Podcasts about Cell therapy

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a compelling array of advancements and strategic shifts that are shaping the healthcare landscape across the globe.In recent times, the pharmaceutical and biotech sectors have showcased remarkable resilience and innovation, driving forward with significant scientific breakthroughs and clinical trial results. A standout achievement comes from Novo Nordisk, whose recent Phase 2 trial results for its triple agonist targeting obesity reported a remarkable weight loss of up to 19.7% in patients over 24 weeks. This promising development positions Novo Nordisk as a formidable contender in the obesity treatment market, potentially affecting giants like Eli Lilly. With obesity being a significant global health challenge, these findings underscore the potential of multi-targeted approaches in managing this complex condition.Regulatory landscapes continue to evolve, with pivotal approvals marking milestones for therapies targeting rare diseases. Immedica Pharma's Loargys received FDA approval for treating hyperargininemia associated with arginase 1 deficiency, highlighting perseverance in overcoming regulatory hurdles after a prior rejection. Additionally, Sanofi and Regeneron's Dupixent achieved its ninth FDA approval, underscoring its versatile potential across multiple indications. These approvals not only reflect regulatory progress but also emphasize the critical role of persistence in drug development.Ethical considerations remain at the forefront of industry discussions, particularly highlighted by Novartis' settlement in a lawsuit concerning the use of Henrietta Lacks' cells without consent. This resolution underscores ongoing ethical challenges within biomedical research, emphasizing the need for ethical vigilance as companies increasingly rely on human-derived materials.Significant business trends are shaping strategic directions within the industry. Pfizer's acquisition of marketing rights for Sciwind's GLP-1 receptor agonist in China exemplifies a calculated move to dominate the obesity treatment market. This strategic acquisition allows Pfizer to leverage China's vast market potential for type 2 diabetes medications and positions it favorably for future weight loss treatments.On the manufacturing front, AbbVie has made substantial investments in U.S. infrastructure, committing $380 million to new North Chicago API plants as part of a decade-long strategy to inject $100 billion into U.S. operations. This initiative highlights a commitment to bolstering domestic production capabilities amidst global supply chain uncertainties.The complexities of drug development are further illustrated by Roche's decision to halt the development of Enspryng for Duchenne muscular dystrophy due to unsatisfactory progress. This shift in focus reflects the inherent challenges of drug repurposing and the necessity of robust clinical evidence to support new indications.Geopolitical factors also play a significant role in shaping industry dynamics, with recent U.S. Supreme Court decisions impacting international trade agreements. Such geopolitical influences can significantly affect pharmaceutical companies' operations and strategic planning.The collaboration between Astellas and Vir Biotechnology reflects another significant trend in strategic partnerships within the industry. Their $1.7 billion deal centered on a novel bispecific T-cell engager for prostate cancer underscores the growing importance of immuno-oncology and innovative approaches to targeting hard-to-treat cancers.The regulatory front continues to see transformative changes with the FDA unveiling draft guidance for a new approval pathway tailored for bespoke gene-editing therapies. This initiative could expedite personalized genetic treatments and transform patSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into a wide array of topics, from groundbreaking therapies and strategic corporate moves to regulatory shifts and industry trends shaping the future of healthcare.We begin with Eli Lilly, which is making significant strides with its combined Zepbound and Taltz therapy, showing promising results in the treatment of psoriasis and obesity. This combination therapy, initially successful in psoriatic arthritis, is set to transform treatment protocols by bridging gaps between psoriasis and obesity. This dual-targeting approach highlights a burgeoning trend in the industry: the use of combination therapies to enhance patient outcomes while streamlining treatment regimens. This strategy not only promises better management of interconnected conditions but also reflects a broader industry goal of maximizing therapeutic reach with existing drugs.Novartis is advancing its portfolio with the oral BTK inhibitor, Rhapsido, following a successful Phase 3 trial for a new chronic hives subtype. This development underscores Novartis's commitment to addressing niche markets and unmet medical needs, emphasizing the pharmaceutical industry's focus on expanding the utility of existing drugs. Additionally, Novartis has entered into a notable partnership with Macrocycle Biotech for cardiovascular drug development. This collaboration represents a broader trend where large pharmaceutical companies seek innovative partnerships to expand their therapeutic portfolios through cutting-edge biotech solutions.Meanwhile, Moderna's influenza vaccine submission has been accepted by the FDA after an initial rejection, illustrating a responsive regulatory environment crucial for timely access to vaccines amid potential flu outbreaks.Johnson & Johnson's $1 billion investment plan in the U.S., focusing on cell therapy, aligns with broader industry trends towards personalized medicine and advanced therapeutic approaches. This investment is part of a strategic pivot towards cell and gene therapies that promise to redefine treatment pathways for complex diseases. Similarly, Bayer's $7.25 billion settlement over Roundup litigation shows an industry keen on resolving legal challenges swiftly to refocus efforts on innovation.Regulatory reforms are also gaining attention, with proposals aimed at streamlining FDA processes to enhance drug access and reduce burdens. These reforms could significantly impact drug development timelines and market entry strategies, reflecting an ongoing discourse on balancing regulation with fostering innovation.On a global scale, Stada's €85 million investment in Saudi Arabia points to a strategic move towards enhancing supply chain resilience in the Middle East and North Africa. This aligns with industry trends focusing on regional manufacturing capabilities to ensure drug availability while reducing logistical complexities.A notable example of strategic resource allocation is Eli Lilly's $100 million upfront payment for CSL's IL-6 antibody development rights. This reflects an adaptive approach where initial clinical setbacks are seen as opportunities for new therapeutic ventures rather than dead ends.Turning our attention to Alzheimer's research, Korsana Biosciences has emerged from stealth mode with substantial funding aimed at developing an anti-amyloid antibody capable of crossing the blood-brain barrier. This effort addresses a critical need within Alzheimer's disease treatment—a field marked by intense competition and scientific challenge. Parallelly, IQVIA Biotech highlights the importance of accelerating early-stage interventions for Alzheimer's through data analytics and optimized clinical trials. Such efforts aim to expedite regulatory approvals and bring new therapies to market faster—a trend indicative of leveragSupport the show

