HQM Research Center’s Post

The Importance of Specialized Training in Advanced Cellular Therapy Management. This new article explores the importance of specialized training in managing advanced cellular therapies, with a focus on CAR-T therapies. It discusses how these therapies are revolutionizing the treatment of certain forms of #cancer, the skills required to manage a #CART center, the importance of a robust quality system, and the challenges in managing clinical trial projects. Additionally, it emphasizes the need for continuous improvement to maintain high standards of quality and #safety. 📖 Read the full article to delve deeper. Obtain the qualification of Car-T Specialist and Gene Therapy Expert through a comprehensive training program that covers all aspects of treatment, from theory to practice, including organizational protocols and current regulations. You will also be officially recognized in the international registry. 👨🏫 Speakers: Dr. vincenzo iaconianni, CEO of ICMED srl, internationally recognized expert in quality system design. Dr. Giorgia Saporiti, hematologist, director of the clinical unit at Policlinico di Milano, affiliated with the Milano Transplant Network. Dr. @Alessandra Bo, director of the cell manipulation laboratory at IRCCS Policlinico San Martino in Genoa. Prof. Vincenzo Salvatore, Full Professor of European Union Law at the University of Insubria, Varese, expert in ATMP. 📚 Program: What is a gene therapy product and CAR-T? Drug or Tissue? Production or Administration European and national regulatory framework The role of the Cell Therapy and Car-T Specialist from the Clinical Center perspective The role of the Cell Therapy and Car-T Specialist from the Pharmaceutical Company perspective 📅 When: Friday 27/09/24: ⏲10-13 & 14-17  / Saturday 28/09/24: ⏲9-13 Friday 04/10/24: ⏲10-13 & 14-17 / Saturday 05/10/24: ⏲9-13 🌐 Mode: In-person at Hotel The Building, via Montebello n. 126 – ROME 📜 Certification: Car-T Specialist and Gene Therapy Expert Certificate Registration in the international registry of Car-T Specialist and Gene Therapy Experts More details and registration: https://lnkd.in/d-NmM7tz #InducedPluripotentStemCells #AutologousAdultStemCells #BoneMarrowTransplantation #MultipotentProgenitorCells #EmbryonicStemCells #Neurogenesis #BoneTransplantation #Cytotherapy #StemCellsDevelopment #CancerStemCells #TransplantationLymphoma #PeertechzPublications #EmbryonicStemCells #BlastocystStemCells #CellBasedTherapies #HematopoieticStemCell #MesenchymalStemCells #MultipotentStemCells #MyeloidStemCells #PluripotentStemCells #StemCellGenomes #StemCellPreservation #StemCellTransplantation #EmbryonicStemCells #CellBasedTherapies #CordBloodStemCells #StemCellAndDiseases #StemCellBiology #StemCellGeneration #StemCellMarkers #StemCellRegulationAndRepair #StemCellResearch #StemCellsInRegenerativeMedicine #StemCellsInToxicityDetection

🚀 𝗔𝗱𝘃𝗮𝗻𝗰𝗶𝗻𝗴 𝗶𝗣𝗦𝗖-𝗗𝗲𝗿𝗶𝘃𝗲𝗱 𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀: 𝗞𝗲𝘆 𝗜𝗻𝘀𝗶𝗴𝗵𝘁𝘀 𝗳𝗿𝗼𝗺 𝘁𝗵𝗲 𝗟𝗮𝘁𝗲𝘀𝘁 𝗜𝗻𝗱𝘂𝘀𝘁𝗿𝘆 𝗥𝗲𝘃𝗶𝗲𝘄! 🌿 The recent review by the JSRM-ISCT, International Society for Cell & Gene Therapy iPSC Committee delves into the critical 𝘤𝘩𝘢𝘭𝘭𝘦𝘯𝘨𝘦𝘴 𝘢𝘯𝘥 𝘰𝘱𝘱𝘰𝘳𝘵𝘶𝘯𝘪𝘵𝘪𝘦𝘴 𝘧𝘰𝘳 𝘪𝘗𝘚𝘊-𝘣𝘢𝘴𝘦𝘥 𝘵𝘩𝘦𝘳𝘢𝘱𝘪𝘦𝘴. Here are 3 take-home messages: 𝟭- 𝗛𝗮𝗿𝗺𝗼𝗻𝗶𝘇𝗶𝗻𝗴 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗦𝘁𝗮𝗻𝗱𝗮𝗿𝗱𝘀 🌍: Navigating the diverse global regulatory frameworks is essential for ensuring the safe and efficient development of iPSC-derived therapies. Early engagement with regulatory bodies can help streamline product approval. 𝟮- 𝗙𝗼𝗰𝘂𝘀 𝗼𝗻 𝗤𝘂𝗮𝗹𝗶𝘁𝘆 𝗖𝗼𝗻𝘁𝗿𝗼𝗹 & 𝗖𝗼𝘀𝘁 𝗠𝗮𝗻𝗮𝗴𝗲𝗺𝗲𝗻𝘁 💡: The path to commercialization requires rigorous quality control and thoughtful cost-of-goods (COGs) strategies. Optimizing manufacturing processes through advanced automation can significantly reduce expenses, making therapies more accessible. 𝟯- 𝗜𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝘃𝗲 𝗔𝗽𝗽𝗿𝗼𝗮𝗰𝗵𝗲𝘀 𝘁𝗼 𝗧𝘂𝗺𝗼𝗿𝗶𝗴𝗲𝗻𝗶𝗰𝗶𝘁𝘆 𝗥𝗶𝘀𝗸𝘀 🔬: Addressing the risks of genomic instability and tumorigenicity remains a major challenge. Collaborative efforts and new assay developments are key to ensuring the safety and efficacy of these transformative treatments. #CellTherapy #iPSC #RegenerativeMedicine #BiotechInnovation #QualityControl #GlobalHealth https://lnkd.in/eHXRz7e2 Stephen Sullivan, PhD, MBA, FRSM iPSirius Lindville Bio Veronica Falco Dominic Wall Cell Therapies Pty Ltd Nathan Smith

