Alicia Silver, senior director at ADVI Health, highlights the evolving landscape of cell and gene therapy and the need to improve patient access and payment for these treatments. Availability of these therapies for solid tumors and genetic diseases like sickle cell disease is expanding due to the transition from inpatient to outpatient and community settings. The FDA's decision to remove REMS requirements for specific therapies has accelerated the growth of facilities to provide care, particularly for vulnerable populations.

Alicia explains, "We work with a number of different cell and gene therapy clients throughout the sector. So we work with manufacturers who have commercialized cell and gene therapy products. So they have products that are currently on the market, manufacturers who are going through the process of clinical trials right now, working with the FDA to get approved products. But we also work with trade organizations that are working behind the scenes at the sector level, trying to get different policies and access changes for patients."

"To date, there's probably close to a couple of dozen FDA-approved cell and gene therapies, and they treat everything from blood cancers, which were the first approvals in something called CAR T. We saw blood cancers as the first approvals, and then everything through to solid tumors in oncology. But also, we have newer gene therapies for conditions like sickle cell disease. And that's an area that's been incredibly underserved and definitely will benefit from a durable gene therapy that hopefully corrects some of the issues that patients with sickle cell disease have, like pain crises that end up in a hospital. So from that perspective, we see a really wide range of treatments available to patients today and many more on the horizon."

"I think the price tag is definitely somewhat of sticker shock for people who don't understand how cell and gene therapy products are valued. And so what we do a lot of times, educating on, is helping payers understand that it's not necessarily $2 million for a treatment that's going to be a recurrent payment, but something that's kind of an investment in the patient's and the plan's future."

#ADVIHealth #CellTherapy #GeneTherapy #AcesstoCellGeneTherapy #ClinicalTrials

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Dr. Stacy Lindborg, President and CEO of IMUNON, has developed a DNA-based immunotherapy candidate for the treatment of ovarian cancer. This therapy works by administering the drug, which utilizes IL-12, a powerful anti-cancer cytokine, directly into the cavity where the cancer resides, thereby affecting the tumor microenvironment. Trials are showing that the drug can produce significant life extension when used in combination with standard chemotherapy.

Stacy explains, "There are about 300,000 women who are newly diagnosed with advanced ovarian cancer every year, about 20,000 in the US, and the frontline standard of care hasn't seen a change in the treatment in about 25 years. So this is for newly diagnosed women. The very first treatment that they would have, which we call frontline treatment, is a platinum-based chemotherapy. So carboplatin and Paclitaxel are both administered through IV over an hour for carboplatin, about three hours for Paclitaxel. So most women go directly to chemotherapy and then surgery, and then chemotherapy. Some women will go straight to surgery and then have this chemotherapy afterwards."

"Our approach is a DNA-based immunotherapy candidate that we have in Phase 3. We refer to the lead candidate as IMNN-001, and this is a non-viral nanoparticle that is administered directly into the cavity of interest. So, what we call the micro-tumor environment is the peritoneal cavity, where the cancer actually resides, and it's delivered through a catheter. And it basically has a very powerful anti-cancer fighting cytokine, IL-12, that is encoded in this immunotherapy. And it causes the cells that are within each woman's body, both cancer and non-cancer alike, to start producing activities that will help fight these complex cancer cells that exist."

#IMUNON #OvarianCancer #DNABasedImmunotherapy #WomensHealth #CancerAwareness

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Donovan Campbell, CEO of Medbridge, empowers clinicians through a platform that is focused on movement-based medicine and a holistic approach to patient care. MedBridge has developed AI tools to perform triage, act as a care coordinator, and serve as a patient-facing coach, offloading administrative tasks and providing better support to patients. These tools are designed to support clinicians by flagging potential issues and increasing patient engagement by delivering immediate digital access to care.

Donovan explains, "We serve primarily physical therapists. And those physical therapists can work either in what's called a private practice outpatient setting or in a hospital setting, whether that's inpatient rehab or outpatient rehab. We also service physical therapy and adjacent practitioners, such as athletic trainers, speech and language pathologists, or occupational therapists. With the release of our new hybrid care platform, we're moving into different care categories, which is really exciting. So we're increasingly working with advanced primary care companies that service the employer health market. That's exciting. And we're exploring other opportunities within women's health and within orthopedics."

"Movement-based medicine is relevant to a really holistic approach to patient care, and it spans multiple clinical specialties. So think about the elderly patient who's just had a right knee replacement. Movement-based medicine is critical to getting them back up and on their feet. For a woman postpartum, pelvic health and movement-based medicine are really important. And pelvic health, movement-based medicine that she can access at home, is really important in her recovery and meeting her where she's at in her phase of life. And then physical therapy obviously is all about movement-based medicine."

#Medbridge #DigitalHealth #HealthcareAI #FutureAI #MovementBasedMedicine

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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

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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

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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

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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

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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

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