At the 2026 Tandem Meetings, CancerNetwork® spoke with a variety of experts who presented on key developments and advancements across hematologic oncology. As part of different oral presentations and poster sessions, researchers and clinicians shared updated findings that may influence the management of myelodysplastic syndromes (MDS), leukemia, lymphoma, and other blood cancer types.First, Fernando Duarte, head of the Bone Marrow Transplant Service at Walter Cantídio University Hospital (HUWC), hematologist and professor at the Federal University of Ceará, and president of the Brazilian Society of Cell Therapy and Bone Marrow Transplant, highlighted his presentation analyzing trends associated with allogenic hematopoietic cell transplantation (allo-HCT) among patients with MDS or myeloproliferative neoplasms (MPN) and other types of MDS. Data from the Brazillian SBTMO and CIBMTR registry revealed that patients receiving allo-HCT for MDS/MPN were typically older with worse performance statuses. Additionally, MDS/MPN independently predicted worse overall survival (OS) and relapse-free survival outcomes.Next, Alfonso Molina, MD, MPH, a third-year Hematology and Medical Oncology fellow at Stanford University, detailed results from a phase 1 trial (NCT05507827) assessing Orca-T, an investigational allogeneic T-cell immunotherapy, among those with high-risk B-cell acute lymphoblastic leukemia (B-ALL). Treatment with Orca-T yielded disease-free survival and OS in all (100%) 18 evaluable patients after a median follow-up of 14 months (range, 3-35), which occurred without graft failure, significant graft-versus-host-disease, or severe CAR-mediated toxicity.Finally, Irtiza N. Sheikh, DO, an assistant professor in the Department of Pediatrics - Patient Care, Stem Cell Transplantation and Cellular Therapy Section of the Division of Pediatrics at The University of Texas MD Anderson Cancer Center, discussed his presentation exploring differences in outcomes with lisocabtagene maraleucel (Breyanzi; liso-cel) across various treatment settings and patient populations with large B-cell lymphoma. Data demonstrated that among patients younger than 50 years old, liso-cel produced enduring responses across real-world and clinical trial settings, which were comparable to outcomes in overall populations. References Duarte FB, Garcia YDO, Hamerschlak N, et al. Comparative outcomes of allogeneic hematopoietic cell transplantation in myelodysplastic/myeloproliferative neoplasms and other myelodysplastic syndromes: Brazilian Sbtmo/CIBMTR registry analysis. Presented at: 2026 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; February 4-7, 2026; Salt Lake City, UT. Presentation 63. Molina A, Shiraz A, Kanegai A, et al. Mature outcomes from the phase I trial of Orca-T and allogeneic CD19/CD22 CAR-T cells for adults with high-risk B-ALL. Presented at: 2026 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; February 4-7, 2026; Salt Lake City, UT. Presentation 31. Sheikh IN, Patel K, Perales MA, et al. Clinical outcomes of lisocabtagene maraleucel (liso-cel) in YOUNGER PATIENTS (Pts) with relapsed or refractory (R/R) large B-cell lymphoma (LBCL). Presented at: 2026 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR; February 4-7, 2026; Salt Lake City, UT. Poster 210.

In this episode of Plugged Into Public Health, Lauren speaks with Dr. Jill Kolesar about ovarian cancer, precision medicine, and the challenges of translating scientific discovery into real-world care. Dr. Kolesar explains why ovarian cancer remains difficult to treat, including late-stage diagnosis and limited response to immunotherapy. She shares her team's work on a novel cell-based therapy designed to convert “cold” tumors into ones the immune system can attack, with the goal of improving effectiveness while reducing toxicity. The conversation also explores molecular tumor boards, the role of pharmacists in precision oncology, and how collaboration and data sharing help bring innovation to community and rural settings. This episode highlights not only cutting-edge science, but the systems required to ensure that breakthroughs actually reach patients. A transcript of this episode will be available here soon. Have a question for our podcast crew or an idea for an episode? You can email them at CPH-GradAmbassador@uiowa.edu You can also support Plugged in to Public Health by sharing this episode and others with your friends, colleagues, and social networks. #publichealth #cancer #cancercare #ovariancancer #precisionmedicine #immunotherapy #healthcare #pharmacology #medicaltechnology #iowacity

 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 Listen to the podcast here

 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 Download the transcript here

Synopsis: At the heart of JPM 2026's biotech buzz, Alok Tayi sits down with Fred Aslan, CEO of Artiva, to explore how bold platform bets, scalable cell therapies, and autoimmune breakthroughs could reshape medicine. Fred traces his journey from medical school in Brazil to consulting at BCG, venture capital, and ultimately founding multiple companies—sharing why following curiosity, not rigid career ladders, shaped his path. Fred dives deep into the bottlenecks holding back traditional CAR-T therapies—manufacturing complexity, cost, hospitalization, and toxicity—and explains how Artiva's off-the-shelf NK-cell platform aims to change the paradigm. The discussion explores why rheumatoid arthritis became Artiva's lead indication, how immune “resets” could redefine autoimmune care, and what's ahead in 2026 as the company prepares registrational trials and expands its basket studies across lupus, myositis, scleroderma, and more. The episode closes with rapid-fire takes on AI in drug development, China's accelerating biotech engine, rare disease trial models, and the strategic principles founders should follow when choosing indications and building durable platforms. Biography: Fred Aslan, M.D., has a 20-year track record as an executive and investor in the life sciences industry. He was most recently President and CBO at Vividion Therapeutics, where he was responsible for business development, finance, alliance and project management, and operations. Dr. Aslan had the opportunity to lead Vividion's Series B financing and $135M-upfront collaboration with Roche. Prior to Vividion, Dr. Aslan had a 12-year affiliation with Venrock. Initially he was an investor from 2006 to 2013, when he cofounded and served as a board member of Receptos Pharmaceuticals (acquired by Celgene for more than $7 billion). Dr. Aslan led Venrock's investment in Zeltiq (acquired by Allergan for more than $2 billion) and was involved in the early formation of Fate Therapeutics. Subsequently as an entrepreneur from 2013 to 2018, he was CEO of Adavium Medical, a Brazilian medical device company, which he grew from zero to 350 employees, sales of over US$40 million, and fully integrated R&D, manufacturing, and commercial capabilities. Prior to Venrock, Dr. Aslan was Director of Business Development and Head of Investor Relations for CuraGen, a Nasdaq-listed oncology-focused biotech company. Prior to CuraGen, he was a consultant at Boston Consulting Group (BCG). Dr. Aslan holds a B.S. in biology from Duke University, an M.D. from Yale School of Medicine, and an MBA from Harvard Business School.

How do you build an organization that can absorb change, learn from failure, and keep patients at the center—even when the science is uncertain? Nelly Viseux shares lessons from over 20 years in biotech and a decade leading cell and gene therapy development.Nelly discusses how she structured a 100-person CMC organization at Regeneron to balance innovation with operational execution, why documenting your assumptions is critical to managing risk, and what it really takes to maintain resilience when you're literally holding patient lives in your hands.A few of Nelly's key takeaways:Resilience is adaptability—build organizations that absorb and anticipate change rather than resist itSeparate innovation from execution with intentional gates for when new approaches are ready to implementDocument your assumptions so you can revisit decisions effectively when circumstances changeFailure is a process problem, not a personal one—root cause analysis should improve systems, not assign blameData is the common language that aligns scientists, regulators, and stakeholdersEveryone is a leader in cell therapy—manufacturing and QC teams hold patient lives in their handsAbout Nelly ViseuxNelly Viseux is Vice President of Cell Therapies Development, Manufacturing, Supply & Quality at Regeneron, leading a 100-person organization supporting autologous cell therapy programs. She has over 20 years of biotechnology experience spanning large pharma (Shire, Biogen, Baxter) and startups, working across cell and gene therapies, biologics, and nanoparticles. Her accomplishments include building a Phase 1 cell therapy manufacturing facility that achieved 100% cGMP success and first IND submission within two years. She holds a Ph.D. in Biochemistry and Molecular Biology from University of Lille and is a member of the Society for Immunotherapy of Cancer and the American Society of Gene & Cell Therapy.About The FDA GroupThe FDA Group helps life science organizations rapidly access the industry's best consultants, contractors, and candidates. Our resources assist in every stage of the product lifecycle—from clinical development to commercialization—with a focus on staff augmentation, auditing, remediation, QMS, and other specialized project work in Quality Assurance, Regulatory Affairs, and Clinical Operations. Learn more: ⁠⁠https://www.thefdagroup.com/