👂🌟 Regeneron's DB-OTO Gene Therapy Shows Promise in Treating Genetic Deafness! 🔍 In a ground-breaking development, Regeneron Pharmaceuticals' investigational gene therapy, DB-OTO, has demonstrated significant efficacy in treating profound genetic deafness in children, as revealed in the Phase 1/2 CHORD trial. DB-OTO, a cell-selective adeno-associated virus (AAV) gene therapy, targets variants of the otoferlin gene responsible for congenital hearing loss. Two young participants showed promising results: one child achieved normal hearing levels within 24 weeks, while the other displayed initial improvements after six weeks. Audiometry tests indicated an average 84dB improvement in key speech frequencies for the first participant and a 19dB average improvement for the second. 💡 These findings suggest DB-OTO's potential as a transformative therapy for otoferlin-related deafness, offering hope for enhanced speech development and quality of life. Regeneron's commitment to innovative medical solutions continues to drive progress in addressing unmet medical needs. Clinical trial investigator and Columbia University’s Department of Otolaryngology – Head & Neck Surgery chair Lawrence Lustig said: “These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individual’s development, especially since early intervention is associated with better outcomes for speech development.” To read similar ground-breaking news, subscribe to the World Pharmaceutical Frontiers page!! #Regeneron #GeneTherapy #CHORDTrial #HearingLoss #MedicalAdvancements #worldpharmaceuticalfrontiers

Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

It was an honor not only to present, but also to connect with the experts and patient advocates advancing the field of gene therapy for rare disorders at the 7th Gene Therapy for Rare Disorders Summit in Boston last week. I can't believe how much we accomplished in two years. 5th Gene Therapy for Rare Disorders Summit was the first face-to-face meeting after the COVID shutdown, attracting a host of experts. We had just completed plasmid manufacturing at VGXI’s facility, and the company decided to invest in my development by inviting me to the conference. I cannot stress enough how much this one meeting helped in the development of AGU gene therapy. At the conference, I was able to meet the people who helped, guided, mentored, and supported our development over the last two years. We would not be at the point of submitting an IND (Investigational New Drug application) for AGU gene therapy if these connections had not happened. Here are my suggestions based on our experience: For big and small pharmaceutical and biotech  companies : please consider sponsoring a patient foundation's attendance at a conference. Such support not only accelerates the advancement of groundbreaking treatments, but also helps in reducing the development costs, as the shared knowledge and insights can help avoid costly mistakes. Additionally, this collaboration fosters a unique synergy between researchers, patients, and industry stakeholders, promoting a more inclusive and informed approach to therapy development. By facilitating these crucial conversations and exchanges, your involvement could be the catalyst for the next major advancement in rare disease therapy. Lets work towards "One company = One patient advocate" sponsoring equation. And for patient foundations: if there is no sponsor available, reach out to the Hanson Wade Group conference manager. They will work with you to help you attend the conference. I reached out to different conference organizers, and Hanson Wade Group is the only company that I found supports patient foundations working to develop cures for ultra-rare diseases. Thank you, Nick Mayne, for giving me the opportunity to speak and Christy Franco, Ph.D. for your continues support of rare diseases. #agucure #raredisease #genetherapy #genetics #raretrait #raregeneticdiseases #aspartylglucosaminuria Terry Pirovolakis, Amber N. Freed, Sierra Phillips, Effie Parks Precision For Medicine, Azzur Group, AGC Biologics, Advanced Medicine Partners, Matica Biotechnology, Inc., VintaBio, Worldwide Clinical Trials, Cryoport Systems, Forge Biologics, Paysign, Inc., Kyfora Bio , SGS

