In a new Nature Portfolio Biotechnology feature, researchers from Mass General Brigham discuss the important role academic medical centers play in the creation and implementation of new cell and gene therapies (CGTs). Many times, when a new CGT is created, it can be hard to translate the treatment into patient care because of the long development process, high cost, and regulatory landscapes. The authors of this piece discuss how academic medical centers can play a crucial role in overcoming CGT development hurdles by adopting a model such as the RISE model (resource sharing, interdisciplinary collaboration, sustainable funding, and education) that is implemented at Mass General Brigham’s Gene and Cell Therapy Institute. A model such as this can help institutions bring life-saving treatments to patients in need in a more efficient way. Mass General Brigham authors included Nandhitha Uma Naresh, Dana Hammill, Roger J. Hajjar, and Nathan L. Yozwiak.
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Don’t miss this must-read on cell and gene therapy by Mass General Brigham Gene and Cell Therapy Institue leadership. I have commented many times on how the GCT system is broken, this paper proposes some novel solutions.
In a new Nature Portfolio Biotechnology feature, researchers from Mass General Brigham discuss the important role academic medical centers play in the creation and implementation of new cell and gene therapies (CGTs). Many times, when a new CGT is created, it can be hard to translate the treatment into patient care because of the long development process, high cost, and regulatory landscapes. The authors of this piece discuss how academic medical centers can play a crucial role in overcoming CGT development hurdles by adopting a model such as the RISE model (resource sharing, interdisciplinary collaboration, sustainable funding, and education) that is implemented at Mass General Brigham’s Gene and Cell Therapy Institute. A model such as this can help institutions bring life-saving treatments to patients in need in a more efficient way. Read more: https://lnkd.in/g3ys_Asb (Mass General Brigham authors included Nandhitha Uma Naresh, PhD, Dana McAfee Hammill, Roger Hajjar, and Nathan Yozwiak.)
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In a new Nature Portfolio Biotechnology feature, researchers from Mass General Brigham discuss the important role academic medical centers play in the creation and implementation of new cell and gene therapies (CGTs). Many times, when a new CGT is created, it can be hard to translate the treatment into patient care because of the long development process, high cost, and regulatory landscapes. The authors of this piece discuss how academic medical centers can play a crucial role in overcoming CGT development hurdles by adopting a model such as the RISE model (resource sharing, interdisciplinary collaboration, sustainable funding, and education) that is implemented at Mass General Brigham’s Gene and Cell Therapy Institute. A model such as this can help institutions bring life-saving treatments to patients in need in a more efficient way. Read more: https://lnkd.in/g3ys_Asb (Mass General Brigham authors included Nandhitha Uma Naresh, PhD, Dana McAfee Hammill, Roger Hajjar, and Nathan Yozwiak.)
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Check out my latest article published in collaboration with our fabulous GCTI team!
In a new Nature Portfolio Biotechnology feature, researchers from Mass General Brigham discuss the important role academic medical centers play in the creation and implementation of new cell and gene therapies (CGTs). Many times, when a new CGT is created, it can be hard to translate the treatment into patient care because of the long development process, high cost, and regulatory landscapes. The authors of this piece discuss how academic medical centers can play a crucial role in overcoming CGT development hurdles by adopting a model such as the RISE model (resource sharing, interdisciplinary collaboration, sustainable funding, and education) that is implemented at Mass General Brigham’s Gene and Cell Therapy Institute. A model such as this can help institutions bring life-saving treatments to patients in need in a more efficient way. Read more: https://lnkd.in/g3ys_Asb (Mass General Brigham authors included Nandhitha Uma Naresh, PhD, Dana McAfee Hammill, Roger Hajjar, and Nathan Yozwiak.)
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To propel regenerative medicine into a new era of possibilities, we must find ways to deliver effective #CellTherapies at scale. President of the Astellas Institute for Regenerative Medicine, Erin Kimbrel, shared her insights with Cell & Gene, on the innovative technologies Astellas is employing to deliver these next-generation therapies to more patients, faster: https://bit.ly/3WRqrw7. #ScienceFirstForPatients #ChangingTomorrow
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🚀 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
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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
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🚀 Exciting Developments in Cell & Gene Therapy! 🚀 The cell & gene therapy landscape is buzzing with two major acquisitions in the last week: 1. Roche's acquisition of Poseida Therapeutics, Inc. Therapeutics for up to $1.5B. This strategic move will bolster Roche's capabilities in off-the-shelf CAR-T therapies, aiming to democratize access to these life-saving treatments. Poseida's innovative non-viral technology platform, rich in T stem cell memory cells, will hopefully enhance the safety and efficacy of CAR-T therapies, potentially benefiting a broader patient population. 2. Meanwhile, Novartis has acquired Kate Therapeutics in a deal valued at up to $1.1B. This acquisition aligns with Novartis' commitment to advancing gene therapies for neuromuscular diseases. Kate Therapeutics' cutting-edge AAV-based gene therapy platform and promising preclinical candidates for conditions like Duchenne muscular dystrophy and myotonic dystrophy type 1 will strengthen Novartis' pipeline and innovation in gene therapy. These acquisitions underscore the relentless pursuit of innovation in the biotech industry, aiming to bring transformative therapies to patients worldwide. #GeneTherapy #HealthcareInnovation #CART
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10 Life Science and Biotech Trends to Watch in 2025 🧬 Key trends for 2025 include personalized medicine, gene therapies, lab sustainability, and advancements in mRNA-based therapeutics. These innovations are set to transform health care and improve patient outcomes. https://lnkd.in/ehrMzhuf
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Thank you to Erin Harris for a great article on 2025 Outlook for Cell and Gene Therapy (CGT): Key Trends to Watch The CGT industry is set for transformative advancements in 2025, focusing on: 1️⃣ Non-Oncology Indications: Broader applications in genetic disorders, autoimmune diseases, and cardiovascular conditions, with scalable manufacturing and affordability driving accessibility. 2️⃣ In Vivo Gene Editing: Enhanced precision and delivery methods, such as lipid nanoparticles and novel AAV capsids, unlocking potential for complex diseases. 3️⃣ Point-of-Care (POC) Innovations: Miniaturized bioreactors, automation, AI-driven optimization, and decentralized production models, enabling cost-effective and accessible therapies globally. It is noted that resilience, scalability, and innovation are essential for advancing CGT in a challenging biotech landscape. The future of CGT promises revolutionary treatments and broader access to life-saving therapies. #CellAndGeneTherapy #BiotechInnovation #GeneEditing #PointOfCare #HealthcareTransformation #PersonalizedMedicine #LifeSciences
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