Tim Lu, CEO of Senti Bio, joins In Vivo to discuss how programmable cell therapies are solving oncology's targeting problem. Lu explains the logic-gated approach behind SENTI-202, an allogeneic CAR-NK therapy for relapsed/refractory AML that achieved 50% response rates in Phase 1 while avoiding the dose-limiting toxicities that have plagued other AML cell therapies. We cover the Phase 1 ASH 2025 data showing 39% complete remission rates (all MRD-negative) with 7.6-month median duration, the rationale for using NK cells over T cells, and why synthetic biology's three-target logic gates can distinguish cancer from healthy bone marrow cells. Lu also discusses plans for pivotal trials following RMAT designation, expansion into solid tumors, and where biotech innovation is accelerating versus where clinical translation bottlenecks remain. For biopharma professionals tracking cell therapy innovation, synthetic biology applications, and AML treatment advances.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. This morning, we're diving into a series of fascinating updates that underline the vibrant and ever-changing landscape of our industry.The J.P. Morgan Healthcare Conference recently set the stage for some intriguing discussions, particularly from Novo Nordisk. The company is diversifying its metabolic drug development portfolio by exploring innovative avenues, reflecting a broader industry trend where firms seek to balance their core expertise with novel therapeutic areas. This strategic diversification is crucial as companies aim to address complex health issues with an expansive approach to innovation. Novo Nordisk's leadership emphasized this strategic pivot towards diversifying their innovation pipeline beyond traditional metabolic disorders, aiming to keep the company at the forefront of pharmaceutical advancements.Meanwhile, Takeda's R&D head, Andy Plump, brought attention to some challenges currently facing U.S. innovation. Despite a sluggish start at the conference, recent months have been buzzing with significant deal-making activities. This scenario highlights the delicate dance between maintaining steady innovation and navigating regulatory hurdles and economic pressures. However, optimism remains high, with strategic investments and collaborations seen as potential catalysts for rejuvenation in research.Amgen made waves by presenting promising results from an exploratory study of Maritide in weight loss maintenance. The study's outcomes signal a significant milestone in obesity management and set the stage for further clinical trials and potential approval processes. This advancement offers hope in addressing what remains a critical public health issue worldwide.A significant development at the National Institutes of Health (NIH) is the leadership change as Dr. Gary Gibbons steps down as Director of the Heart, Lung, and Blood Institute. This shift adds to a growing list of interim leadership roles across NIH's 27 institutes and centers, which could impact continuity in critical research projects and funding initiatives.On the corporate front, Sonoma Pharmaceuticals announced notable workforce reductions due to financial challenges, while Lyra Therapeutics decided to abandon its rhinosinusitis treatment project alongside laying off its entire team. These decisions highlight the financial volatility that smaller biotech firms face amid competitive pressures and regulatory complexities. In contrast, Pretzel Therapeutics has emerged successfully from turbulent times, demonstrating resilience and adaptability within the biotech sector. Strategic restructuring and leadership realignment have positioned Pretzel Therapeutics for future growth in therapeutic development.A major highlight involves AbbVie entering into an agreement with the White House to reduce Medicaid drug prices while committing a substantial $100 billion investment in U.S. R&D over the next decade. This aligns with broader efforts to make healthcare more affordable while encouraging domestic pharmaceutical investment. AbbVie's substantial commitment towards U.S. R&D speaks volumes about their strategy within TrumpRx program contexts that aim at enhancing market penetration while balancing innovation investment against cost management pressures.Medtronic has expressed readiness to engage in mergers and acquisitions, indicating that medtech companies are poised for expansion through strategic buyouts. This move reflects an industry-wide capacity for growth through consolidation and collaboration. As we look towards mergers and acquisitions within medtech highlighted by Medtronic's intentions, it's clear that strategic M&A activities remain vital for companies seeking to expand capabilities and market presence in this competitive landscape.The FDA has aSupport the show

Pascal Touchon unpacks why the next CAR-T innovations, including allogeneic and in vivo therapies, are going to be so exciting.The cell therapy master outlines his central role in bringing two first‑in‑class cell therapies to patients: Kymriah, the first approved CAR‑T, and Ebvallo, the first approved allogeneic T‑cell therapy. We also explore his new position as an operating partner at Jeito and how we need to work harder to find Europe's hidden biotech gems.-----------------------------------------------------------------------Get expert legal advisory support for growing your biotech from Osborne Clarke at https://bit.ly/oc-flotbio----------------------------------------------------------------------⭐️ ABOUT THE SPEAKERWith many years of experience in big pharma, Pascal had top-level positions at Novartis Oncology. He later served as CEO, and most recently Chairman, of Atara Biotherapeutics. Alongside his Jeito role, he sits on the boards of companies including Ipsen, Medincell, Catalym, Xylocor and CDR-Life.

We love to hear from our listeners. Send us a message. On this week's episode of the Business of Biotech, Nick Manusos, CEO at Kenai Therapeutics, talks about his experiences building cell therapy spinouts from FujiFilm Cellular Dynamics, learning from big pharma decision-making processes, and dosing the first patient with Kenai's allogeneic neuron replacement cell therapy for Parkinson's disease. Nick also talks about funding an early-stage cell therapy company and forging key manufacturing and therapy administration partnerships. Access this and hundreds of episodes of the Business of Biotech videocast under the Business of Biotech tab at lifescienceleader.com. Subscribe to our monthly Business of Biotech newsletter. Get in touch with guest and topic suggestions: ben.comer@lifescienceleader.comFind Ben Comer on LinkedIn: https://www.linkedin.com/in/bencomer/

Send us a textGood morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve deep into a series of transformative events that underscore the dynamic nature of our industry, where scientific innovation meets regulatory evolution and market adaptation.We begin with significant regulatory news from Medicare, which recently announced price reductions for 15 prescription drugs, including Novo Nordisk's semaglutide products, Ozempic and Wegovy. This initiative is part of the Inflation Reduction Act aimed at making essential medications more affordable. By potentially increasing accessibility to these treatments, this move highlights a growing trend towards cost containment in drug pricing within the U.S. healthcare system. It reflects a broader effort to ensure that life-saving treatments remain within reach for more patients, emphasizing the need for balance between innovation and affordability.Turning to approvals, Otsuka has secured FDA clearance for Voyxact, a first-in-class treatment targeting IgA nephropathy (IgAN). This positions Otsuka in an increasingly competitive market space populated by major players like Novartis and Vertex. The entry of Voyxact could pave the way for innovative therapeutics in kidney diseases, offering new hope to patients who have had limited treatment options until now.On the other side of the Atlantic, French authorities have conducted a raid on Sanofi's headquarters as part of a tax fraud investigation. This development sheds light on ongoing scrutiny in the pharmaceutical sector regarding financial practices and regulatory compliance. Such investigations can have far-reaching implications on corporate governance and transparency, reminding us of the importance of ethical practices in maintaining industry trust.Novo Nordisk has strategically used its FDA national priority voucher to expedite the review process for a high-dose formulation of Wegovy. This move underscores the importance of regulatory incentives in accelerating drug development timelines, allowing for quicker patient access to potentially life-changing therapies. It's a testament to how strategic navigation through regulatory pathways can significantly impact drug availability.In clinical trials, Sarepta Therapeutics received FDA clearance to conduct a study combining its gene therapy Elevidys with sirolimus in patients with Duchenne muscular dystrophy. The study aims to address liver safety issues associated with Elevidys, which had led to previous label restrictions. This reflects the industry's commitment to enhancing therapeutic safety profiles while expanding treatment indications.In oncology advancements, AstraZeneca's Imfinzi received FDA approval for use in early-stage stomach cancer, marking its third perioperative indication. This approval underscores the expanding role of immunotherapy across various cancer types and stages, offering new treatment paradigms that could improve surgical outcomes and long-term patient survival.Despite these advances, there is skepticism regarding artificial intelligence's role in regulatory compliance submissions among pharmaceutical professionals. A survey reveals that 65% express distrust towards AI-generated outputs, highlighting challenges that AI technologies face in gaining acceptance within highly regulated environments such as pharmaceuticals. However, federal recommendations to revamp U.S. biotechnology research emphasize incorporating AI into scientific processes to maintain global competitiveness. This call reflects concerns over potential declines in innovation leadership and underscores the need for strategic investment in research infrastructure.In antitrust news, the Federal Trade Commission (FTC) outlined its case agaiSupport the show

