Cell&Gene Consulting Inc.

Whatever he says! 😅 😉 👇 👇 👇

🚀 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

🧑🔬 "Unless you know for sure that your technology and manufacturing process cannot supply Phase 3 trials or support the initial commercial product launch, 𝗶𝘁’𝘀 𝗯𝗲𝘀𝘁 𝘁𝗼 𝗮𝘃𝗼𝗶𝗱 𝗰𝗵𝗮𝗻𝗴𝗲𝘀 𝗮𝘁 𝗮 𝗹𝗮𝘁𝗲𝗿 𝘀𝘁𝗮𝗴𝗲 [...]. 𝗟𝗮𝘁𝗲-𝘀𝘁𝗮𝗴𝗲 𝗰𝗼𝗺𝗽𝗮𝗿𝗮𝗯𝗶𝗹𝗶𝘁𝘆 𝗰𝗮𝗺𝗽𝗮𝗶𝗴𝗻𝘀 𝘀𝗮𝗽 𝗿𝗲𝘀𝗼𝘂𝗿𝗰𝗲𝘀 𝗮𝗻𝗱 𝗺𝗮𝘆 𝗱𝗶𝘀𝗿𝘂𝗽𝘁 𝗽𝗿𝗼𝗱𝘂𝗰𝘁 𝘁𝗶𝗺𝗲𝗹𝗶𝗻𝗲𝘀, so avoid making technology and process changes unless it’s absolutely necessary." That's not 𝘶𝘴 saying it, it's the heads of CMC departments at large pharma/biotechs who have an approved commercial product. 🤭 Start with the end in mind (𝙌𝙪𝙖𝙡𝙞𝙩𝙮 𝙗𝙮 𝘿𝙚𝙨𝙞𝙜𝙣) > think 𝗦𝗰𝗮𝗹𝗮𝗯𝗶𝗹𝗶𝘁𝘆, think 𝗣𝗵𝗲𝗻𝗼𝘁𝘆𝗽𝗶𝗰 𝗽𝗿𝗼𝗳𝗶𝗹𝗲, think 𝗘𝗳𝗳𝗶𝗰𝗮𝗰𝘆, think 𝗥𝗲𝗹𝗲𝗮𝘀𝗲... And that's why we're here (𝘶𝘴 this time, Cell&Gene Consulting Inc.): to help you navigate that long and winding road.

🌟 𝗧𝘂𝗺𝗼𝗿 𝗜𝗺𝗺𝘂𝗻𝗲 𝗔𝗿𝗰𝗵𝗲𝘁𝘆𝗽𝗲𝘀 𝗮𝗻𝗱 𝗖𝘆𝘁𝗼𝗸𝗶𝗻𝗲𝘀 𝗠𝗔𝗧𝗧𝗘𝗥 𝗶𝗻 𝗖𝗮𝗻𝗰𝗲𝗿 𝗜𝗺𝗺𝘂𝗻𝗼𝘁𝗵𝗲𝗿𝗮𝗽𝘆 🌟 Although the key findings below won't surprise anyone (CD8+, NK cells and IFNy are associated with better patient outcomes - whoop-de-do! 🤓), this study is a great piece of science landing at the centre of the debate of 𝘄𝗵𝗮𝘁 𝗮 𝗴𝗼𝗼𝗱 𝗶𝗺𝗺𝘂𝗻𝗼𝘁𝗵𝗲𝗿𝗮𝗽𝘆 𝗽𝗿𝗼𝗱𝘂𝗰𝘁 𝗻𝗲𝗲𝗱𝘀 𝘁𝗼 𝗹𝗼𝗼𝗸 𝗹𝗶𝗸𝗲 to be efficient in solid tumours as they outline more detailed immune archetypes than the ones the industry usually relies on. 📈 Moreover, they used 𝟯𝟲𝟰 𝗯𝗶𝗼𝗽𝘀𝗶𝗲𝘀 across 𝟭𝟮 𝘀𝗼𝗹𝗶𝗱 𝘁𝘂𝗺𝗼𝘂𝗿 𝘁𝘆𝗽𝗲𝘀 to run this study, adding 𝗰𝗿𝗶𝘁𝗶𝗰𝗮𝗹 𝘀𝘁𝗮𝘁𝗶𝘀𝘁𝗶𝗰𝗮𝗹 𝗽𝗼𝘄𝗲𝗿 to a life-long discussion over what phenotypic profile is better. 🔋 🔬 𝗜𝗺𝗺𝘂𝗻𝗲-𝗥𝗶𝗰𝗵 𝗔𝗿𝗰𝗵𝗲𝘁𝘆𝗽𝗲𝘀 - Tumors with high levels of CD8+ T cells, natural killer (NK) cells, and cytokines like IFN-γ are associated with better patient outcomes and enhanced responses to immunotherapies. 🧬 𝗖𝘆𝘁𝗼𝗸𝗶𝗻𝗲 𝗦𝗶𝗴𝗻𝗮𝘁𝘂𝗿𝗲𝘀 - The study highlights the importance of specific cytokines: > IFN-γ and TNF-α drive strong anti-tumor responses. > IL-6 and IL-10 may contribute to immune suppression and poor therapy outcomes. 🌈 𝗖𝗵𝗲𝗺𝗼𝗸𝗶𝗻𝗲 𝗘𝘅𝗽𝗿𝗲𝘀𝘀𝗶𝗼𝗻: Elevated chemokines such as CXCL9 and CXCL10 are linked to increased immune cell infiltration, further enhancing the effectiveness of treatments. https://lnkd.in/g4X338rW

