praxis is using insights about genetic epilepsy to understand the underlying imbalances in the brain and develop a precision medicine approach to improve movement disorders.   Marcio explains, "For example, we have four clinical programs right now, four different molecules in the clinic, and many more to come in the future, but four at this given point in time. And one of them is for a very common movement disorder called the essential tremor, which is very common, about 2% of the US population develop this condition. And there is no real understanding of where it comes from. Incredibly debilitating for all the people living with essential tremor. But when you take a step back,

Dr. John Blair, CEO of MedAllies, was created to address the lack of communication and coordination between healthcare providers and the importance of interoperability in healthcare. While healthcare has become more complicated over the years, with advancements in technology and automation, the lack of reimbursement for coordination of care has contributed to the problem of lack of interoperability. There are incentives and penalties associated with interoperability, including information blocking and the Trust Exchange Framework and Common Agreement (TEFCA) initiative, which aims to standardize interoperability platforms and reduce barriers to entry for healthcare organizations. John explains, "But if you look at hospitals, you've got about 70% of the hospitals in the country connected to a national network. That means there's 30% that are not. And then, if you start to break that down and look at rural areas versus urban, there's a big disconnect. These smaller rural hospitals tend to be far less connected

Dave Mehalick, Chairman, Co-Founder, President, and CEO of Coeptis Therapeutics, focuses on developing cell therapies for oncology and respiratory diseases. They aim to create a universal cell therapy that can be used for various types of cancer and move it from a later line of treatment to a frontline therapy. The company has developed a unique CAR therapy called SNAP-CAR, which utilizes off-the-shelf cells and a targeting mechanism to attack cancer cells at different points, making it difficult for the cells to mutate and evade treatment. Coeptis is conducting clinical trials using its cell generation platform, including a trial for COVID-19 patients and a trial for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) patients. Dave explains, "Coeptis is a cell therapy company mainly focused on oncology, although we have recently broadened into respiratory disease. And really, the prime mission of the company would be to what we would term universalized cell therapy to make it available for the

Ross Bundy, President and CEO of CRISPR QC, shines a light on the current state of CRISPR gene editing technology and how the field has advanced rapidly since the discovery of CRISPR's potential to edit the human genome. The focus of CRISPR technology has primarily been on curing rare and genetic diseases, but there is also potential for targeting cancer tumors' resistance to chemotherapy and the development of customized therapies for various indications. The services offered by CRISPR QC include a unique sensor platform and data analytics capability to monitor and control the gene editing process. The company aims to create a database of information to improve the understanding and control of CRISPR chemistry. The CRISPR Chip is a new semiconductor technology that integrates carbon electronics to analyze complex biological processes. Ross explains, "What we find is that it can be very difficult to get that chemistry to target exactly in the right place. We don’t know yet how much the right amount is. Is i

Nicolas Poirier, CEO of OSE Immunotherapeutics, uses immunology and molecular engineering to develop innovative immunotherapies for oncology, inflammation, autoimmune diseases, and organ transplant.  The same immune cells, lymphocytes and macrophages, are involved in both immuno-oncology and immuno-inflammation, and OSE aims to re-educate these cells to recognize and eliminate cancer cells or restore immune tolerance in autoimmune diseases. He also discusses the potential of cancer vaccines in re-educating the immune system to target cancer cells, and the importance of combination therapies in improving treatment outcomes. Nicolas explains, "There are two big families in the immune system. We have the adaptive immunity generated by lymphocytes. We have several types of lymphocytes: T-lymphocytes, B-lymphocytes, and so on. This is a very specific immune response that humans and other species have developed to recognize, very specifically some antigens from viruses, for tumors, and so on. We can control these

Ben Casavant, Co-Founder and CEO of Tasso, focuses on making diagnostics more accessible to patients that allow collection of blood samples at home. Their customers include pharmaceutical companies conducting research and healthcare providers. Using Tasso's technology in decentralized clinical trials allows for easier recruitment, increased patient participation, and more data collection. The simplicity and ease of use of Tasso's blood collection device have resulted in positive patient responses and improved retention rates in clinical trials. They offer liquid and dried blood collection options to accommodate different lab processes and sample types.   Ben explains, "When we think about the customer bases we're serving, we're working with many different pharmaceutical companies conducting critical and life-changing research. We just announced a partnership with the Parkinson's Foundation so they can help find ways to gather information and data about patients living with Parkinson's disease. But they need