Can lost hearing cells be replaced? Lineage Cell Therapeutics CEO Brian Culley returns to discuss the company's new collaboration with William Demant Invest. Together, they're advancing ReSonance™ (ANP1) — an experimental cell therapy designed to replace damaged auditory neurons and potentially restore communication between the ear and the brain.Culley explains how the partnership combines Lineage's regenerative cell technology with the audiology expertise of Eriksholm Research Centre to move this therapy toward first-in-human testing. Backed by up to $12 million in research funding, the multi-year effort could represent a new frontier in treating hearing loss in the future.Be sure to subscribe to our channel for the latest episodes each week and follow This Week in Hearing on LinkedIn, Instagram and X.- https://x.com/WeekinHearing- https://www.instagram.com/thisweekinhearing/- https://www.linkedin.com/company/this-week-in-hearingVisit us at: https://hearinghealthmatters.org/thisweek/

We love to hear from our listeners. Send us a message.Episode 115 of Cell & Gene: The Podcast features Host Erin Harris' talk with Aliya Omer, Vice President and Global Head of Hematology and Cell Therapy at AstraZeneca. Omer shares valuable insights from her rich experience leading cell therapy development across multiple top companies. She highlights the critical importance of collaboration by breaking down silos across research, manufacturing, regulatory, and commercial teams to deliver innovative therapies efficiently. She also discusses AZ's diverse and ambitious cell therapy portfolio, encompassing autologous CAR-T, TCR-T, in vivo gene therapies, and regulatory T-cell therapies. She candidly addresses current challenges in manufacturing scalability, patient access, and healthcare system readiness and describes how AZ is prioritizing fast manufacturing platforms and ecosystem-wide partnerships to surmount these hurdles. Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Novartis started the week early with a Sunday afternoon announcement of the acquisition of neuromuscular drug developer Avidity Biosciences for $12B. That's the second biggest buy of the year after Johnson & Johnson's January acquisition of Intra-Cellular. The Avidity buy could read through positively to Dyne Therapeutics, as both are aiming to treat neuromuscular ailments with RNA-targeting therapies. Dyne shares have nearly doubled over the past month, jumping approximately 40% after Novartis' news dropped.  The Avidity deal is the latest in an uptick on the pharma M&A front. Also this week, Eli Lilly doubled down on gene therapy with a pick up of Adverum Biotechnologies and its lead program for wet age-related macular degeneration. And Roche, which last month acquired 89bio in a $3.5 billion deal centered on a MASH candidate, said in its third-quarter earnings call on Thursday that more deals could be in the future. Finally, beyond the big guys, Leerink Partners predicts which small- to mid-cap firms might also be on the hunt for new pipeline goodies.  Following the dealmaking news, Novartis held its earning call on Tuesday. CEO Vas Narasimhan downplayed the deals Pfizer, AstraZeneca and Amgen have made with the White House, saying they don't address the root of the drug pricing problem President Donald Trump hopes to solve.   On other earnings calls, BioMarin announced plans to divest the hemophilia gene therapy Roctavian. Regeneron faced further questions about Eylea and issues with the Catalent plant that's been tripping up its regulatory applications. But the company didn't address last week's news that it was dropping a CAR T asset picked up from 2seventy bio. These are but two of the latest examples of underperforming assets in the cell and gene therapy space.  BridgeBio had positive news for patients with limb-girdle muscular dystrophy this week after acing a Phase III trial for an investigational substrate supplementation therapy. Analysts predict the asset could be before the FDA later this year or early next.   Finally, with the U.S. government shutdown going on a month, BioSpace takes a look at how the FDA is operating. 

Most founders dream of raising millions. Few survive the 153 “no's” it takes to get there.Behind every biotech breakthrough lies exhaustion — late-night calls, failed rounds, and investors who walk away at the finish line.What separates the ones who make it isn't luck or timing — it's resilience built into process.In this episode, Jason Foster, CEO of Ori Biotech, shares how he transformed relentless rejection into a billion-dollar trajectory. From rebuilding cell-therapy manufacturing to leading global teams through economic storms, Jason reveals how founders can systematize grit, master storytelling, and survive when everything seems to fall apart.You'll learn how to navigate fundraising winters, why leadership begins with self-care, and how to build companies that endure long after the hype fades. If you've ever doubted your path as a builder, this conversation will remind you that resilience is not a trait — it's a practice.

We love to hear from our listeners. Send us a message.On episode 114 of Cell & Gene: The Podcast, Host Erin Harris talks to Brian Culley, CEO of Lineage Cell Therapeutics, about advancing cell therapy beyond oncology and into transformative treatments for conditions such as spinal cord injury and blindness. Culley shares how Lineage's allogeneic, off-the-shelf approach, anchored by its OPC1 program, aims to replace lost or damaged cells to restore function, starting with patients who have plateaued after chronic spinal cord injury. He details the DOSED study's innovative delivery device and thaw-and-inject formulation, both designed to simplify administration, enhance safety, and improve patient access. Harris and Culley also explore Lineage's broader vision for commercial viability in cell transplantation and its collaborative work with the Christopher & Dana Reeve Foundation to drive awareness, research, and investment in spinal cord injury therapies.Subscribe to the podcast!Apple | Spotify | YouTube Visit my website: Cell & Gene Connect with me on LinkedIn

Viral pneumonia can cause severe lung damage and make recovery long and life-threatening for patients. Current treatments only provide support, not repair, but Northwestern Medicine scientist Benjamin Singer, MD, wants to change that. In this episode, he details a recent discovery from his team that found laboratory-modified T-cells or induced regulatory T-cells (iTregs), can promote lung tissue repair in mice with severe influenza pneumonia. Singer discusses this breakthrough and how it could lead to an effective cellular therapy for patients with severe viral pneumonia and acute respiratory distress syndrome in the future. 

