Podcasts about astellas

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we'll explore a series of pivotal advancements and challenges shaping the industry landscape—ranging from scientific breakthroughs to regulatory maneuvers, all underscoring the sector's dynamic evolution. Novartis has made headlines with its investigational drug, Delpacibart Braxlosiran, which successfully met primary endpoints in a Phase 1/2 trial for treating Facioscapulohumeral Muscular Dystrophy (FSHD). This RNA therapy utilizes an antibody-oligonucleotide conjugate to target the DUX4 gene, a significant cause of FSHD. The promising results not only mark a potential therapeutic breakthrough for this debilitating neuromuscular disease but also highlight Novartis' commitment to innovative treatments in rare diseases. In parallel, Enliven Therapeutics has reported positive outcomes from a Phase 1 trial of ELVN-001 for patients with Chronic Myeloid Leukemia (CML), who are heavily pretreated. This small molecule tyrosine kinase inhibitor shows improved efficacy, offering hope for those who have exhausted existing treatment options. As ELVN-001 progresses into later-stage trials, it may significantly enhance therapeutic options and outcomes for CML patients. Regulatory activities remain at the forefront, exemplified by Amgen's engagement with the Duke Clinical Research Institute to defend Tavneos amid an FDA push for market withdrawal. This illustrates the intricate complexity of regulatory processes and the importance of evidence-based advocacy in preserving access to critical therapies. In another regulatory development, Guardant Health's Guardant360 CDx has received FDA approval as a companion diagnostic for Boehringer Ingelheim's Hernexeos targeting HER2-mutant non-small cell lung cancer. The approval emphasizes the growing significance of liquid biopsy technologies in personalized oncology care, offering precise and less invasive diagnostic solutions. In business development news, strategic collaborations are gaining momentum. Simcere Pharmaceutical has partnered with Stanford Medicine to develop first-in-class therapies for Idiopathic Pulmonary Fibrosis. Meanwhile, Ildong Pharmaceutical and Welt are joining forces to create AI-driven digital therapeutics. These alliances reflect an increasing trend towards integrating cutting-edge technology and cross-disciplinary expertise to accelerate drug discovery and development. Funding dynamics within the sector are evolving, as seen with Neion Bio's successful Series A fundraising of $23 million aimed at enhancing its biologics manufacturing platform. Similarly, Human Continuum secured $5.13 million to advance its exosome-based regenerative medicine platform. These investments underscore a strong focus on innovative platform technologies promising to transform therapeutic modalities and manufacturing processes. Not all news is positive, however; Jazz Pharmaceuticals and PharmaMar faced setbacks as their Phase 3 trial for Zepzelca (Lurbinectedin) failed to meet its overall survival endpoint in metastatic small cell lung cancer. This highlights the inherent challenges in oncology drug development and underscores the need for continuous innovation. Regulatory hurdles persist as well, evidenced by Camurus receiving a second complete response letter from the FDA regarding Oclaiz (Octreotide) due to manufacturing concerns. The industry is also navigating cybersecurity challenges, as seen with Novo Nordisk's recent data breach incident. This breach underscores vulnerabilities within biopharma companies concerning intellectual property and patient data protection. It serves as a stark reminder of the necessity for robust cybersecurity measures to safeguard sensitive information critical to clinical trials and corporate integrity. Meanwhile, strategic planning remains essential as Astellas confronts a looming $6 billion patent cliff with Xtandi, its prostate cancer drug. The company's five-year strategy focuses on cost savings while sustaining innovation and competitiveness amidst impending revenue declines. The biotechnology sector continues to thrive amidst these challenges, evidenced by record levels of initial public offerings (IPOs). Despite broader economic uncertainties, investor confidence remains robust, highlighting biotech's potential for groundbreaking advancements and lucrative returns. Finally, artificial intelligence is making significant strides across health agencies, with a reported 148% increase at the FDA by 2025 alone. This surge in AI integration highlights its transformative potential in regulatory processes and healthcare delivery—offering opportunities to enhance efficiency and decision-making within the sector. In summary, today's developments encapsulate the vibrant pharmaceutical and biotech industries characterized by scientific innovation, regulatory intricacies, strategic collaborations, and financial maneuvers. These advancements offer new hope for patients while navigating complex landscapes that demand continuous innovation and compliance—a balance that defines success in this ever-evolving sector.Support the show

Recorded in Chicago, Dr. Aly-Khan Lalani and Dr. Chris Wallis unpack a massive slate of prostate cancer data, fresh from the 2026 ASCO Annual Meeting. The conversation spans the entire disease spectrum, beginning with a deep dive into the PROTEUS trial. Shifting to advanced disease, Dr. Lalani and Dr. Wallis discuss the TALAPRO-3 and the A-DREAM/Alliance A032101 trials. Concluding with a review of sequencing chemotherapy and radioligand therapy in the PLUDO trial, this episode provides an excellent overview of the prostate cancer landscape presented at ASCO 2026.This podcast has been made possible through unrestricted financial support by Johnson & Johnson, Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.ca

Dr. Aly-Khan Lalani and Dr. Chris Wallis discuss how new data from the RAMPART trial strengthens the argument for administering  immune-boosting therapies to patients after surgery. This episode also touches on the human side of cancer care, exploring new ways to measure patient side effects and reduce post-treatment regret through improved  counseling. The doctors finish the conversation by highlighting a promising new combination of repurposed drugs that is offering renewed hope to young patients fighting Renal Medullary Carcinoma.This podcast has been made possible through unrestricted financial support by Johnson & Johnson, Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.ca

Dr. Aly-Khan Lalani and Dr. Chris Wallis dive into the latest bladder cancer breakthroughs, starting with crucial long-term updates from the landmark EV-302 trial. They share encouraging survival data for patients, while exploring which treatments work best if the cancer eventually progresses. The doctors also discuss the future of localized disease, reviewing strategies from the POTOMAC and SAKK trials. By highlighting a range of advanced monitoring tools, they map out how modern care is helping patients safely avoid major surgery while maintaining excellent disease control.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.caThis podcast has been made possible through unrestricted financial support by Johnson & Johnson, Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a myriad of events shaping the industry, ranging from regulatory hurdles to strategic acquisitions and scientific breakthroughs. A significant milestone has been achieved by Outlook Therapeutics with its product Lytenava (bevacizumab), which recently won an appeal with the FDA for wet age-related macular degeneration treatment. This antibody therapy, aimed at VEGF inhibitors, marks a notable advancement in ophthalmology, potentially transforming management strategies for this debilitating condition. Monoclonal antibodies like Lytenava continue to underscore their importance in treating chronic diseases, offering hope for patients and setting benchmarks for similar therapeutic innovations. On a less favorable note, AstraZeneca encountered a setback when their breast cancer treatment camizestrant faced rejection from an FDA advisory committee. Despite robust phase 3 data for hormone receptor-positive breast cancer, the advisory committee's decision delays the drug's review process. This serves as a stark reminder of the stringent requirements oncology treatments must meet and highlights the critical need for comprehensive clinical data to ensure patient safety and drug efficacy in this competitive field. Amidst these developments, Astellas is proactively addressing upcoming patent expirations, particularly for Xtandi in 2026. The company has announced a five-year growth strategy focused on pipeline acquisitions, illustrating broader industry trends where diversifying portfolios through acquisitions and partnerships is crucial for maintaining competitiveness and delivering innovative therapies. Business development activities continue to be a highlight within the sector. Apogee Therapeutics and Blackstone Life Sciences have entered into a $1.3 billion royalty financing agreement to advance an eczema drug, underscoring ongoing investments in autoimmune treatments. Similarly, AGC Biologics' partnership with Teikoku Seiyaku on microbial CDMO services aims at advancing musculoskeletal therapies, showcasing how collaborations can leverage specialized manufacturing capabilities for therapeutic progress. Clinical trials also made headlines as Junshi Biosciences reported that its drug toripalimab met primary endpoints in phase 3 trials for non-small cell lung cancer perioperative treatment. This achievement underscores the growing influence of immunotherapies in oncology, promising improved surgical outcomes and enhancing their role within cancer treatment paradigms. Funding continues to play a pivotal role in sustaining innovation within the industry. Editas Medicine recently raised $125 million to propel its CRISPR-based gene therapy pipeline forward. Such financial backing is instrumental in transitioning promising therapies from preclinical stages to market readiness, ensuring that groundbreaking science translates into patient-accessible treatments. Regulatory landscapes remain dynamic, with ongoing discussions about updating COVID-19 vaccine formulations to target emerging subvariants. The FDA's commitment to adapting vaccine guidelines reflects a proactive stance in infectious disease management, crucial for maintaining vaccine efficacy against evolving pathogens. In acquisition news, Olympus' purchase of Bioprotect for $270 million adds biodegradable balloon spacer technology to its prostate cancer radiation therapy offerings. This acquisition highlights how medical device innovations can complement pharmaceutical approaches, enhancing treatment efficacy and patient quality of life. These developments collectively paint a vibrant picture of the biotech and pharmaceutical industries—a landscape where scientific advancements, regulatory challenges, strategic planning, and financial investments converge. The implications are far-reaching, potentially redefining treatment paradigms across various therapeutic areas as these sectors continue their pursuit of innovation and improved patient care outcomes. Navigating this landscape also involves addressing broader challenges such as policy shifts and pricing pressures within key markets like the United States. Companies must remain adaptable, balancing growth strategies with compliance demands amidst changing regulatory environments—a theme echoed by recent surveys indicating heightened pressure on healthcare firms to maintain profitability. Moreover, geopolitical considerations are influencing cross-border investments in biotechnology as policymakers debate strategies best suited for managing foreign influence—reflecting broader concerns about national security and economic competitiveness within this critical sector. As we continue monitoring these dynamic shifts within pharmaceuticals and biotechnology, it's evident that scientific innovation remains tightly interwoven with regulatory scrutiny and financial dynamics. The ongoing dance between these elements will undoubtedly shape future trajectories in healthcare advancements globally, promising new horizons in patient care while addressing pressing health challenges worldwide. That's all for today's episode of Pharma Daily—where we keep you informed on the latest developments driving progress within pharmaceuticals and biotechnology. Thank you for tuning in; until next time!Support the show