𝗨𝗻𝗹𝗼𝗰𝗸𝗶𝗻𝗴 𝘁𝗵𝗲 𝗣𝗼𝘁𝗲𝗻𝘁𝗶𝗮𝗹 𝗼𝗳 𝗶𝗡𝗞𝗧 𝗖𝗲𝗹𝗹𝘀 𝗶𝗻 𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 🚀🧬 Recent research highlights the power of CD19-CAR-iNKT cells in cancer therapy. These engineered cells not only target leukemia directly but also activate NK cells, boosting anti-tumor activity. 🔥 Plus, they help prevent graft-versus-host disease (GVHD) in transplants, making them a game-changer for allogeneic cell therapies. 🛡️ 𝗪𝗵𝘆 𝗶𝗡𝗞𝗧 𝗖𝗲𝗹𝗹𝘀 𝗠𝗮𝘁𝘁𝗲𝗿:  💥 𝗗𝘂𝗮𝗹 𝗔𝗰𝘁𝗶𝗼𝗻: Combines tumor-killing with immune regulation.  🛠️ 𝗡𝗞 𝗖𝗲𝗹𝗹 𝗔𝗰𝘁𝗶𝘃𝗮𝘁𝗶𝗼𝗻: Amplifies the overall therapeutic effect.  🔄 "𝗢𝗳𝗳-𝘁𝗵𝗲-𝗦𝗵𝗲𝗹𝗳" 𝗣𝗼𝘁𝗲𝗻𝘁𝗶𝗮𝗹: Lower risk of GVHD opens the door for universal donor cells. 𝗖𝗵𝗮𝗹𝗹𝗲𝗻𝗴𝗲𝘀: Complex manufacturing and potential cell exhaustion could limit scalability and long-term effectiveness. 🧪 But with ongoing innovation, iNKT cells could soon transform cancer treatment and transplant outcomes. At Cell&Gene Consulting Inc., we developed an expertise, as well as a tight network, that can support #CellTherapy companies developing these very promising iNKT products. #iNKT #CARtherapy #Biotech #Innovation #immunotherapy #advancedtherapies https://lnkd.in/gxENNbAq

🚀 Exciting Breakthrough in CAR T Cell Therapy! A recent study published in Science Immunology 𝗵𝗮𝘀 𝗶𝗱𝗲𝗻𝘁𝗶𝗳𝗶𝗲𝗱 𝗖𝗗𝟱 𝗮𝘀 𝗮 𝗻𝗲𝘄 𝘁𝗮𝗿𝗴𝗲𝘁 𝘁𝗼 𝘀𝘂𝗽𝗲𝗿𝗰𝗵𝗮𝗿𝗴𝗲 𝗖𝗔𝗥 𝗧 𝗰𝗲𝗹𝗹 𝘁𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀. Researchers at Penn Medicine used CRISPR-Cas9 to knock out 𝗖𝗗𝟱, 𝗮 𝗽𝗿𝗼𝘁𝗲𝗶𝗻 𝗮𝗰𝘁𝗶𝗻𝗴 𝗮𝘀 𝗮𝗻 𝗶𝗺𝗺𝘂𝗻𝗲 𝗰𝗵𝗲𝗰𝗸𝗽𝗼𝗶𝗻𝘁 𝘁𝗵𝗮𝘁 𝗵𝗶𝗻𝗱𝗲𝗿𝘀 𝗧 𝗰𝗲𝗹𝗹 𝗲𝗳𝗳𝗲𝗰𝘁𝗶𝘃𝗲𝗻𝗲𝘀𝘀. The approach of knocking out the CD5 protein in CAR T cell therapy shows immense promise but also comes with several challenges that need addressing as it moves towards clinical application. 🌟 𝗣𝗿𝗼𝗺𝗶𝘀𝗲𝘀: • 𝗘𝗻𝗵𝗮𝗻𝗰𝗲𝗱 𝗘𝗳𝗳𝗶𝗰𝗮𝗰𝘆 𝗶𝗻 𝗦𝗼𝗹𝗶𝗱 𝗮𝗻𝗱 𝗟𝗶𝗾𝘂𝗶𝗱 𝗧𝘂𝗺𝗼𝗿𝘀: By removing CD5, CAR T cells can proliferate and survive longer, showing more robust tumor-killing activity. This opens up the possibility of using CD5 knockout CAR T cells against more challenging cancers like pancreatic and prostate cancers. • 𝗜𝗺𝗽𝗿𝗼𝘃𝗲𝗱 𝗠𝗮𝗻𝘂𝗳𝗮𝗰𝘁𝘂𝗿𝗶𝗻𝗴: From a GMP perspective, the CD5 knockout improves the production of CAR T cells by preventing self-targeting which results in more efficient and scalable cell manufacturing processes. 🛑 𝗖𝗵𝗮𝗹𝗹𝗲𝗻𝗴𝗲𝘀: • 𝗦𝗮𝗳𝗲𝘁𝘆 𝗖𝗼𝗻𝗰𝗲𝗿𝗻𝘀: The long-term effects of knocking out CD5 in CAR T cells are not fully understood. While CD5 deletion boosts anticancer activity, its broader impact on immune regulation could lead to unintended side effects, such as autoimmunity or off-target effects. • 𝗖𝗼𝗺𝗽𝗹𝗲𝘅𝗶𝘁𝘆 𝗶𝗻 𝗦𝗼𝗹𝗶𝗱 𝗧𝘂𝗺𝗼𝗿𝘀: While CD5 knockout shows promise in preclinical models of solid tumors, the tumor microenvironment in these cancers remains a significant hurdle. Solid tumors often have physical barriers and immunosuppressive factors that make it hard for CAR T cells to penetrate