It's been a tough year for cell and gene therapy — patient deaths, high-profile companies pulling out of the space, and sour investment sentiment. But the field has had some promising readouts in the clinic recently, and it continues to mature, showing steady progress despite challenging market conditions. On the latest BioCentury This Week podcast, Evotec's Bernd Mühlenweg joins BioCentury's analysts to give his view of the field and offer takeaways from this month's Cell & Gene Meeting on the Mesa in Phoenix. This episode of BioCentury This Week is sponsored by Evotec. View full story: https://www.biocentury.com/article/657278To submit a question to BioCentury's editors, email the BioCentury This Week team at podcasts@biocentury.com.Reach us by sending a text

Johnson & Johnson kicked off Q3 earnings season on Tuesday with the announcement that it is splitting its orthopedics and medtech operations and that it has yet to reach a drug pricing deal with the White House, though CEO Joaquin Duato did say those discussions are ongoing.  In advance of its Nov. 5 earnings call, Novo Nordisk, under the direction of new CEO Maziar Mike Doustdar, has been busy making moves, doubling down on MASH last week with its $5.2 billion buy of Akero. Then this week, Novo became the latest company to cut cell therapy, following Takeda's recent exit from the space. Meanwhile, as the government shutdown continues, so too does the chaos at the CDC, where more than 1,000 employees received termination notices last Friday—only for hundreds to be told never mind. At the FDA, however, there is some consistency despite the overhaul, with the agency on track for an average number of approvals this year. And a recent report on breakthrough designations shows that the regulatory award often leads to an FDA greenlight. On the legislative front, the BIOSECURE Act is back, as a slimmed down version passed the Senate last week as part of the defense spending bill. The latest version of BIOSECURE, which is meant to distance American biopharma from Chinese collaborators, doesn't name specific companies as previous iterations did and must still gain the Senate's support. 

Dr. Peter Altman, President and CEO of BioCardia, is focused on treating ischemic heart failure and chronic myocardial ischemia using autologous cells from the patient's bone marrow.  The company has developed a cost-effective model that utilizes a pre-procedure diagnostic to select the optimal patients for this point-of-care system, which processes their cells in a single procedure. A key technology is the Helix catheter system, which enables minimally invasive delivery of cells into the heart, aided by the Heart3D Fusion imaging platform that provides a 3D map for precise cell delivery. Peter explains, "BioCardia's focus is on ischemic etiology of heart disease. This is heart disease that results from poor perfusion, typically following, for example, a heart attack, which is an ischemic disease. And the two diseases we're working on are ischemic heart failure, which is often characterized by a large dilated heart. And in the second is chronic myocardial ischemia, which is characterized by what's called refractory angina or chest pain that actually can be incredibly severe. And so are our focus areas. And we're advancing cell therapies that have been shown to inherently improve the cells we're looking at, which are cells from the bone marrow space, the mononuclear cells that contain the CD34 and CD133 cell populations. They're actually involved in forming new microvasculature and supply." "What we've done is come at it from a different perspective. Instead of selecting the cells and then manufacturing in a remote facility, we have a pre-procedure diagnostic that enables us to select the patients who have appropriate cells and then process them with a point-of-care cell processing platform, which involves just a simple disposable cartridge. And then we can treat these patients in a standard interventional cardiology setting at relatively low cost compared to all cell therapies."      #BioCardia #HeartDisease #AutologousCellTherapy #CellTherapy #3DImaging #Heart3DFusion #CardiAMP biocardia.com Listen to the podcast here

Dr. Peter Altman, President and CEO of BioCardia, is focused on treating ischemic heart failure and chronic myocardial ischemia using autologous cells from the patient's bone marrow.  The company has developed a cost-effective model that utilizes a pre-procedure diagnostic to select the optimal patients for this point-of-care system, which processes their cells in a single procedure. A key technology is the Helix catheter system, which enables minimally invasive delivery of cells into the heart, aided by the Heart3D Fusion imaging platform that provides a 3D map for precise cell delivery. Peter explains, "BioCardia's focus is on ischemic etiology of heart disease. This is heart disease that results from poor perfusion, typically following, for example, a heart attack, which is an ischemic disease. And the two diseases we're working on are ischemic heart failure, which is often characterized by a large dilated heart. And in the second is chronic myocardial ischemia, which is characterized by what's called refractory angina or chest pain that actually can be incredibly severe. And so are our focus areas. And we're advancing cell therapies that have been shown to inherently improve the cells we're looking at, which are cells from the bone marrow space, the mononuclear cells that contain the CD34 and CD133 cell populations. They're actually involved in forming new microvasculature and supply." "What we've done is come at it from a different perspective. Instead of selecting the cells and then manufacturing in a remote facility, we have a pre-procedure diagnostic that enables us to select the patients who have appropriate cells and then process them with a point-of-care cell processing platform, which involves just a simple disposable cartridge. And then we can treat these patients in a standard interventional cardiology setting at relatively low cost compared to all cell therapies."      #BioCardia #HeartDisease #AutologousCellTherapy #CellTherapy #3DImaging #Heart3DFusion #CardiAMP biocardia.com Download the transcript here

Can regenerative medicine move beyond managing symptoms to actually restore natural hearing?Brian Taylor speaks with Simon Chandler, CEO of Rinri Therapeutics, about the company's pioneering cell therapy for hearing loss. They discuss Rincell-1, Rinri's first-in-human clinical trial, and how regenerative medicine could complement or even surpass today's treatments like hearing aids and cochlear implants.The conversation also explores Rinri's innovative surgical delivery approach, trial design, and future pipeline, including next-generation therapies aimed at restoring hair cells. Chandler shares his vision for how regenerative medicine could reshape hearing care and open the door to restoring hearing for millions worldwide.Learn more about Rinri and Rincell-1 at: https://www.rinri-therapeutics.comBe sure to subscribe to our channel for the latest episodes each week and follow This Week in Hearing on LinkedIn, Instagram and X.- https://x.com/WeekinHearing- https://www.instagram.com/thisweekinhearing/- https://www.linkedin.com/company/this-week-in-hearingVisit us at: https://hearinghealthmatters.org/thisweek/

Cell therapy holds immense potential to transform the way we treat conditions in oncology, ophthalmology, autoimmune disorders, and a wide range of other therapeutic areas. But how do these engineered cells work, and how do scientists ensure they are safe and function as intended? In this episode, co-host Danielle Mandikian sits down with Todd McDevitt, Head of Cell Therapy, to discuss how cell therapies operate within the body, the challenges researchers face in this field, and the advancements needed to unlock their full potential for patients. Read the full text transcript at www.gene.com/stories/cells-at-work

“Once a scientist, forever a scientist,” says Legend Biotech CEO Ying Huang as he joins Bloomberg Intelligence analyst Sam Fazeli to share his unconventional journey from the lab bench to Wall Street and back to biotech leadership. Dr. Huang explains how a dream led him from equity research to developing one of the most important CAR-T therapies for multiple myeloma. They explore the science behind BCMA targeting, lessons from the pivotal Johnson & Johnson partnership as well as the company’s ambitions in allogeneic and in-vivo therapies.See omnystudio.com/listener for privacy information.