From practical risk stratification to the real-world challenges of delivering BCG, Dr. Aly-Khan Lalani and Dr. Christopher Wallis lead a conversation with Dr. Kulkarni about non-muscle invasive bladder cancer and its rapidly evolving treatment landscape. Dr. Kulkarni is a urologic surgeon at the Princess Margaret Cancer Centre, University Health Network, and a surgeon-scientist and Assistant Professor at the University of Toronto. As a thought leader in bladder cancer, he shares key clinical decision points, patient-centred considerations, and practical strategies that clinicians can apply today.This podcast has been made possible through unrestricted financial support by Johnson & Johnson, Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.ca

In Part 2 of the Bladder Cancer Awareness Month series, Dr. Kulkarni, Dr. Lalani and Dr. Wallis examine a "systemic therapy first" approach. Using a real-world patient scenario, the doctors break down the latest clinical trial data and discuss how the surge in neoadjuvant therapy is reshaping everyday decision-making.The conversation also tackles the unique challenges of Upper Tract Urothelial Carcinoma. From evidence gaps in upper tract care to the complexities of trial design, this episode offers a forward-looking map of the evolving urothelial cancer landscape.This podcast has been made possible through unrestricted financial support by Johnson & Johnson, Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.ca

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 fascinating array of industry shifts and scientific breakthroughs that are shaping the future of medicine and patient care. Sun Pharma's acquisition of Organon for $11.75 billion is making waves as it marks a significant expansion for the Indian pharmaceutical giant into international markets. This deal not only amplifies Sun Pharma's footprint but also integrates Organon's strong focus on women's health and biosimilars into its portfolio. It's a strategic move that underscores the increasing trend of cross-border mergers as companies seek to diversify and enhance their global presence. As the largest-ever overseas acquisition by an Indian firm, this transaction is emblematic of the growing importance of global consolidation in the pharma industry. Meanwhile, Astellas Pharma faces a mixed fiscal outlook with its prostate cancer drug Xtandi. While Xtandi experienced a modest sales increase in 2025, a projected decline for 2026 has prompted Astellas to pivot towards developing new medicines. This highlights the critical dependence on innovation within the industry to sustain growth, especially as competition intensifies. In the realm of ATTR amyloidosis treatments, Pfizer has reached a settlement with two generic manufacturers over its drug Vyndamax. This resolution is crucial in stabilizing the market amidst rising generic competition, which can significantly influence pricing and market dynamics. Such settlements are an integral part of maintaining competitive positioning in crowded therapeutic spaces. Ligand Pharmaceuticals' strategic purchase of XOMA for $739 million signals a broader trend of consolidation among biotech royalty aggregators. This acquisition aligns with Ligand's strategy to augment its portfolio through mergers and acquisitions—a recurring theme as biotech companies strive to streamline operations and expand their competitive edge. The competition within the oral GLP-1 market is intensifying, particularly with Eli Lilly's delayed launch of Foundayo inadvertently benefiting Novo Nordisk's market position. This rivalry underscores the fierce race to dominate the lucrative obesity management sector, highlighting how pivotal timing can be in gaining market share. The FDA's recent allocation of national priority vouchers to Compass Pathways, Usona Institute, and Transcend Therapeutics marks a significant regulatory shift towards embracing psychedelic therapies. These vouchers could expedite development timelines, thereby accelerating patient access to novel treatments that have traditionally been underexplored. Thermo Fisher Scientific's robust start to 2026 showcases how strategic acquisitions like that of Clario can drive growth. This reflects broader industry trends where biopharma companies leverage technological advancements to enhance capabilities in clinical trial support and data management. In gene editing news, Intellia Therapeutics is advancing towards FDA approval for its in vivo CRISPR therapy following successful Phase 3 trials. This progress heralds a new era for gene editing technologies in treating genetic disorders, underscoring their potential to revolutionize therapeutic approaches. Eli Lilly continues its aggressive acquisition strategy by purchasing Ajax Therapeutics for $2.3 billion. This acquisition focuses on next-generation JAK inhibitors for myelofibrosis, aligning with Lilly's broader goal to diversify its pipeline and maintain its leadership in innovative therapeutics. Recent studies have also shed light on how an endoscopic procedure used in Europe can aid patients on Eli Lilly's tirzepatide in maintaining weight loss post-treatment. This finding could significantly impact long-term obesity management strategies, offering new insights into sustainaSupport the show

In this episode of the IDEA Collider, host Mike Rea sits down with Marianne De Backer, CEO of Vir Biotechnology, to explore how she is leading one of biotech's most complex transformations.  After the rapid rise and decline of COVID-19 revenues tied to sotrovimab, Marianne stepped into Vir Bio in 2023 and led a bold strategic reset—refocusing the company on immuno-oncology, infectious disease, and platform-driven innovation.  The conversation dives into Vir Bio's next chapter, including its masked T-cell engager (TCE) pipeline and the PRO-XTEN® masking platform, which is designed to overcome the safety challenges of TCEs in the treatment of solid tumors by shielding therapies until they reach the tumor microenvironment.  They also discuss Vir Bio's advancing hepatitis delta program, currently in registrational Phase 3 trials, and the company's growing pipeline leveraging the synergy of its AI-driven discovery, protein engineering capabilities, and universal PRO-XTEN® masking technology.  Marianne shares what it takes to lead through a biotech downturn—from restructuring and capital discipline to rebuilding culture, integrating new teams, and positioning for long-term growth.  This episode is a deep dive into biotech turnaround strategy, next-generation cancer therapies, and leadership in times of uncertainty. Episode  Timestamps  00:00 – Introduction and Vir's transformation story  00:40 – Marianne De Backer's 30+ year pharma journey  02:42 – Vir's origins and post-COVID strategic pivot  04:42 – Taking over as CEO during a crisis  06:33 – Lessons from the biotech downturn (“biotech winter”)  08:56 – Astellas partnership and T-cell engager strategy  09:52 – ProXtend platform: masked T-cell engagers explained  13:24 – Clinical data, safety, and tumor targeting  15:32 – Integrating new teams and scientific expertise  17:38 – Expanding the pipeline (HER2, EGFR, oncology)  19:50 – Hepatitis delta program and commercialization plans  22:11 – Funding strategy and biotech market outlook  25:37 – FDA interactions and regulatory perspective  28:13 – AI in drug discovery and clinical trials (Daisy platform)  31:34 – Culture: grit, ingenuity, collaboration, authenticity  34:21 – Personal reflections and leadership mindset  35:46 – Closing thoughts  Don't forget to Like, Share, Subscribe, Rate, and Review!      Keep up with Marianne De Backer;  LinkedIn: https://www.linkedin.com/in/marianne-d-de-backer-msc-phd-mba-73403411/  Website: https://www.vir.bio/      Follow IDEA Pharma On;  Website: https://www.ideapharma.com/  LinkedIn: https://www.linkedin.com/company/idea-pharma   Listen to more fantastic podcast episodes: https://ideacollider.simplecast.com/