and function effectively. • 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗮𝗻𝗱 𝗖𝗹𝗶𝗻𝗶𝗰𝗮𝗹 𝗛𝘂𝗿𝗱𝗹𝗲𝘀: As with any new gene-editing strategy, rigorous testing in clinical trials is required to ensure safety, efficacy, and scalability. Moving from preclinical success to human trials brings logistical and regulatory challenges, especially given the need for CRISPR-based gene editing. • 𝗖𝗼𝘀𝘁 𝗮𝗻𝗱 𝗔𝗰𝗰𝗲𝘀𝘀𝗶𝗯𝗶𝗹𝗶𝘁𝘆: CAR T therapies are already expensive, and incorporating CRISPR technology for CD5 knockout could increase costs, potentially limiting patient access. Ensuring this treatment remains accessible while balancing development costs is a significant challenge for the biotech community. At Cell&Gene Consulting Inc., we make it our mission to overcome safety, regulatory, and manufacturing challenges to transition great scientific discoveries into viable clinical assets.💡🔬 #Immunotherapy #CellTherapy #CAR #CRISPR #Biotech #GMP Vittoria Biotherapeutics, Inc. Aditya Nimmagadda https://lnkd.in/eD4D7NmK

🚀 𝗔𝗱𝘃𝗮𝗻𝗰𝗶𝗻𝗴 𝗖𝗔𝗥-𝗧 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗳𝗼𝗿 𝗔𝘂𝘁𝗼𝗶𝗺𝗺𝘂𝗻𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀: 𝗔 𝗕𝗿𝗲𝗮𝗸𝘁𝗵𝗿𝗼𝘂𝗴𝗵 𝗔𝗽𝗽𝗿𝗼𝗮𝗰𝗵 Exciting new data from Wang et al. demonstrates the transformative potential of TyU19, a 𝘩𝘦𝘢𝘭𝘵𝘩𝘺 𝘥𝘰𝘯𝘰𝘳-𝘥𝘦𝘳𝘪𝘷𝘦𝘥, CRISPR-engineered CD19-targeting CAR-T therapy. This allogeneic "off-the-shelf" treatment achieved safe and deep remission in patients with severe autoimmune diseases, including immune-mediated necrotizing myopathy and diffuse cutaneous systemic sclerosis. 🌟 𝗞𝗲𝘆 𝗛𝗶𝗴𝗵𝗹𝗶𝗴𝗵𝘁𝘀 𝗼𝗳 𝘁𝗵𝗲 𝘀𝘁𝘂𝗱𝘆: > Rapid B cell depletion and alleviation of organ damage in highly refractory autoimmune cases. > No cytokine release syndrome (CRS) or graft-versus-host disease (GVHD) observed. > The treatment shows promise in reversing fibrosis and resetting the immune system—offering a scalable, cost-effective alternative to personalized autologous therapies. 🤞 𝗣𝗿𝗼𝗺𝗶𝘀𝗲𝘀 𝗼𝗳 𝗔𝗹𝗹𝗼𝗴𝗲𝗻𝗲𝗶𝗰 𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀 𝗳𝗼𝗿 𝗔𝘂𝘁𝗼𝗶𝗺𝗺𝘂𝗻𝗲 𝗗𝗶𝘀𝗲𝗮𝘀𝗲𝘀: > Rapid intervention: In fast-progressing autoimmune diseases, where time is of the essence, having an "off-the-shelf" solution can enable much quicker intervention and improve patient outcomes. Moreover, 𝘀𝘁𝗮𝗿𝘁𝗶𝗻𝗴 𝗮 𝗽𝗿𝗼𝗱𝘂𝗰𝘁𝗶𝗼𝗻 𝘄𝗶𝘁𝗵 𝗵𝗲𝗮𝗹𝘁𝗵𝘆 𝗱𝗼𝗻𝗼𝗿𝘀' 𝗰𝗲𝗹𝗹𝘀 𝗶𝗻𝘀𝘁𝗲𝗮𝗱 𝗼𝗳 𝗮𝗻 𝗮𝗹𝗿𝗲𝗮𝗱𝘆 𝗶𝗺𝗽𝗮𝗶𝗿𝗲𝗱 𝗶𝗺𝗺𝘂𝗻𝗲 𝘀𝘆𝘀𝘁𝗲𝗺 is a promising strategy to further improve the outcome.  > Reduced treatment burden: for patients with chronic autoimmune diseases, the ability to avoid ongoing immune suppression through a single or few infusions of CAR-T cells could significantly improve quality of life and reduce long-term side effects. > Targeted immune modulation: by selectively depleting pathogenic B cells, these therapies offer the promise of not just treating symptoms but inducing long-term remission or even reversing disease progression, particularly in conditions like systemic sclerosis and severe myopathies. 🔍 𝗖𝗵𝗮𝗹𝗹𝗲𝗻𝗴𝗲𝘀 𝗼𝗳 𝗔𝗹𝗹𝗼𝗴𝗲𝗻𝗲𝗶𝗰 𝗖𝗔𝗥-𝗧 𝗧𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀: > Immune rejection: overcoming the risk of rejection by the patient's immune system remains a key obstacle. The use of CRISPR technology to remove immune-activating HLA molecules helps, but long-term persistence is still a challenge. > Graft rejection: while engineered to avoid GVHD, the allogeneic nature of these therapies may still trigger immune responses over time, leading to potential rejection of the donor cells. > Durability of response: ensuring that these "off-the-shelf" CAR-T cells remain effective over extended periods without the need for repeated infusions is a critical area of ongoing research. This study opens new horizons for affordable and accessible CAR-T cell therapies, poised to revolutionize treatment for autoimmune disorders. #biotech #celltherapy #CAR-T #autoimmunedisease #CRISPR #innovation https://lnkd.in/g4e5YgCX

🔬 𝗜𝗻 𝗩𝗶𝘃𝗼 𝗖𝗔𝗥 𝗧-𝗖𝗲𝗹𝗹 𝗧𝗵𝗲𝗿𝗮𝗽𝘆: 𝗚𝗠𝗣 𝗢𝗽𝗽𝗼𝗿𝘁𝘂𝗻𝗶𝘁𝗶𝗲𝘀 & 𝗖𝗵𝗮𝗹𝗹𝗲𝗻𝗴𝗲𝘀 🔬 In vivo CAR T-cell therapy is emerging as a transformative approach, offering reduced manufacturing times and cost-efficiency by directly modifying T cells in patients. However, there are several critical challenges to overcome for GMP compliance and successful clinical translation. Cell&Gene Consulting Inc. can support navigating these challenges, based on the Tech Ops and CMC expertise we have developed: 𝟭- 𝗩𝗶𝗿𝗮𝗹 𝗩𝗲𝗰𝘁𝗼𝗿 𝗦𝗮𝗳𝗲𝘁𝘆 & 𝗤𝘂𝗮𝗹𝗶𝘁𝘆 𝗖𝗼𝗻𝘁𝗿𝗼𝗹: Viral vectors, like LVV and AAV, play a crucial role in gene delivery but carry risks such as insertional mutagenesis, contamination, and immunogenicity. Rigorous testing is required to ensure they meet safety and purity standards. GMP-friendly viral production must guarantee batch-to-batch consistency, vector integrity, and sterility. 𝟮- 𝗚𝗲𝗻𝗲 𝗘𝗱𝗶𝘁𝗶𝗻𝗴 𝗥𝗶𝘀𝗸𝘀: The use of advanced gene-editing tools like CRISPR introduces risks of off-target effects, which could disrupt healthy gene functions or cause unwanted immune responses. Developing high-fidelity editing systems and comprehensive monitoring for off-target events is essential to ensure therapeutic safety and efficacy. 