🚨 Important Update: Protecting Patient Safety in Cell and Gene Therapy 🚨 The recent Utah bill (SB0199), which took effect on May 1, 2024, raises significant concerns about undermining the US FDA's authority to regulate placental stem cell therapies. At BioVivium, we firmly believe in the importance of rigorous regulatory oversight to ensure the highest standards of patient safety and treatment efficacy. 🛡️ By challenging the FDA's regulatory power, this bill poses significant risks for patient safety and the entire field of cell and gene therapy. The FDA plays a crucial role in safeguarding public health, and any legislative actions that weaken its authority could have serious implications for patient care. We stand with ISCT in advocating for policies that prioritize patient well-being and support the robust regulation of medical treatments. 📢 Stay informed and join us in supporting the FDA's mission to protect patients. Your health and safety are our top priorities! #PatientSafety #FDA #HealthCare #BioVivium #RegulatoryStandards #MedicalEthics #CellTherapy #GeneTherapy 🔗https://lnkd.in/ecGeMT9v #PatientSafety #FDA #HealthCare #BioVivium #RegulatoryStandards #MedicalEthics #CellTherapy #GeneTherapy

Cell and gene therapy poses evolving challenges. The current article summarizes the discussions held by European Regional Committee of the International Society for Cell & Gene Therapy and the European Society for Blood and Marrow Transplantation (The EBMT) on the current challenges in this field, focusing on the European setting. This article emphasizes the imperative assessment of real-world cell and gene therapy activity, advocating for expanded registries beyond hematopoietic transplantation and chimeric antigen receptor–T-cell therapy. Accreditation's role in ensuring standardized procedures, as exemplified by #JACIE (The Joint Accreditation Committee of ISCT, International Society for Cell & Gene Therapy-Europe and EBMT), is crucial for safety. Access to commercial products and reimbursement variations among countries underscore the need for uniform access to advanced therapy medical products (#ATMPs). Academic product development and point-of-care manufacturing face barriers to patient access. Hospital Exemption's potential, demonstrated by some initial experiences, may increase patient accessibility in individual situations. Regulatory challenges, including the ongoing European ATMPs legislation review, necessitate standardized criteria for Hospital Exemption and mandatory reporting within registries. Efforts to combat unproven therapies and fraud involve collaboration between scientific societies, regulatory bodies and patient groups. Finally, is important to highlight the vital role of education and workforce development in meeting the escalating demand for specialized professionals in the ATMP field. Collaboration among scientific societies, academic institutions, industry, regulatory bodies and patient groups is crucial for overcoming all these challenges to increase gene and cell therapy activity in Europe.

Novartis has acquired Kate Therapeutics, a San Diego-based #genetherapy company, in a deal valued at up to $1.1 billion. Kate Therapeutics specializes in #preclinical programs targeting Duchenne muscular dystrophy (#DMD), facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (#DM1). The company employs its proprietary DELIVER platform, which utilizes directed evolution of adeno-associated virus (#AAV) capsids to create #liver-#detargeted, muscle-tropic #vectors. Acknowledging recent challenges in muscular dystrophy treatments, such as Pfizer's investigational gene therapy for DMD failing to demonstrate efficacy in a Phase 3 trial, Novartis CEO Vas Narasimhan emphasized the company's commitment to learning from past experiences in the #musculardystrophy domain. By acquiring Kate Therapeutics, Novartis aims to leverage innovative #delivery #technologies to overcome previous obstacles in gene therapy for #neuromuscular #diseases. Muscular Dystrophy Association Muscular Dystrophy UK Muscular Dystrophy Canada Facioscapulohumeral Muscular Dystrophy Duchenne UK Duchenne Heroes #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting https://lnkd.in/giRnS_6G