La quinta temporada de Escuela de Calor ya está aquí y arranca con un tema que seguramente te ha ocupado la cabeza en algún momento de tu vida (y probablemente también ahora mismo): el peso, el cuerpo y la presión de estar delgadas.Nuestra invitada es Sofía Giaquinta, nutricionista especializada en TCA y nueva colaboradora habitual de este podcast. Nos sentamos a hablar de la vuelta de la delgadez extrema, de lo que nadie te cuenta sobre los GLP-1, y resolvemos la gran duda: ¿por qué engordamos con la edad? Una lección sin culpas, sin dietas milagro y con mucha información.Si quieres aprender sobre la menopausia y uno de sus síntomas más habituales: los sofocos, entra en menopausiaysofocos.es / En colaboración con Astellas (publi).ATENCIÓN: la guía descargable todavía no está disponible, pero la subiremos muy pronto y avisaremos por Instagram :) Disculpa las molestias ESCUELA DE CALOR es un podcast de Bloom. Puedes seguirnos en @‌bebloomers

In this episode, Dr. Aly-Khan Lalani, Dr. Christopher Wallis, and Dr. Alex Wyatt walk through the fundamentals of clinical genomics and why it matters for routine practice. They discuss which alterations matter in prostate cancer, germline vs. somatic testing, ctDNA test use cases and limits, and more.Dr. Alex Wyatt is an Associate Professor in Urologic Sciences and holds the President's Excellence Chair in Precision Oncology at the University of British Columbia. He is also a Senior Scientist at the Vancouver Prostate Centre and BC Cancer, where his work focuses on advancing clinical genomics and precision oncology in prostate cancer.This podcast has been made possible through unrestricted financial support by Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.ca

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're exploring a fascinating realm where technology and biology converge, starting with a deepening relationship between biopharma and artificial intelligence. Novartis CEO Vas Narasimhan's recent appointment to the board of AI company Anthropic signals the strategic integration of AI into drug discovery and development processes. This collaboration highlights a growing trend where pharmaceutical companies are increasingly leveraging AI to optimize clinical trials, streamline drug discovery, and personalize patient care strategies. Similarly, Novo Nordisk has announced a strategic partnership with OpenAI to integrate AI technologies across various facets of its operations, including drug discovery and manufacturing. By leveraging OpenAI's machine learning capabilities, Novo Nordisk aims to streamline research efforts and accelerate therapeutic identification—a collaboration reflecting AI's growing role as an essential tool for maintaining competitiveness in drug development. Additionally, Amazon Web Services' launch of the Amazon Bio Discovery AI tool marks another milestone. Designed to expedite antibody design and drug discovery processes, it provides researchers with robust AI-driven platforms enhancing therapeutic design speed and accuracy. The emphasis on monoclonal antibodies aligns with industry trends focusing on targeted therapies for diseases such as cancer. Meanwhile, Eli Lilly's new obesity treatment, Foundayo, has caught the FDA's attention due to potential safety concerns. Despite progressing with its launch, the FDA has requested additional safety information to address unexpected serious risks associated with the drug. This highlights the ongoing regulatory scrutiny that accompanies novel treatments, especially in areas like obesity where patient populations are large and diverse. In another strategic move, Eli Lilly's acquisition of Crossbridge Bio for up to $300 million aims to bolster its oncology pipeline with dual-payload antibody-drug conjugates (ADCs). This acquisition reflects a strategic move enhancing Eli Lilly's position in oncology by integrating cutting-edge ADC technologies known for delivering cytotoxic agents directly to cancer cells while minimizing off-target effects. On another front, Travere Therapeutics is mapping a pathway to a potential $3 billion opportunity in the U.S. market following significant approval for its treatment Filspari, targeted at rare kidney diseases. This approval underscores the increasing focus on rare diseases, which present lucrative opportunities for pharmaceutical companies due to significant unmet needs and often high-cost treatments. Astellas' manufacturing strategy underscores the importance of reliable supply as a critical bridge from research to patient care. Led by Chief Manufacturing Officer Rao Mantri, this strategy highlights how manufacturing excellence can significantly impact drug availability and patient outcomes. It emphasizes that production reliability is vital in ensuring groundbreaking research translates into accessible medical treatments. In contrast, a slowdown in IPOs has been noted amidst an aggressive merger and acquisition spree by major pharmaceutical companies. This consolidation trend reflects strategic shifts within the industry as companies seek to bolster pipelines through acquisitions rather than organic growth. Such dynamics indicate a strategic pivot as firms prioritize acquiring promising assets over developing them from scratch. Ionis Pharmaceuticals' recent win in a drug naming competition exemplifies the complexities involved in branding within the pharmaceutical sector. Crafting a drug name that is memorable yet distinctive involves balancing marketability with regulatory requirements—a reflection of the intSupport the show

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we dive into a series of transformative updates that reflect the dynamic landscape of our industry. Johnson & Johnson is making strategic moves in the immunology space, with Tremfya and the newly launched Icotyde playing pivotal roles in their portfolio. This development indicates J&J's commitment to capturing a significant share of the immunology market, aiming for a staggering $100 billion in annual revenue. Their collaboration with Protagonist Therapeutics for Icotyde highlights the importance of partnerships in driving innovation and maintaining a competitive edge in this rapidly evolving sector. In regulatory news, Travere Therapeutics has achieved a milestone with Filspari becoming the first FDA-approved treatment for focal segmental glomerulosclerosis. This approval comes after overcoming initial setbacks and offers new hope for patients suffering from this rare kidney disease. It exemplifies the perseverance required to navigate the complex regulatory landscape and underscores the significance of providing novel therapies where none existed before. Novo Nordisk is taking a leap into digital transformation by integrating artificial intelligence through a partnership with OpenAI. By embedding AI into their R&D and manufacturing processes, Novo aims to streamline data analysis and accelerate drug discovery timelines. This move reflects broader industry trends towards leveraging advanced technologies to enhance efficiency and innovation, ultimately benefiting patient outcomes. This approach aligns with trends towards precision medicine and improved patient care outcomes. However, not all news is positive. Pfizer recently faced FDA scrutiny over misleading advertisements for its cancer drug Adcetris on Facebook. This incident serves as a reminder of the critical importance of transparency and compliance in advertising practices, essential for ensuring patient safety and maintaining regulatory standards. The FDA has also issued reminders to clinical trial sponsors to report study results, revealing that 30% of registered studies remain unreported. This call to action is crucial for fostering transparency and accountability in clinical research, which are vital for understanding drug efficacy and safety profiles comprehensively. On the restructuring front, Astellas is closing its stem cell therapy unit in Seattle as part of strategic realignment efforts. Similarly, Click Therapeutics is downsizing its workforce following a commercial deal restructuring. These changes highlight ongoing challenges in resource allocation within the biotech sector. Financially, Harbinger Health has secured $100 million for its blood-based cancer detection tests, signaling growing interest in non-invasive diagnostics. Meanwhile, Alamar Biosciences prepares for an IPO amidst a surge in life sciences public offerings, indicating robust investor confidence in biotech innovations. In other news, Boehringer Ingelheim and Amgen have discontinued early-stage immunology candidates due to insufficient clinical promise. Such decisions underscore the rigorous evaluation processes inherent in drug development pipelines, emphasizing strategic prioritization necessary for advancing viable therapeutic candidates. Eli Lilly's acquisition of Crossbridge Bio for $300 million highlights their interest in antibody-drug conjugates (ADCs), underscoring a growing trend towards targeted cancer therapies. ADCs offer enhanced efficacy by combining cancer-specific antibodies with potent cytotoxic agents, reducing systemic toxicity while improving therapeutic outcomes. These updates illustrate an industry at the intersection of scientific innovation and strategic realignment. As companies navigate complex regulatory landscapes and adapt to market dynamicSupport the show