𝟯- 𝗧𝗿𝗮𝗻𝘀𝗳𝗲𝗰𝘁𝗶𝗼𝗻 𝗘𝗳𝗳𝗶𝗰𝗶𝗲𝗻𝗰𝘆 & 𝗧𝗮𝗿𝗴𝗲𝘁𝗶𝗻𝗴: Achieving high transfection efficiency in vivo without affecting non-target cells is a major technical hurdle. Nanoparticles and viral vectors need precise engineering to enhance T cell targeting while minimizing off-target effects, toxicity, and immunogenicity. 𝟰- 𝗖𝗠𝗖 𝗖𝗼𝗻𝘀𝗶𝗱𝗲𝗿𝗮𝘁𝗶𝗼𝗻𝘀: Ensuring the stability, scalability, and quality control of gene delivery systems under GMP guidelines is key. Manufacturing processes need to be optimized for reproducibility, and quality assurance protocols must be robust to handle large-scale production while maintaining regulatory compliance. 𝟱- 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗖𝗼𝗺𝗽𝗹𝗶𝗮𝗻𝗰𝗲: The transition from preclinical research to clinical applications involves navigating complex regulatory pathways (e.g., FDA, EMA) for product approval. This includes toxicology studies, safety testing, long-term follow-ups, and meeting stringent criteria for purity, potency, and identity of the final product. As in vivo CAR T-cell therapies continue to evolve, addressing these GMP challenges will be critical to bringing this innovative treatment to broader patient populations. #GMP #CAR #CellTherapy #Biomanufacturing #CMC #RegulatoryAffairs #Biotech #Immunotherapy https://lnkd.in/gzraZYmD

🚨 Excellent discussion on the Tech Transfer of Assays between AD and QC. 🚨 Sadly, the forgotten child of GMP #techtransfer remains the transfer of #analytical assays, as sponsors see these assays like less of a hurdle than the big scary GMP manufacturing 👻 . This is a mistake. Assays are critical as they define the product throughout and at the end of the manufacturing process. As such, analytical assays need to be at the centre of your Tech Transfer strategy - from Analytical Development to #QC. I will not repeat what’s been said very accurately by the panelists here. One concept I want to insist on is **INTERNAL PROCESS CONTROL (IPC)**. During your preclinical studies, it is important to collect IPCs at all critical steps of your process (leukapheresis, intermediate product, drug substance, drug product etc.) and characterize them to the best of your abilities using your analytical tests. Do it using a well characterized donor for whom you already have a lot of data on. 