In this episode of Idea Collider, host Mike Rea interviews Dr. Christian Rommel from Bayer. Dr. Rommel discusses his journey in molecular oncology from the Max Planck Institute, through roles at Roche, to overseeing global R&D at Bayer. He shares insights on turning scientific discovery into novel medicines, collaboration between scientists and commercial teams, and the importance of maintaining scientific integrity. Dr. Rommel also delves into the impact of AI in drug development, the potential of genetic medicines, and the complexities of launching new medicines on a global scale. The conversation also touches on embracing failure, internal and external partnerships, and the evolving landscape of clinical translation. 00:00 Introduction and Guest Welcome00:25 Christian Rommel's Journey in Oncology03:02 The Importance of Collaboration in Innovation05:16 Balancing Risk and Reward in Drug Development18:07 The Role of AI and Data in Modern R&D22:33 Partnerships and External Learning26:16 Balancing Legacy and Innovation in Biotech27:18 Global Expansion and Leadership Diversity27:27 Courage in Biotech Management27:54 Inspiration from Roche Genentech30:26 Commitment to Product Supply and Market Readiness32:23 Challenges of Global Launches35:53 Emerging Trends in Pharma: AI and Genetic Medicines42:20 Decision-Making in Pharma47:30 Reflections on Academic and Professional Journey Don't forget to Like, Share, Subscribe, Rate, and Review! Keep up with Christian Rommel;LinkedIn: https://www.linkedin.com/in/christian-rommel/Website: https://www.bayer.com/en/innovation/science-research-and-innovation Follow Mike Rea On;Website: https://www.ideapharma.com/X: https://x.com/ideapharmaLinkedIn: https://www.linkedin.com/in/bigidea/ Listen to more fantastic podcast episodes: https://podcast.ideapharma.com/

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Hear about cutting-edge advances in regenerative medicine, from lab breakthroughs to patient impact. Catriona Jamieson, MD, PhD, introduces efforts at the Sanford Stem Cell Institute to develop therapies that enhance the body's ability to heal itself. Dan Kaufman, MD, PhD, shares progress in cancer immunotherapy using engineered natural killer cells derived from pluripotent stem cells. Karen Christman, PhD, explains how her team creates injectable hydrogels from pig heart tissue to support heart repair and regeneration after a heart attack. Tiffani Manolis highlights industry support for making cell and gene therapies more accessible. Patient advocate Justin Graves describes his life-changing experience receiving a stem cell-based therapy for epilepsy, underscoring the real-world promise of these innovations. Series: "Stem Cell Channel" [Health and Medicine] [Science] [Show ID: 40705]

Synopsis: From stargazing in rural Tennessee to reimagining the future of immune health, Kevin Caldwell's journey is anything but conventional. In this episode of Biotech2050, Rahul Chaturvedi speaks with the CEO, Co-Founder & President of Ossium Health about how personal experiences with a reactive healthcare system—and a deep curiosity about the universe—sparked a mission to extend human healthspan through regenerative medicine. Kevin shares how Ossium built the first scalable bone marrow bank sourced from deceased organ donors—unlocking a powerful, overlooked source for life-saving cell therapies. He discusses Ossium's fully integrated model, commercialization strategy, and why rigorous company-building must go hand-in-hand with scientific ambition. The conversation unpacks hard-won lessons from raising $130M+, navigating regulatory pathways, and leading with long-term conviction. A bold vision for transforming cell therapy—and a candid look at the mindset needed to build for impact. Biography: As CEO, Co-Founder & President of Ossium Health, Kevin Caldwell has built Ossium from a small startup into a clinical stage bioengineering company. Mr. Caldwell set the company's mission to improve human health through bioengineering and designed its platform-based model for cellular therapeutics development. Mr. Caldwell has led the company's successful pursuit, negotiation, and execution of more than 50 business relationships, including 5 successful fundraisings and dozens of supply partnerships, clinical partnerships, and commercial contracts with biopharmaceutical companies. After seven years of strategic engagement and networking, Mr. Caldwell drove the team to successfully secure a transformative federal contract with BARDA (Biomedical Advanced Research and Development Authority) that validates Ossium's innovative approach. This milestone represents the culmination of persistent relationship-building, targeted proposals, and our unwavering commitment to addressing national biomedical challenges through cutting-edge technology and collaborative partnerships. Prior to founding Ossium, Mr. Caldwell served as an Engagement Manager at McKinsey's San Francisco office where he advised clients in the biotechnology and healthcare sectors. His projects ranged from due diligence of acquisition targets in the biotech startup ecosystem to restructuring distressed biopharma companies. Mr. Caldwell led more than 20 engagements with more than a dozen clients, leading teams that advised clients on revenue growth, go to market strategy, and organizational restructuring. Before McKinsey, Mr. Caldwell served as a Senior Investment Associate at Bridgewater Associates where he did quantitative research for the firm's global macro investments. Mr. Caldwell studied Physics and Economics at MIT before receiving his JD from Harvard Law School. In addition, he is a member of the Young Presidents Organization (YPO), and a Fellow of the Leaders in Tech Program.

Ming-Wei Chen and Fangheng Zhou, the scientists behind RephImmune, join hosts Lynnsey and Matthew to discuss how their biotech startup is developing a next-generation cell therapy platform designed to overcome the limitations of CAR-T.  With a focus on ovarian cancer, RephImmune's RACE-T technology targets solid tumors by re-engineering immune cells to better locate, penetrate, and survive in the harsh tumor environment. Ming-Wei and Fangheng share their personal motivations, the science behind their approach, and how programs like SCbio DRIVE are helping them move closer to clinical trials. Tune in now!

Dr. Jeanne Loring is a Co-Founder of Aspen Neuroscience and Professor Emeritus at Scripps Research.  She talks about generating gametes from the functionally extinct northern white rhinoceros, Aspen's early days and its recent clinical trial results for Parkinson's cell therapy, and sending her own stem cells into space. She also discusses the need for genetic variation in iPSC lines.

Good morning from Pharma and Biotech daily: the podcast that gives you only what's important to hear in Pharma e Biotech world.George Tidmarsh, a biopharma veteran and adjunct professor at Stanford's medical school, has been appointed as the new head of the FDA's Center for Drug Evaluation and Research (CDER). His extensive industry experience will be valuable in his new government position. Data secrecy among cell and gene therapy developers is hindering progress in the field, causing fragmentation, stalling innovation, and delaying access to treatments. Meanwhile, Sarepta is facing challenges with its elevidys shipments and has lost platform designation for its technology. At the same time, Roche's phase III trial in COPD has failed, impacting the market path for astegolimab. Layoffs are happening at companies like GSK, Sail, and BioNTech. Experts are exploring new ways to overcome barriers in cell therapy production.Data secrecy among cell and gene therapy developers continues to hinder progress and access to treatments. Acadia has introduced a new team and pipeline with ambitious goals. Patients are fighting for access to Brainstorm's ALS drug after promising real-world data. Moderna's withdrawal of its flu vaccine has left combination flu/COVID-19 vaccines in limbo. In other news, Sarepta is facing challenges with its DMD gene therapy, Ultragenyx's gene therapy for Sanfilippo syndrome is rejected by the FDA, GSK's Blenrep loses an adcomm vote, and BMS' anemia drug Reblozyl fails a Phase III trial. The FDA is experiencing layoffs and employee turnover amid an overhaul. Vinay Prasad overruled reviewers on Moderna's COVID-19 shot for kids. Upcoming events include a webinar on AI for real-world research and job opportunities in the biopharma industry. Readers are encouraged to provide feedback and suggest topics for future coverage.