🚀 Advanced Modalities in 2024: Challenges and Opportunities While PTC’s recent FDA approval of a gene therapy for a fatal enzyme disorder (AADC deficiency) marks progress, the cell and gene therapy landscape remains challenging, especially on the investment front. We recently took a look back at the news from 2024 in CGT and RNA space, some key takeaways. 🔬 Gene Replacement Therapies (AAV): Big pharma interest is strong, with Astellas investing in AviadoBio and Roche partnering with Dyno Therapeutics for better AAVs (delivery is still key!). Yet, even validated targets face hurdles—Pfizer's Phase III DMD trial failed to show functional improvements despite significant dystrophin production. 🧠 Therapeutic Areas (TAs): Ophthalmology, CNS, metabolic, and musculoskeletal indications dominate, while cardiovascular and even renal diseases are gaining traction. Emerging players like Purespring are advancing kidney-targeted therapies. 🧬 Gene Editing Progress: Following the first FDA approval of a gene editing therapy in December 2023, in vivo approaches are accelerating – some companies we are watching and their programs: Intellia (HAE), CRISPR Therapeutics’s cardio programs, Editas Medicine (pivot to in vivo therapies) 🔒 Safety Concerns Persist: Approved therapies like Skysona for CALD still face challenges with cases of blood malignancies reported. Yet, for conditions like CALD, these therapies represent transformative hope. 📈 RNA Editing First Clinical Data and New Modalities: Wave Life Sciences delivered the first therapeutic RNA editing data in AATD (>100k US patients). Epigenome editing is nearing clinical trials, with Tune Therapeutics receiving CTA approval for HBV studies. Circular RNA technologies are exploring in vivo CAR applications with the potential to bypass conditioning regimens. 📉 Investment and Workforce Pressures: Layoffs and low funding dominate the sector. Even with a large initial backing, Tome Biosciences recently scaled back operations. Investors seem to favour lower-risk technologies with clearer market pathways. 🌍 Broader Indications on the Horizon: Orphan diseases remain a priority, but many companies are pushing toward larger patient populations (e.g., wAMD, HBV,...). What does this mean for the future of advanced modalities? Can these cutting-edge therapies scale to serve broader populations while addressing persistent challenges? Let’s discuss and feel free to reach out #GeneTherapy #Biotech #CellTherapy #GeneEditing #RNAEditing #AdvancedModalities #PharmaResearch #Biopharma #FutureOfMedicine

Can a single gene injection resurrect a failing heart – sounds like science fiction, isn’t it? Except, it’s not. It is a breakthrough in medical science unfolding right now. Researchers at the University of Utah's Nora Eccles Harrison Cardiovascular Research and Training Institute (CVRTI) have made groundbreaking advancements in treating heart failure through innovative gene therapy. This novel approach, focusing on the cardiac bridging integrator 1 (cBIN1) gene, has demonstrated unprecedented efficacy in reversing heart failure in a large animal model. The study, published in NPJ Regenerative Medicine, utilized a porcine model of non-ischemic heart failure with reduced ejection fraction (HFrEF). The therapy involves administering a harmless virus vector carrying the cBIN1 gene, which is delivered to cardiac cells via bloodstream injection. Key findings of the study: 1. Functional Improvement: The gene therapy resulted in a remarkable 30% improvement in heart function, significantly surpassing previous therapeutic attempts that typically achieved only 5-10% improvement. 2. Survival Rate: All treated subjects survived the entire six-month study duration, a notable outcome given the typically poor prognosis of untreated heart failure in this model. 3. Reverse Remodeling: The treatment induced reverse remodeling of the heart, with treated hearts exhibiting less dilation and thinning, more closely resembling non-failing cardiac morphology. 4. Molecular Mechanism: cBIN1 functions as a centralized signaling hub, regulating multiple downstream proteins and organizing critical heart cell functions. This scaffolding role of cBIN1 is hypothesized to be key to its therapeutic efficacy. 5. Microscopic Improvements: The therapy led to better-organized heart cells and proteins at the microscopic level, suggesting a comprehensive restoration of cardiac ultrastructure. The researchers, led by Dr. Robin Shaw and Dr. TingTing Hong, posit that cBIN1's role as a master regulator of heart cell architecture could introduce a new paradigm in heart failure treatment, directly targeting heart muscle function. While these results are encouraging, human clinical trials are still needed to determine the therapy's effectiveness and safety in people. In collaboration with TikkunLev Therapeutics, the team is adapting the therapy for human application, with plans to seek FDA approval for clinical trials in fall 2025. This research represents a potential paradigm shift in cardiac medicine, as the ability to halt the progression and potentially reverse cardiac damage could revolutionize treatment strategies and significantly improve patient outcomes. #MedicalInnovation #CardiacResearch #GeneTherapy https://lnkd.in/ewkQeSM7