Are you walking into biotech panel interviews and talking to every person the same way? That approach can cost you the offer, even if your science is solid.In this episode, Carina Clingman and returning guest Stacey Tannenbaum, PhD break down how to build rapport with each type of interviewer you'll meet on a panel day, from the VP who hasn't read your CV to the peer scientist who feels threatened by what you bring to the table.In this episode, you'll learn how to:Talk to senior leaders about strategic impact instead of getting stuck in technical detailsShow hiring managers you want this specific role, not just any job, and why that distinction is the number one thing they evaluateRecognize the three reasons peer interviewers become skeptics: fear of change, territorial expertise, and feeling intimidated by what they don't understandUse empathy, curiosity, and "I need your help" to turn resistant colleagues into alliesFrame your science in terms that matter to cross-functional interviewers from other departmentsAsk better questions at every stage to make interviews feel like a two-way conversationLeverage your hiring manager as an ally to get intel on difficult personalities before you walk in the roomUse presentation techniques like reading facial expressions, spotting "thought bubbles," and asking for feedback to build real connection during job talksStacey has spent 20+ years leading teams in pharmacometrics at companies including Astellas, Metrum Research Group, and Novartis. She's sat on both sides of the interview table and now coaches scientists and technical professionals on communication and leadership through her consultancy, SJTPharm LLC.Whether you're preparing for your first biotech panel interview or your tenth, this episode gives you a practical playbook for winning over every person in the room.Connect with Stacey: https://www.linkedin.com/in/stacey-tannenbaum-phd-fisop/Learn more about the Collaboratory Career Hub community and access our free resources:Join our Skool CommunityTake the Free 7-day Interview Sprint ChallengeCheck out our sister podcast: Building BiotechsSend Carina a connection request on LinkedIn!Stay connected with us:

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/TRG865. CME/MOC/AAPA/IPCE credit will be available until March 26, 2027.Personalizing Care for Patients With Prostate Cancer: Collaborative Strategies for Modern Integration of Systemic Therapy In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc., and Bayer HealthCare Pharmaceuticals Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/TRG865. CME/MOC/AAPA/IPCE credit will be available until March 26, 2027.Personalizing Care for Patients With Prostate Cancer: Collaborative Strategies for Modern Integration of Systemic Therapy In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc., and Bayer HealthCare Pharmaceuticals Inc.Disclosure information is available at the beginning of the video presentation.

In der heutigen Folge sprechen die Finanzjournalisten Anja Ettel und Holger Zschäpitz über einen Warnschuss für Novo Nordisk, Amazons Mega-Emission und eine Dividendenperle mit Deindustrialisierungsgefahr. Außerdem geht es um Oracle, Verizon, AB Inbev, CVS, Microsoft, Meta, Nvidia, Vertiv, GE Vernova, Pershing Square, Berkshire Hathaway, Eli Lilly, NIO, Hugo Boss, Lufthansa, Bristol Myers Squibb, Astellas, Volkswagen, Audi, Stellantis, Ferrari. Wir freuen uns an Feedback über aaa@welt.de. Noch mehr "Alles auf Aktien" findet Ihr bei WELTplus und Apple Podcasts – inklusive aller Artikel der Hosts und AAA-Newsletter. Hier bei WELT: https://www.welt.de/podcasts/alles-auf-aktien/plus247399208/Boersen-Podcast-AAA-Bonus-Folgen-Jede-Woche-noch-mehr-Antworten-auf-Eure-Boersen-Fragen.html. Der Börsen-Podcast Disclaimer: Die im Podcast besprochenen Aktien und Fonds stellen keine spezifischen Kauf- oder Anlage-Empfehlungen dar. Die Moderatoren und der Verlag haften nicht für etwaige Verluste, die aufgrund der Umsetzung der Gedanken oder Ideen entstehen. Hörtipps: Für alle, die noch mehr wissen wollen: Holger Zschäpitz können Sie jede Woche im Finanz- und Wirtschaftspodcast "Deffner&Zschäpitz" hören. +++ Werbung +++ Du möchtest mehr über unsere Werbepartner erfahren? Hier findest du alle Infos & Rabatte! https://linktr.ee/alles_auf_aktien Impressum: https://www.welt.de/services/article7893735/Impressum.html Datenschutz: https://www.welt.de/services/article157550705/Datenschutzerklaerung-WELT-DIGITAL.html

Dr. Aly-Khan Lalani and Dr. Christopher Wallis also discuss the RAMPART trial, LITESPARK-011, and the K-COMPASS model. This final recap episode highlights how evolving adjuvant strategies and combination sequencing are reshaping the kidney cancer landscape. Be sure to listen to Episodes 35 and 36 for the full ASCO GU recap, covering key updates in prostate and bladder cancer!The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.caThis podcast has been made possible through unrestricted financial support by Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.

Dr. Aly-Khan Lalani and Dr. Christopher Wallis also highlight Canadian-led innovation in the GUNS trial and early data from the PAnTHA study, and discuss how Prostate Cancer Working Group 4 may redefine trial design. Episode 35 is the first of three ASCO GU recap episodes, so don't miss Episode 36 on bladder cancer and Episode 37 on kidney cancer!The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.caThis podcast has been made possible through unrestricted financial support by Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.

Dr. Aly-Khan Lalani and Dr. Christopher Wallis discuss practice-changing data in non-muscle invasive and muscle-invasive bladder cancer, including perioperative strategies,bladder-sparing approaches, and emerging targeted therapies. Don't forget to watch or listen to Episode 35 and Episode 37 for updates on prostate and kidney cancer!The View on GU with Lalani & Wallis integrates key clinicaldata from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.caThis podcast has been made possible through unrestricted financial support by Pfizer, Tolmar, AbbVie, Astellas, Eisai, Ipsen, Merck, Bayer, TerSera.

On this week's episode, Tess Cameron, Josh Schimmer, Brian Skorney, and special guest Adam Feuerstein kick off with regulatory updates, including the FDA's rejection of Atara Biotherapeutics and Pierre Fabre Pharmaceuticals' cell therapy, Ebvallo -- a therapy that should have been approvable. The co-hosts then highlight ongoing inconsistencies at the agency and the challenge it creates for investors and companies as the regulatory goal posts continue to shift. Next, they discuss a New York Post editorial from the Alliance for Regenerative Medicine CEO Tim Hunt, who outlined how last‑minute reversals on rare disease and CGT approvals are leaving patients and biotech companies in limbo. The conversation then shifts to deals, including Gilead's $7.8B acquisition of Arcellx for full control of anito-cel for relapsed/refractory multiple myeloma, and Vir's pivot to oncology through a $1.7B collaboration with Astellas. In data news, the co-hosts cover CagriSema's head-to-head trial results against Lilly's Zepbound, Gossamer Bio's lung disease drug, seralutinib, which did not meet the primary endpoint in its Phase 3 pulmonary arterial hypertension study, and Palvella Therapeutics' positive topline results from the Phase 3 study of QTORIN rapamycin in microcystic lymphatic malformations. The episode closes with company updates, including Sarepta CEO Doug Ingram's retirement and Xenon Pharmaceuticals' upcoming seizure drug readout. *This episode aired on February 27, 2026.