Kevin Caldwell, CEO, President, and Co-Founder of Ossium Health, discusses the opportunities for regenerative stem-cell therapies and the challenges of obtaining bone marrow from donors for bone marrow transplants.  Ossium Health is collecting bone marrow from healthy, young donors and utilizing a cryopreservation process that enables long-term storage and on-demand availability of these cells. Ongoing clinical trials using allogeneic bone-marrow-derived cells to treat patients with acute leukemia and myelodysplastic syndrome are showing promising results. Kevin explains, "Our mission is to improve the health, longevity, and vitality of human beings through bioengineering. We believe that regenerative therapeutics, which involve cell and gene therapies that can permanently and positively improve human biology, will be fundamental to the prevention of disease and the preservation of health as we age. One of the most powerful tools we have in achieving this goal is engineering the immune system. And in particular at Ossium, we develop therapeutics that enable us today in the clinic to treat life-threatening malignancies of the immune system, like leukemia, in patients with dire need. Into the future, the goal is to make it possible to reconstitute, reprogram, and replace a blood and immune system in a manner that can treat patients with chronic diseases and ultimately do so in a preventive manner."   "Traditionally, the major application of bone marrow in the clinic is bone marrow transplants for patients with blood cancers like leukemia. And so the patient's native immune system is ablated or annihilated and replaced with a bone marrow infusion from a healthy donor, related or unrelated. And if that bone, new bone marrow from the donor in grafts in the patient, then it replaces or it constitutes their blood and immune system."  "And so one of the things that we're working on at Ossium is, okay, how do we address these problems? How do we make bone marrow more available to deliver and deploy on demand and easily, without needing to track down a volunteer donor? What can we do to reduce the risk of rejection so that the transplant is safer? What can we do to improve the likelihood of engraftment the first time and the speed of engraftment so the patients aren't immunocompromised for as long? All of these are things that we're working on to really make it possible to bring this lifesaving procedure to more patients."  #OssiumHealth #StemCellTherapy #RegenerativeMedicine #BoneMarrowTransplants ossiumhealth.com Download the transcript here

Kevin Caldwell, CEO, President, and Co-Founder of Ossium Health, discusses the opportunities for regenerative stem-cell therapies and the challenges of obtaining bone marrow from donors for bone marrow transplants.  Ossium Health is collecting bone marrow from healthy, young donors and utilizing a cryopreservation process that enables long-term storage and on-demand availability of these cells. Ongoing clinical trials using allogeneic bone-marrow-derived cells to treat patients with acute leukemia and myelodysplastic syndrome are showing promising results. Kevin explains, "Our mission is to improve the health, longevity, and vitality of human beings through bioengineering. We believe that regenerative therapeutics, which involve cell and gene therapies that can permanently and positively improve human biology, will be fundamental to the prevention of disease and the preservation of health as we age. One of the most powerful tools we have in achieving this goal is engineering the immune system. And in particular at Ossium, we develop therapeutics that enable us today in the clinic to treat life-threatening malignancies of the immune system, like leukemia, in patients with dire need. Into the future, the goal is to make it possible to reconstitute, reprogram, and replace a blood and immune system in a manner that can treat patients with chronic diseases and ultimately do so in a preventive manner."   "Traditionally, the major application of bone marrow in the clinic is bone marrow transplants for patients with blood cancers like leukemia. And so the patient's native immune system is ablated or annihilated and replaced with a bone marrow infusion from a healthy donor, related or unrelated. And if that bone, new bone marrow from the donor in grafts in the patient, then it replaces or it constitutes their blood and immune system."  "And so one of the things that we're working on at Ossium is, okay, how do we address these problems? How do we make bone marrow more available to deliver and deploy on demand and easily, without needing to track down a volunteer donor? What can we do to reduce the risk of rejection so that the transplant is safer? What can we do to improve the likelihood of engraftment the first time and the speed of engraftment so the patients aren't immunocompromised for as long? All of these are things that we're working on to really make it possible to bring this lifesaving procedure to more patients."  #OssiumHealth #StemCellTherapy #RegenerativeMedicine #BoneMarrowTransplants ossiumhealth.com Listen to the podcast here

Want to share your thoughts about the podcast? Text a Message!Welcome to the "Partner with Your Mind" Podcast Series!Episode 2: Command Cell Therapy – Remembering Your Powerful Mind-Body ConnectionIn this episode, you'll get to learn how Command Cell Therapy works within Rapid Transformational Therapy® to boost the power of healing physical and emotional issues. Hear how Beth healed her thyroid issue by using this combination, as well as two profound client transformational stories. You'll even get a sample of Command Cell Therapy at the very end! Be sure to check out the page mentioned here: https://www.inspiredriding.com/dude Please leave a review and share this with friends, if you enjoyed this episode!

In this episode of BioTalk, Rich Bendis welcomes Dr. Helen Sabzevari, President and CEO of Precigen, to discuss the company's cutting-edge science in gene and cell therapy. Dr. Sabzevari shares how Precigen's unique AdenoVerse® platform has powered the development of PRGN-2012, a potential first-in-class therapeutic currently under FDA priority review for the treatment of adults with recurrent respiratory papillomatosis (RRP), a rare and devastating disease. She also highlights advances across Precigen's broader pipeline in immuno-oncology and autoimmune disease and reflects on how Maryland's BioHealth Capital Region has supported the company's innovation and growth.   Editing and post-production work for this episode was provided by The Podcast Consultant.   Dr. Helen Sabzevari is the President and CEO of Precigen, Inc., and a leading expert in immunotherapy-based therapeutics. She previously served in executive roles at Compass Therapeutics and Merck KGaA/EMD Serono, where she advanced numerous immuno-oncology programs. Dr. Sabzevari began her career at the National Cancer Institute and has been widely recognized for her leadership and scientific contributions, including honors from NIH, SITC, and Forbes.

Send us a textEnlivex Therapeutics CEO Oren Hershkovitz, PhD joins us on another WTR Small-Cap x Biotech Spotlight crossover episode to talk about how the company's cell therapy, Allocetra, reprograms macrophages to treat inflammatory diseases. We dive into the recent Phase 1 data in knee osteoarthritis and what to expect from the randomized Phase 2 readout later this year, as well as the market opportunity. Listen to learn more about Enlivex Therapeutics and its off-the-shelf cell therapy platform. 