📊 That way, you increase your understanding of both the assay and the biological material. These IPCs will allow you to transfer any relevant analytical assay in a reliable and time effective manner ⏱ : you’ll already know what data range you are aiming at (given your accumulated dataset on *that* donor at *that* step with *that* assay) so the #comparability studies necessary for the tech transfer will become much less painful. Think ahead, build you IPC and characterize it - you will save time, money, energy (and frustration) during your AD to QC Tech Transfer. 👇👇👇 Thanks to the panelists for a truly great and needed discussion: Hadar Adams Ilya Shestopalov, PhD Kitman Yeung Takele Teklemariam BioInsights bluebird bio Atara Biotherapeutics Miltenyi Biotec https://lnkd.in/g6TGhNiM

📊 Today, we talk Lot-to-Lot Variability and why it matters in #advancedtherapies. The ultimate goal of an early stage biotech is to transform promising science 🔬 into a quality-controlled, safe and (hopefully) efficacious product for patients. 💊 But in an industry in which the starting material is likely coming from a human being (patient for #autologous or healthy donor for #allogeneic), Variability becomes a factor that matters. This is one of the reasons why "Start with the end in mind" is so important. As Donna Rill states in this Cell & Gene interview, Quality by Design (QbD) are principles that will help you control variability in your process and product. Knowing what your product should look like (Quality Target Product Profile/QTPP) will help you design it in a reproducible and reliable manner. "In the migration from, for example, academic R&D to translational and early/late-stage GMP manufacturing, it is important to prioritize an understanding of the [#QTPP], such as final product expectations, proposed mechanism of action, etc. [...] Start with expectations of product CQAs that can be refined as your dataset increases. #QbD has a huge impact on #celltherapy and #genetherapy product manufacturing. It helps control variability not only in the manufacturing process but also within reagents and consumables as you progress from early-stage translational development and scale to GMP manufacturing." Cell&Gene Consulting Inc. can help you on the path from "Amazing Science" to "(hopefully) Amazing Product" 🚀 by designing a controlled #CMC strategy relying on data and outcomes. Right from the start, so you don't waste efforts down the line. 💸 https://lnkd.in/gwY9QN7n Triumvira Immunologics, Inc. Deloitte Consulting