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

Giant bladder cancer paper in NEJM this week with the highly anticipated results of the perioperative EV-Pembro trial (EV-303 / KN-905). Already we can say this is a landmark publicaion in bladder cancer that will change practice and even challenge the role of cystectomy (maybe)! Declan Murphy and Renu Eapen caught up with first author Christof Vulsteke (Integrated Cancer Centre, Ghent, BEL) when we were at ESMO Asia recently and had a chat about this work and how it will change practice. This is a Themed Podcast supported by our Platinum Partner, Astellas. Even better on our YouTube channelLinks:EV-303 in NEJM 

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/NCPD/CPE/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/DBK865. CME/NCPD/CPE/AAPA/IPCE credit will be available until February 8, 2027.Paving the Path for Optimal Prostate Cancer Care: Preparing Advanced Practice Providers to Integrate Modern Therapies Into Tailored Treatment Plans In support of improving patient care, PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by educational grants from Astellas and Pfizer, Inc.Disclosure information is available at the beginning of the video presentation.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we're diving into a series of transformative events reshaping the industry landscape, from regulatory advancements to scientific breakthroughs and strategic business maneuvers.Kicking off with a significant regulatory update, the FDA's Rare Pediatric Disease Voucher Program has been rejuvenated through a newly signed government funding bill. This initiative is designed to expedite the development of treatments for rare pediatric diseases, offering crucial incentives to companies targeting this critical healthcare segment. By reauthorizing this program, there's an expectation of stimulating innovation and potentially bringing more treatments to market for conditions with limited existing therapies. This move underscores a broader commitment to addressing unmet medical needs through incentivized innovation.Turning to corporate developments, Eli Lilly is anticipating substantial growth in revenue despite facing pricing pressures on its key products, Mounjaro and Zepbound. The company projects revenues between $80 billion and $83 billion for 2026, marking a 25% increase from 2025 at the midpoint. This growth is attributed to strong product performance and strategic maneuvers within their pipeline. Eli Lilly has also made strategic decisions by optimizing its pipeline through dropping three clinical-stage drugs, including a gene therapy acquired via Prevail Therapeutics. This move points towards Lilly's focus on concentrating efforts on more promising candidates within their expansive pipeline. Additionally, Eli Lilly is expanding its GLP-1 franchise beyond metabolic diseases into immunology and inflammation with ongoing clinical trials in conditions such as asthma, psoriatic arthritis, Crohn's disease, and ulcerative colitis. This strategic expansion could lead to novel therapeutic options for chronic inflammatory diseases.Similarly, Bristol Myers Squibb is focusing on new growth drivers amid declining sales of legacy drugs. With $48.2 billion in revenue projected for 2025 largely stemming from newer products, BMS is strategically repositioning itself to maintain momentum amidst market changes.Novartis faces its largest patent expiry challenge but remains optimistic about its trajectory. CEO Vas Narasimhan suggests robust strategies are in place to counteract these patent expiries, indicating a strong focus on innovation and strategic planning to navigate these hurdles. Novartis is also refining its oncology strategy by cutting early-stage cancer candidates while adding new ones focused on promising therapeutics—a broader trend of adopting data-driven approaches to streamline drug development pipelines.Meanwhile, AbbVie continues its stronghold in the inflammatory bowel disease market with its blockbuster immunology drugs Skyrizi and Rinvoq. These products significantly contribute to AbbVie's $61.1 billion revenue, highlighting their commitment to maintaining leadership in immunology despite competitive pressures from rivals like Johnson & Johnson.Astellas has exceeded expectations with its cancer drug Vyloy overcoming a trial setback to quadruple sales in the third quarter fiscal year 2025 results. This success underscores the resilience and potential of innovative oncology treatments even when faced with clinical challenges.In financial markets, Veradermics successfully raised $256 million through its IPO, signaling strong investor interest in biotech firms with promising dermatological applications. Concurrently, Eikon Therapeutics marked the largest biotech IPO since 2024 with a $381 million listing on Nasdaq, reflecting renewed investor confidence in biotech ventures. Industry trends indicate a resurgence of interest in public markets exemplified by Eikon Therapeutics' upsized IPO alongside Veradermics' successful Support the show

Jerry spoke to Taoiseach Micheál Martin who is visiting the RDI Hub and Fexco in Killorglin and the Astellas plant. Jerry quizzed him about the St Patrick’s Day visit to the White House, housing, and the controversy over hundreds of thousands of euro being set aside this year to pay for new secretarial support for former presidents. Jerry asks where the request for this support came from.

In Episode 34, Dr. Aly-Khan Lalani and Dr. Christopher Wallis sit down with Dr. Fleshner to explore how an academic surgeon transitioned from traditional clinical and research roles into medical entrepreneurship. From tackling Canada's BCG shortage to co-founding a pharmaceutical company that sold for over a billion dollars, Dr. Fleshner walks through how he has built and scaled successful businesses in medicine.This episode delivers a masterclass on growing your impact beyond the clinic without losing your connection to medicine.The View on GU with Lalani & Wallis integrates key clinical data from major conferences and high impact publications, sharing meaningful take home messages for practising clinicians in the field of genitourinary (GU) cancers. Learn more about The View on GU: theviewongu.caThis podcast has been made possible through unrestricted financial support by AbbVie, Astellas, Ipsen, and Merck.

Good morning from Pharma Daily: the podcast that brings you the most important developments in the pharmaceutical and biotech world. Today, we delve into the latest innovations, strategic shifts, and regulatory updates shaping the industry.Johnson & Johnson's Tecvayli, a promising monotherapy for multiple myeloma, has shown remarkable efficacy in clinical trials. By reducing the risk of disease progression or death by 71% among patients resistant to anti-CD38 therapies, Tecvayli is setting a new standard in oncological treatment. This breakthrough underscores not only its potential as a standalone therapy but also highlights significant advancements in managing multiple myeloma—a cancer notoriously difficult to treat and manage.In regulatory developments, Sanofi has recently completed pricing negotiations with U.S. policymakers, gaining much-needed clarity on drug pricing regulations. However, Sanofi remains cautious as evolving policies could impact future operations. This scenario is emblematic of the broader industry challenge: navigating an uncertain regulatory landscape while maintaining stability and profitability in volatile markets.Astellas Pharma is adapting to its looming patent cliff for the prostate cancer drug Xtandi by focusing on innovation as a strategy for sustainable growth. With patent protection set to expire next year, Astellas emphasizes innovation over acquisitions for revenue protection, reflecting a broader industry trend towards long-term growth strategies rather than short-term gains.Meanwhile, Jazz Pharmaceuticals has sold a priority review voucher for $200 million, highlighting the persistent value of these vouchers which expedite FDA review processes. Such vouchers are becoming essential strategic assets as companies seek competitive advantages through faster market entry.On the technological front, Becton Dickinson's $110 million investment to expand syringe production capabilities demonstrates efforts to meet rising demand for injectable medications like GLP-1s. This initiative not only addresses immediate supply chain needs but also aligns with broader discussions about reshoring pharmaceutical manufacturing in the U.S., enhancing domestic production capabilities.Financially, strategic trends were a focal point at the recent J.P. Morgan Healthcare Conference. Although large-scale mergers and acquisitions were anticipated, none materialized, suggesting an evolving focus towards strategic partnerships and incremental innovations over mega-mergers among industry stakeholders.Looking ahead, AbbVie's projection of strong growth into the 2030s is driven by a robust pipeline and strategic initiatives. As companies navigate a complex landscape defined by innovation demands and regulatory changes, such outlooks underscore the industry's commitment to sustaining growth and innovation while improving patient outcomes through novel treatments and technologies.Italian pharmaceutical company Alfasigma's acquisition of rights to an injectable treatment for HSV encephalitis from a German biotech firm further exemplifies strategic investments aimed at expanding therapeutic portfolios with innovative solutions addressing critical health needs. This move is significant given the limited therapeutic options available for this severe condition.The Novo Nordisk Foundation's $860 million investment in Denmark's BioInnovation Institute underscores efforts to strengthen local biotech ecosystems. By nurturing homegrown biotech and deep tech companies, this initiative positions Denmark as a prominent hub for research and development while facilitating the translation of scientific discoveries into viable therapeutic solutions.Ocugen's promising mid-phase data for its gene therapy targeting eye disease positions it as a potential competitor against market leaders like Apellis and Astellas. The positive phase 2 results highlight gene therapy's growiSupport the show