Chris Darland, President and CEO of Peerbridge Health, discusses technology breakthroughs that enable early, low-cost detection of heart issues with a cardiac monitor that uses AI to analyze heart rhythms over time to detect patterns that may indicate heart failure. The device is based on ECG technology that drives the Holter monitor and delivers a full 12-lead ECG with a volumetric 3D view of the heart. The goal is to reach more patients who may not proactively seek medical attention and provide early intervention and treatment.  Chris explains, " I think the problem with the current state is largely that it's too late. And I say that to mean that 80% of all heart failure cases today are getting caught after an emergency room visit. So, it's not necessarily that we don't have the tools to find heart failure and the cardiologists and expertise to identify it, but we're not getting it in time. So if you are one of those patients who, let's say, are caught earlier or maybe unfortunately when you come through

Dr. David Stein, CEO and Director of Babson Diagnostics, a healthcare technology company that focuses on putting the patient at the center of the blood testing experience, recognizing the importance of blood testing in informing clinical decisions and monitoring health. They aim to redefine blood testing by eliminating the anxiety and inconvenience associated with traditional blood testing methods by offering a more accessible and convenient approach. Blood samples are collected with an innovative device from the patient's fingertip at retail pharmacy locations, and patients use a digital app and QR code system to order and track tests and receive test results. David explains, "Babson Diagnostics is a science-first healthcare technology company that has reimagined the end-to-end blood testing experience. In that experience, the patient and customer are the strong focus. We've all experienced times when we've had a blood test ordered for us, and we’ve felt anxious. We've tried, as much as we can, to eliminate

Joan Butters, CEO and Co-Founder of Xsolis works with health systems and health plans to automate medical necessity decisions and improve efficiency. Using data and AI, Xsolis helps clinicians make more informed decisions and reduces manual administrative work, resulting in time and cost savings and improved patient care. Addressing the challenges and pushback faced when introducing AI into healthcare, Joan emphasizes the importance of understanding the limitations and complexities of generative AI and the need for subject matter expertise in developing prompts to properly address friction points between providers and payers in the healthcare environment. Joan explains, "More important to this audience are the administrative aspects and the impact of that back and forth on the cost of healthcare. We were very fortunate that we found a niche in what we were trying to solve for. Again, that was the medical necessity decision whether a patient required hospital-based care. And then, with that laser focus, we d

Sanjeev Redkar, Co-Founder, Executive Director, and President of Apollomics, focuses on difficult-to-treat cancers, such as lung cancer, brain cancer, and leukemias, not served by immune checkpoint inhibitors. Their approach targets the thematic pathway in cancer cells responsible for their growth and proliferation. In their drug development, AI and data analysis drive precision-targeted therapies based on tumor molecular profiling. They are developing the drugs Vebreltinib and Uproleselan, which can be used as standalone treatments or in combination with other therapies to treat rare cancers. Sanjeev explains, "There's a pathway that is very important for the growth of cancer cells. This is proliferation, motility, migration, and invasion of cancer cells, and that's the thematic pathway. This is an important receptor, which is upstream in cell signaling of these cancer cells. This is when these pathways work well, these cells grow as normal homeostasis, normal growth of cells. When this pathway is disrupted

Kristen Helton, CEO and Co-Founder of Herself Health, provides comprehensive primary care designed specifically for women over 65, considering the unique health challenges these women face. The emphasis is on the importance of addressing women's health beyond reproductive health and the need for tailored treatment and screening for conditions that affect women differently than men. Kristen notes the underrepresentation of women, especially older women, in clinical trials and the need for further investigation. They aim to reduce bias and improve the quality of care for older women by adopting a person-centric approach, listening to patients' concerns, and building trust. Kristen explains, "Almost half of the burdens of disease that women experience stem from conditions that affect women differently than men. So, a lot of these diseases, cardiovascular, chronic illness, gastroenterology, and depression, affect men differently than they affect women. We're very focused on how we treat women and screen them for