Welcome to the NeurologyLive® Mind Moments® podcast. Tune in to hear leaders in neurology sound off on topics that impact your clinical practice. In this episode, "Bemdaneprocel and the Future of Cell Therapy in Parkinson Disease" Rajesh Pahwa, MD, director of the Parkinson's Disease and Movement Disorder Center at the University of Kansas Medical Center, discusses the phase 3 exPDite-2 trial evaluating bemdaneprocel, an investigational cell therapy for Parkinson disease (PD). Pahwa explains the rationale behind cell replacement therapy as a means to restore dopamine production, outlining the progression from fetal tissue implants to stem cell–derived dopaminergic neurons. He details key elements of the study design, including patient selection criteria, trial endpoints, and long-term goals. Reflecting on decades of research in this space, he emphasizes the potential impact of a one-time, hardware-free surgical option and how this novel approach could complement or shift the current treatment landscape. While many questions remain, Pahwa highlights the promise of bemdaneprocel as a next-generation option in Parkinson care. Looking for more Movement disorder discussion? Check out the NeurologyLive® Movement disorder clinical focus page. Episode Breakdown: 1:00 – Mechanism and rationale behind bemdaneprocel as a dopaminergic cell therapy 3:00– Key design considerations for conducting a registrational phase 3 cell therapy trial 5:25 – Trial structure, eligibility criteria, and ongoing patient recruitment 6:35 – Neurology News Minute 8:45 – Significance of reaching phase 3 with a cell therapy in Parkinson disease 10:20 – Future role of cell therapy within the Parkinson disease treatment landscape 12:10 – Unique aspects of the exPDite-2 trial and what sets it apart from earlier efforts The stories featured in this week's Neurology News Minute, which will give you quick updates on the following developments in neurology, are further detailed here: BTK Inhibitor Fenebrutinib Demonstrates Long-Term Suppression of MS Activity in Open-Label Extension REGENXBIO Reports Positive Phase 1/2 Data for Higher Dose of RGX-202 in Duchenne Muscular Dystrophy NewAmsterdam Pharma Reports Positive Topline Data of Alzheimer Agent Obicetrapib from Phase 3 BROADWAY Trial Thanks for listening to the NeurologyLive® Mind Moments® podcast. To support the show, be sure to rate, review, and subscribe wherever you listen to podcasts. For more neurology news and expert-driven content, visit neurologylive.com.

In this episode of The Top Line, Fierce Biotech’s Chris Hayden sat down with Dr. Juergen Eckhardt of Bayer and Dr. Seth Ettenberg of BlueRock Therapeutics to discuss an innovative partnership that’s reshaping how big pharma teams up with biotech startups. BlueRock, a wholly owned subsidiary of Bayer, operates with the independence of a small biotech while benefiting from Bayer’s global reach and infrastructure. The “arm’s-length” model gives BlueRock the agility to innovate while leveraging Bayer’s resources in manufacturing, clinical trials and commercialization. The collaboration has already led to major milestones, including the development of a promising cell therapy for Parkinson’s disease, which is expected to enter Phase 3 trials later this year. “This isn’t just a partnership—it’s a relationship,” Eckhardt said. “We’re combining the best of both worlds: biotech speed and pharma scale.” The episode also explores how Bayer’s experience in biologics manufacturing has helped BlueRock overcome one of regenerative medicine’s biggest challenges—scaling complex cell therapies. The model has proven so effective that Bayer has since replicated it with other companies, signaling a broader shift in how the company approaches innovation. To learn more about this unique partnership and what it means for the future of neurological disease treatment, listen to the full episode of The Top Line.See omnystudio.com/listener for privacy information.

This episode focuses on the operational considerations for the use of in vivo and viral based gene therapies in health-system pharmacies and the appropriate stakeholders. CE for this episode expires on May 31, 2027.   The information presented during the podcast reflects solely the opinions of the presenter. The information and materials are not, and are not intended as, a comprehensive source of drug information on this topic. The contents of the podcast have not been reviewed by ASHP, and should neither be interpreted as the official policies of ASHP, nor an endorsement of any product(s), nor should they be considered as a substitute for the professional judgment of the pharmacist or physician.

Despite the long holiday weekend, news in biopharma never slows down. In this week’s episode of "The Top Line," the Fierce team breaks down some of the biggest stories from the past week. On the pharma side, Eric Sagonowsky and Kevin Dunleavy examine Big Pharma’s first-quarter 2025 performance. While most of the top 25 companies are still delivering strong sales growth despite emerging uncertainties in Washington, D.C., a few major players are starting to see a slowdown. Each company tells a different story, and Sagonowsky and Dunleavy dive into the nuances of the current commercial landscape. Later, Fierce Biotech's Gabrielle Masson and Darren Incorvaia highlight key data from the American Society of Gene & Cell Therapy conference and preview what the team is watching at this week’s American Society of Clinical Oncology annual meeting. To learn more about the topics in this episode: Seven top pharmas posted revenue declines in Q1. The common thread? All are US firms Atsena eye disease gene therapy hits safety goals, closes retinal splits in phase 1/2 ASGCT: Analysts see Rocket gene therapy setting 'a new bar' for efficacy in heart condition Rocket crashes as gene therapy patient dies, FDA imposes hold This episode is sponsored by Cencora. See omnystudio.com/listener for privacy information.

Dr. Jacqueline Barry is the Chief Clinical Officer at Cell and Gene Therapy Catapult, Dr. Kapil Bharti is a Senior Investigator at the National Eye Institute at the National Institutes of Health, and Dr. Jack Mosher is the Scientific Director at the International Society for Stem Cell Research (ISSCR). They discuss their work on the ISSCR's Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies. They talk about the need for consistency and rigor in cell therapy development, and how the document can be navigated and applied across jurisdictions.

This episode reviews the viral replication process, common viral vectors for in vivo gene therapy, and Food and Drug Administration-approved therapies. The episode will also highlight novel in vivo gene therapies in the development pipeline. CE for this episode expires on May 25, 2027.   The information presented during the podcast reflects solely the opinions of the presenter. The information and materials are not, and are not intended as, a comprehensive source of drug information on this topic. The contents of the podcast have not been reviewed by ASHP, and should neither be interpreted as the official policies of ASHP, nor an endorsement of any product(s), nor should they be considered as a substitute for the professional judgment of the pharmacist or physician.

The informant's microphone picked up the din of the busy beachfront restaurant. Sheriff's deputies were stationed in a covert van nearby. They had a camera trained on one of the large windows, focusing on the target's hands passing an envelope of money to the informant. Still clear enough over the customer's chatter, the informant asked when he wanted it done. “Yesterday” was the response…*** LISTENER CAUTION IS ADVISED *** This episode was researched and written by Eileen Macfarlane.Edited by Joel Porter at Dot Dot Dot Productions.Script editing, additional writing, illustrations and production direction by Rosanna FittonNarration, additional audio editing, script editing, and production direction by Benjamin Fitton.To get early ad-free access, including Season 1, sign up for They Walk Among PLUS, available from Patreon or Apple Podcasts.More information and episode references can be found on our website https://theywalkamonguspodcast.comMUSIC: Sabotage by Cody Martin Darker Days by Alternate Endings Far From Home by Cody Martin Illusion by Cody Martin Playing Games by Cody Martin A Construct by Falls Moonbeam by Falls Fractured Light by Stephen Keech Fleet Street by Stephen Keech Glass Houses by Third Age Bingdu by Kevin Graham Liminal by Chelsea McGough Blackstone by Lincoln Davis Stratos by Lone Canyon Life Giving by Moments Cocoon Of Light by Phillip Mount Blinding Light by Salon Dijon Distances by Salon Dijon Harboring by Salon Dijon The Matter At Hand by Salon Dijon Worth by Shimmer Stasis Interrupted by Dresden, the Flamingo Winds Of Change by Four Trees SOCIAL MEDIA: https://linktr.ee/TheyWalkAmongUsSupport this show http://supporter.acast.com/theywalkamongus. Hosted on Acast. See acast.com/privacy for more information.