Jessica Haynes, OD, of Charles Retina Institute, stresses that primary ECPs play a decisive role in early intervention for patients with geographic atrophy (GA). “First, the patient has to get a diagnosis,” she says. “Everything starts at that point.”This editorially independent content is sponsored by Astellas.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/BQU865. CME/MOC/AAPA/IPCE credit will be available until January 2, 2027.Innovative Approaches in Prostate Cancer: Bridging Genomics and Patient-Centric Treatment In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by independent educational grants from Astellas and Pfizer, Inc., AstraZeneca, Bayer HealthCare Pharmaceuticals Inc., Johnson & Johnson, and Novartis Pharmaceuticals Corporation.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/BQU865. CME/MOC/AAPA/IPCE credit will be available until January 2, 2027.Innovative Approaches in Prostate Cancer: Bridging Genomics and Patient-Centric Treatment In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by independent educational grants from Astellas and Pfizer, Inc., AstraZeneca, Bayer HealthCare Pharmaceuticals Inc., Johnson & Johnson, and Novartis Pharmaceuticals Corporation.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/BQU865. CME/MOC/AAPA/IPCE credit will be available until January 2, 2027.Innovative Approaches in Prostate Cancer: Bridging Genomics and Patient-Centric Treatment In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by independent educational grants from Astellas and Pfizer, Inc., AstraZeneca, Bayer HealthCare Pharmaceuticals Inc., Johnson & Johnson, and Novartis Pharmaceuticals Corporation.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/BQU865. CME/MOC/AAPA/IPCE credit will be available until January 2, 2027.Innovative Approaches in Prostate Cancer: Bridging Genomics and Patient-Centric Treatment In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by independent educational grants from Astellas and Pfizer, Inc., AstraZeneca, Bayer HealthCare Pharmaceuticals Inc., Johnson & Johnson, and Novartis Pharmaceuticals Corporation.Disclosure information is available at the beginning of the video presentation.

This content has been developed for healthcare professionals only. Patients who seek health information should consult with their physician or relevant patient advocacy groups.For the full presentation, downloadable Practice Aids, slides, and complete CME/MOC/AAPA/IPCE information, and to apply for credit, please visit us at PeerView.com/BQU865. CME/MOC/AAPA/IPCE credit will be available until January 2, 2027.Innovative Approaches in Prostate Cancer: Bridging Genomics and Patient-Centric Treatment In support of improving patient care, this activity has been planned and implemented by PVI, PeerView Institute for Medical Education, and ZERO Prostate Cancer. PVI, PeerView Institute for Medical Education, is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.SupportThis activity is supported by independent educational grants from Astellas and Pfizer, Inc., AstraZeneca, Bayer HealthCare Pharmaceuticals Inc., Johnson & Johnson, and Novartis Pharmaceuticals Corporation.Disclosure information is available at the beginning of the video presentation.

In this episode, Rob Jones, Professor and Honorary Consultant in Medical Oncology at the University of Glasgow, UK,  joins us to discuss his journey into oncology, the future of the specialty, and the evolving treatment landscape for genitourinary cancers. From clinical trial design and emerging therapies to European Society for Medical Oncology (ESMO) 2025 takeaways and thoughts on prostate cancer screening, Jones shares practical insights drawn from both research and frontline clinical practice.  Timestamps:     00:00 – Introduction  00:43 – Rob's journey into oncology  04:11 – The next generation of oncologists  08:16 – Clinical trial work  10:20 – Intersection of oncology and nephrology  14:05 – Cardiovascular toxicity  19:25 – European Society for Medical Oncology (ESMO) 2025 bladder cancer takeaways  23:53 – Challenges in designing trials  26:35 – Emerging treatment strategies  30:18 – Prostate cancer screening  34:00 – Magic wishes for healthcare    Disclosure: Jones has received research grants from Astellas, Clovis, Exelixis, Bayer, and Roche; advisory board fees from Janssen, Astellas, Bayer, Novartis, Pfizer, Merck Serono, MSD, Roche, Ipsen, and Bristol Myers Squibb; lecture honoraria from Astellas, Janssen, Bayer, Pfizer, Merck Serono, MSD, Roche, Ipsen, and Bristol Myers Squibb; support for conference attendance from Bayer and Janssen; and participated on a Data Safety Monitoring Board or Advisory Board for Roche.

Nicola Longo MD, PhD, and Mark Roberts, MDDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Doctors Roberts and Longo will discuss treatment with gene therapies.Question: Can one administer AAV-mediated gene therapy repeatedly?Mark Roberts, MDI think the traditional view would have been no. One can think of gene therapy as a silver bullet. Hopefully, it will reach its target. But if it's not effective, that bullet has been shot, the immunological response has occurred, and it means redosing, at least with that particular vector, may become difficult. But this situation is changing and evolving as we have better understanding of immunological modulation for repeat testing. We were discussing this yesterday evening, weren't we, Professor Longo?Nicola Longo MD, PhDCorrect. Basically, the current AAV-based gene therapy cannot be readministered. It is either effective, or it doesn't work. The other thing is that even though in theory, one could utilize a different AAV vector with different immunogenicity, there is many times cross-reactivity among the different adenovirus, adeno-associated viruses. Now, there are approaches in animal models in which you give a strong immune suppression to prevent the creation of the immune response against the adeno-associated virus, and at least in the animal model, it has been possible to give some of the gene therapy repeatedly.The second approach that is being tested is with gene correction therapy, in which by using an RNA guide and the CRISPR/Cas9 system delivered by lipid nanoparticles, you basically correct some of the effective genetic information. Obviously, since this is done by lipid nanoparticles and not by an AAV, the immunity that you create is really not there. You can give this one repeatedly, and in theory, it can be given more than one time. But again, you are absolutely correct. The current gene therapy cannot be given twice, and either it works or it doesn't work.Question:vWill gene-therapy-treated patients be able to go back to the standard of care or enzyme replacement therapy?Mark Roberts, MDI think when we're talking to patients about the potential benefits of gene therapy and the amelioration of the requirement to have these infusions on a regular basis of ERT, the hope is that will work, but they need to be reassured that we can potentially go back to the ERT. Gene therapy is an important treatment, but we don't know the destination of the patient at the beginning, and we have to make it available to them to go back to ERT.One of the crucial questions, of course, though, is the basis of the immunological reaction that perhaps prevented the gene therapy being effective. If it's against the viral vector, well, okay. If it's against the transgene, not great. If it's against the functional protein, that becomes more difficult. It is somewhat, I think at this time, to be fair to say to patients, think of gene therapy as a trial treatment. It is somewhat a leap of faith and an important observation, of course, for the patient community, but just be aware there may be downsides.Nicola Longo MD, PhDThey totally agree with Dr. Roberts. In general, they should be able to go back to enzyme replacement therapy if the gene therapy is not effective. However, what we are starting to appreciate is that we need to understand the immune response, not just to the enzyme replacement therapy, but also to gene therapy. What this field is doing is forcing geneticists to deal with the immune response. I feel that historically has not been dealt together. The two things need to be integrated. The advantage of the gene therapy is that the protein is produced endogenously. There should be the development of some degree of tolerance with time in the body towards the endogenous continuous production of a protein.Now, will that happen all the time? I still do not know. Again, we need to understand much better what is the integration of the immune system with the response to gene therapy in the ongoing clinical trials.

Nicola Longo MD, PhD, and Mark Roberts, MD Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfesor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025 and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice.The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Roberts will discuss vectors, different strategies, modes of administration and targets in gene replacement therapies.Mark Roberts, MDNow in the broader sense, gene replacement therapy seeks to actually deliver genetic material directly into the host cell to influence gene expression. In the most simple idea, one of course has a vector, this is most commonly but not exclusively a virus, which can then be given intravenously for example, and can hope to potentially correct the condition within the individual cells using novel transgenes. Suitable candidate conditions for this as examples of genetic conditions are now well understood. And crucially, this applies not only towards some more recessive, but dominant and even accident conditions.Across the piece, one can see for example, mitochondrial problems, spinal muscular atrophy as is well known, X-linked myotubular myopathy, Duchenne muscular dystrophy, a very common condition affecting one in 3000 male individuals, Pompe disease of course, an important focus of the meeting here, but other very common conditions, for example, cystic fibrosis, immunological conditions and perhaps obviously very crucial in early work on gene therapy, hemophilia.Let's now think about the approaches to gene therapy. One can seek to work at the DNA level and gene replacement. In essence, one is trying to put a new transgene through into the nucleus that will ultimately be transcribed and translated and produce the important functional protein that is lost. Gene editing which is a very exciting new technology or CRISPR technology actually seeks to actually modify in vivo the actual mutations that are responsible for the pathogenic production of abnormal proteins and correcting these and actually producing a more normalized protein.But of course there are also RNA approaches where one seeks to actually repair the mRNA transcripts copied from the mutated gene. For example, this may be a novel approach that could be extremely useful in myotonic dystrophy, a multisystem condition. When we talk about the viral vectors, predominantly we're talking about viruses. Those such as adenoviruses and AAV viruses which have the virtue of not integrating into the host genome or at least not in a large amount, and those which deliberately seek to integrate into host genome such as retroviral or lentiviral systems that may be particularly useful for ex vivo systems.There are of course other ways to get genetic payloads into the nucleus, various polymers, nanoparticles and even cell penetrating peptides. Nanoparticles in particular is certainly on the ascendant. That being said, in a recent review of the clinical trials in gene therapy, it was certainly the viral vectors that stood out both in direct gene replacement with lentivirus and AAV, but also actually as delivery systems, for example, for gene editing. An example of what one is seeking to do with AAV, so of course one seeking to remove the native DNA, insert the new transgene directly into the vector and of course keen to make sure that there's a high transmission into the capsid producing a recombinant AAV, which then can be given as a treatment and hopefully produce a therapeutic increase in the functional protein that is deficit in the disorder.In the next part, Dr. Roberts will discuss immune responses and other safety concerns related to gene therapies.

Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfesor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this 8-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th-7th 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Roberts will discuss immune responses and other safety concerns related to gene therapies.Mark Roberts, MDUndoubtedly, the immune system is a major issue in these patients. It would be fantastic if we could immunotolerize our patients and indeed prevent the rejection of the therapy. We've talked about the fact that these are viral vectors and of course there may be high seroprevalence of antibodies to these viral vectors, and it's very important in the pre-screening of patients who might be eligible to understand that at the beginning. These of course can have developed over the years and of course can be part of immunological memory and therefore extremely difficult and probably impractical to actually shift.On giving the treatment though as I think we're all aware there is this problem of the innate immunity and potential therefore for acute toxicities and then a learned or adaptive response with cytotoxic T cells and antibodies which may of course become high tighter neutralizing antibodies and potentially antibodies not only against the viral vector, even the functional protein, even the transgene are all theoretical possibilities with time. The capsid, the transgene, and even the protein product can all potentially induce an immunological event. Of course, all of these would lead to both potential patient changes and then a lack of efficacy of the treatment.Indeed, there have been some serious and indeed fatal problems in the gene therapy development program as I think we're all aware. Though many of these are thankfully been overcome. Spinal muscular atrophy has a gene therapy which is licensed, but there were early patients who actually had significant problems. A patient of just 6 months of age who developed kidney failure, two other patients who actually developed liver failure.In Duchenne muscular dystrophy, a very common condition, again there were significant issues and crucially in these patients who all have cardiomyopathy, it was heart failure and cardiac arrest that were big concerns and pulmonary edema and this was seen even with a CRISPR-based technology and is perhaps is best known but has been addressed the excellent myotubular myopathy patients, four patients died and crucially quite a long time after the gene therapy emphasizing the need to monitor these patients extremely carefully and these patients died of cholestatic liver failure albeit that they had a degree of liver dysfunction.That's changed our screening of course of patients, we're now all looking in myotubular patients for liver involvement and Rett syndrome as well. Now these immunoprophylaxis treatment regimes to hopefully try and reduce the immunological reaction against the gene are certainly evolving.This is just a summary of some of the other immunosuppressive regimes used in other disorders, for example, spinal muscular atrophy, but Pompe and MPS as examples of LSDs. Certainly these regimes will continue to evolve and are going to be very important in seeking to make sure that these treatments are effective. It reminds me somewhat of what's happened with enzyme replacement therapy that the use of these immunological strategies in infants has revolutionized the utility of those treatments in early patients.In the next part, Dr. Roberts will discuss lessons learned from gene therapy trials.

Nicola Longo MD, PhD, and Mark Roberts, MDNicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs.Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lessons learned from gene therapy trials.Mark Roberts, MDWhen we think about the challenges of actually doing clinical trials with these gene therapies, there's a huge development stage in terms of picking the right viral vector with the right surface receptor. That's a major piece of work. That can often take years. The preclinical work is obviously very important as indeed is understanding the natural history because it's really not practical to do placebo-controlled trials of gene therapies.In contrast to other studies, when we turn to phase 1 and phase 2, you'll notice that the patient numbers are often quite small. One is having to think carefully about surrogate measurements of response. Especially when in phase 3 studies, we may be thinking about withdrawing the existing, for example, enzyme replacement therapy because we believe the gene therapy will then be effective.That's just a few snapshots of where we've come and there's a lot more work to be done.In the next part, Dr. Longo will discuss the current treatment landscape and limitations in lysosomal disorders.

Nicola Longo MD, PhD, and Mark Roberts, MDDrs. Longo and Roberts discuss the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at WORLDSymposium 2025 in San Diego, California on February 4th-7th 2025 and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss the current treatment landscape and limitations in lysosomal disorders.Nicola Longo MD, PhDWhat I want to do today, is just place gene replacement therapy within the current landscape of lysosomal storage disorder treatment therapy. Gene therapy obviously has the potential of treating lysosomal disorder to correct the root cause of lysosomal storage disorder. The gene is defective, and what happen is that you can potentially either fix the gene or bypass the lack of the genetic product. But there are already therapies that are existing and are functioning. Obviously, in many cases, the lysosomal disorder is caused by defective production of an enzyme, which is defective.We can either replace the enzyme with enzyme replacement therapy, or provide chaperone for specific mutations that retain the synthesis of the enzyme, that however is not very functional. Another avenue that it is being reported is the utilization of substrate reduction therapy. A substrate accumulates, you prevent the synthesis of the substrate to reduce the accumulation of toxic material. What we know now is that this is not enough to produce many lysosomal disorders. In many cases, the lysosomal disorder result sometime in impairment of intracellular trafficking, and sometime in the function of other organelles.At the end, it results in the activation of the macrophagic system and inflammation. Already we have some therapy acting at this level. The end result of lysosomal storage disorder, there will be cell suffering and cell death, leading to a progression of the disease, and morbidity and mortality. Now, what therapy do we have available already? Obviously, hematopoietic stem cell transplantation has been around for quite some time.It has been the same thing that we do with gene therapy, except that instead of reintroducing the gene of the subject, we place gene of a subject who is not affected of the disease. This therapy has been proven effective in cases of MPS-1 and alpha-mannosidosis. But in many cases this has to be given way before symptoms start to be affected.Enzyme replacement therapy has been around for quite some time, starting with Gaucher disease, and now that it is available for a list of diseases that are there, so it's like Fabry, Gaucher, Pompe, different types of mucopolysaccharidosis, alpha-mannosidosis, acid lipase deficiency, 1 neuronal ceroid lipofuscinosis, and Niemann-Pick type A and B.Obviously the advantage of this therapy, they give back the enzyme that it is defective. But the disadvantage that many time they cannot enter specialized areas such as the brain. There is already the second generation of enzyme replacement therapy that it is available. With this second generation, some of the newer drugs are more effective in terms of cellular uptake, or in terms of having a prolonged half-life and prolonged activity.Then there are pharmacological chaperone therapy, and the one which is FDA approved is migalastat for Fabry disease, under study is ambroxol for Gaucher disease. The disadvantage of this therapy that only a selected number of mutations respond to this therapy.Substrate reduction therapy has been introduced for Gaucher disease many years ago with miglustat, and it was followed by eliglustat. Both of them are effective, and some of them more effective than other, simply because of the fewer side effects of eliglustat as compared to miglustat. But at the same time, eliglustat does not pass the blood brain barrier.Finally, the newer agents that are already administered, N-acetyl-L-leucine and arimoclomol, both approved for Niemann-Pick type C, they act more on the downstream effect of the lysosomal storage disorder, either by stabilizing neuronal cell activity or by reducing the inflammation that is present in the brain.In the next part, Dr. Longo will discuss gene replacement therapy in lysosomal disorders.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight part podcast series. This is derived from the symposium that was presented at World Symposium 2025, in San Diego, California, on February 4th through 7th, 2025, and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established, and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss gene replacement therapy in lysosomal disorders.Nicola Longo MD, PhDLet's go back a second to gene therapy. Gene therapy obviously has the potential of answering many of the questions that we still have open in lysosomal disorder because they could restore the activity of the lysosome pretty much in the whole body, or at least in multiple tissues. As you have seen, gene therapy can be done ex vivo where we take cells from the affected patient, we correct the gene, or we put an extra gene that it is functional. Then we put them back by doing a bone marrow transplant, basically creating space for the cells that have been genetically modified to correct the lysosomal defect. The biggest approach this is done usually by lentiviruses that they integrate inside the genome.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only. This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses. The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas. In this part, Dr. Longo will discuss ongoing gene therapies in lysosomal disorders.Nicola Longo MD, PhDI'm going to present to discuss some example of ongoing gene therapy for lysosomal disorder. There are gene therapy in development for both Fabry disease and some of this involve ex vivo gene therapy, many others involve systemic administration with an AAV, Gaucher disease type 1 that affect the periphery, and Gaucher disease type 2, where the replacement should occur within the central nervous system because this condition affects the brain. There is already one approved gene therapy for lysosomal disorder, which is for the early onset metachromatic leukodystrophy. This has been approved both in Europe and now even in the United States, which consists of ex vivo gene therapy with the administration of an extra gene that restore the function of the defective enzyme. Now there are many others that are ongoing for the same indication. There are gene therapy programs for GM1 and GM2 gangliosidosis, and at least one for Krabbe disease. It is important to know that some of these condition are actually included in the recommended uniform screening panel. Basically, we would have access to patients in a timely manner for some of these conditions. Then there are several gene therapy under development for the mucopolysaccharidoses, including MPS-IH, MPS-II, MPS-IIIA and MPS-IV.There are different type of lysosomal disorders, the one caused by mutation, integral membrane protein, not enzyme within the lysosome, but protein that are present on the membrane of the lysosome. This gene therapy that have been tested, it is for cystinosis, that it is caused by a defective lysosomal and for Danon disease, which is caused by a deficiency of an integral membrane part. Finally, one lysosomal disorder, which obviously seems a metabolic condition, but it is really not, is glycogen storage disease type 2 or Pompe disease, in which there is the intralysosomal accumulation of glycogen. There are several ongoing clinical trials to try to correct the problem in this condition.Now, I'm going to discuss some of the most advanced program in the lysosomal storage disorder. This include one for Fabry, which is on an accelerated approval pathway with phase 1 and 2 data, one for Gaucher disease type 1. Obviously, I'm going to discuss the one that has been already approved for metachromatic leukodystrophy. There is one for Hunter syndrome, and the difference of the one for Hunter syndrome, it is an example of the direct administration of gene therapy within the central nervous system.Finally, there is one ongoing for glycogen storage disease type 2 or Pompe disease in adult patients. In gene therapy for metachromatic leukodystrophy, it was the first gene therapy approved for lysosomal disorder in human, and this requires harvesting the CD34 cell from affected patient and then introducing the [inaudible 00:04:32] gene back in this cell, and then placing them back inside the patient again. This has been very effective in patients who were treated early, and obviously, the treatment needs to occur before there is irreversible brain damage in this patient.In the next part, Dr. Roberts and Longo will discuss treatment with gene therapies.

Nicola Longo MD, PhDProfessor and Vice Chair of Human Genetics,Allen and Charlotte Ginsburg Chair in Precision Genomic Medicine,Division of Clinical Genetics, Department of Human Genetics,University of California at Los Angeles (UCLA), Los Angeles, CA, USAMark Roberts, MDProfessor and Consultant Neurologist,University of Manchester, Manchester, UKResearch Lead for Adult Metabolic Medicine at Salford Care Organisation, Manchester, UKDrs. Longo and Roberts discussed the current status of gene therapies in rare neuromuscular disorders in this eight-part podcast series. This is derived from the symposium that was presented at World Symposium 2025 in San Diego, California on February 4th through 7th, 2025, and is intended for healthcare professionals only.This podcast includes information about investigational compounds that do not yet have a regulatory approval or authorization for a specific indication. The safety and efficacy of the agents under investigation have not been established, and contents of this podcast shall not be used in any manner to directly or indirectly promote or sell the product for unapproved uses.The views, thoughts, and opinions expressed in this presentation belong solely to the author and are subject to change without notice. The contents of this presentation do not constitute an endorsement of any product or indication by Astellas.In this part, Dr. Roberts will discuss lysosomal disorders and the potential for gene therapies.Mark Roberts, MDI'm going to give an overview of what is gene therapy, emphasizing the current challenges and the development issues and needs that there will be as we try and enable gene therapy for our patients, particularly those with lysosomal storage disorders.I'm going to try and make a case for why lysosomal storage disorders are an extremely good group of conditions for the potential benefits of gene modifying therapies. Firstly, whilst we all recognize that these conditions are inherently individually rare, they're certainly severe. Collectively, with over 70 LSD disorders, 1 in 5,000 may be afflicted by these conditions ultimately in their life and can be detected, for example, by newborn screening programs.Secondly, there's certainly a significant clinical burden with these patients with the current standard of care, so a large unmet need exists. Existing enzyme replacement therapies have undoubtedly changed the natural history of many of these conditions, but there are limitations and often initial benefits and later deteriorations.Unfortunately, for most lysosomal storage disorders, it's only symptomatic treatments and indeed, care that is available for these patients with no specific treatment. Thirdly, these conditions are extremely well-characterized, monogenic singleton and problems of inborn errors of metabolism. We know the functional protein that is deficient in these conditions. Because of that, and knowing that these are critical for lysosomal function, and using preclinical models, we can model the potential benefits of gene therapies very well in a number of systems, including, of course, soon, muscle chip experiments as well.Finally, with these conditions, they may potentially be really useful targets whilst not perhaps curing the condition, at least ameliorating the phenotype, and enabling the addition of other treatments as well, potentially. I've noted, some of these therapies can be directly delivered to certain tissues, so muscle tissue, which is my main interest, but also, crucially, the central nervous system, which is very important when we consider ameliorated phenotypes, for example, treated by enzyme replacement therapy, but where the children who become the adults have significant learning disability as a major component to their problems.In the next part, Dr. Roberts will discuss vectors, different strategies, modes of administration, and targets in gene replacement therapies.

‍ ‍Drs. Akshay Thomas and Sarwar Zahid join for a journal club episode discussion of three recent publications:‍ ‍ ‍ ‍Fellow Eye PVD (https://journals.sagepub.com/doi/full/10.1177/24741264251379842)‍ ‍Syfovre versus Izervay (https://journals.sagepub.com/doi/full/10.1177/24741264251379842)‍ ‍Litigation Involving Intravitreal Injections (https://www.ophthalmologyretina.org/article/S2468-6530(25)00439-7/abstract)‍ ‍ ‍ ‍Disclosures: Dr. Sridhar has consulted for Apellis and Astellas in the past 3 years. ‍ ‍

When Procter & Gamble asked Atsushi Kitamura to move from finance analysis into running a manufacturing plant, he didn't hesitate. “They always give me next challenge to stretch me,” he tells us. Managing one of P&G's large diaper plants in Japan forced him to apply finance in real-time operations—a proving ground that shaped his comfort with change and appetite for transformation.That readiness carried him from consumer goods to logistics, restaurants, and electronics before arriving at Astellas Pharma, where the stakes are now measured in science and strategy. When he joined in 2023, Astellas had just completed a $6 billion acquisition that shifted it from a net-cash to a net-debt position. Hired to “put financial disciplines and make the balance sheet stronger,” Kitamura tells us he views the moment as a “transformative timing” for the company. Loss of exclusivity on a prostate-cancer drug representing “more than 40 percent of our revenues” demands reinvention.His three-part playbook focuses on growing core brands, investing in science creation, and executing what he calls “sustainable margin transformations.” The approach has begun to pay off—“top line 19 percent, profit 40 percent,” he tells us—signaling a finance organization in renewal.Now he's turning to technology to sustain that momentum. Describing the shift from RPA to “agentic AI,” Kitamura explains, “I just ask agent AI to do that booking.” He calls it a tipping point that will “change significantly the way how we work.”Still, he adds, leadership begins with listening. “Don't pretend I know everything,” he tells us. For Kitamura, finance transformation starts not with machines or metrics—but with humility.