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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

For those who missed it, Jo Shorthouse, editor, InVivo, a Citeline Commercial publication, discusses a range of #CGT topics with Miguel Forte, CEO, Kiji Therapeutics and ISCT, International Society for Cell & Gene Therapy President. This is a vital read as they chew over some critical topics for the #cellandgenetherapy space, from the perspective of Miguel’s efforts for Kiji to take flight as a developer of next generation #gene engineered induced pluripotent stem cell-derived mesenchymal stem cells (#iPSC-#MSCs). These topics include the potential of the next generation of #CGTs to tackle #patient access for new indications, how the current #regulatory framework is supporting this, and navigating #investors to fund this new vital #innovations. “Commercialization is a vision that needs to be present on everything you do from day one. We need to make a product that is easy to use and cost effective. All those elements, all those parameters, are going to be key to a successful commercialization. Even before the first clinical trial, in everything that I do, I am already thinking about how I can improve my chances of success at the final stop, which is commercialization.” https://lnkd.in/dmKPvDjq

Cell and gene therapies are at the very center of healthcare innovation and are among the fastest-growing therapeutics. These therapies treat, prevent, and cure genetic and acquired diseases, with transformational health outcomes. Cold Chain Technologies and Cellbox Solutions, Germany, are launching a strategic collaboration to expand the use of innovative transport solutions for difficult-to-freeze cells, enabling clinical developers to provide therapies to patients under the best physiological conditions. Applications include transport of primary cells, organoids, NK cells, stem cells, neurons, for use in cell & gene therapy, tissue engineering, 3D bioprinting, organ-on-a-chip, targeting oncology, cardiology, diabetes, and other therapeutic areas. The Cellbox portable CO2 incubator provides a controlled environment for the transport of living cells and biological structures, with temperature and CO2 levels adjusted to ensure optimal incubation conditions. This, combined with CCT Smart Solutions digital platform, offers the capability for clinical developers to plan, track, and optimize their biomedical shipments using state-of-the-art technology. Cellbox adds to the broad portfolio of thermal assurance solutions that CCT offers, addressing the growing need of improved cell quality for the development and use of life-saving therapies. #CCTCell&Gene

🔷 Kiji Takes Flight With Off-The-Shelf Stem Cells • Who should read this?  Commercial, competitive intelligence and R&D executives, and investors of companies in the cell therapy space. • What is it? An interview with Miguel Forte, president and chairman of the board at the International Society of Cell and Gene Therapy, about the new company he is leading, Kiji Therapeutics. Kiji is developing IPSC-MSC-engineered cell therapy to treat graft-versus-host disease and inflammatory diseases. • Why does it matter? There has been growth in the number and maturity of companies in the cell therapy space, but manufacturing efficiency and cost challenges continue to hold the field back. Companies like Kiji could disrupt the space with engineered cells that have the potential to improve efficacy and manufacturing quality, consistency, and flexibility. Citeline Citeline Commercial #pharmainsights #stemcellresearch

RNA interference (#RNAi) and CRISPR–Cas9 gene editing technology are attractive approaches for treating #glioblastoma. Small interfering RNA (#siRNA) is a noncoding #RNA molecule that is used to regulate gene expression. It is double stranded RNA that silences genes by binding to a target transcript to ultimately cleave mRNA. Delivering these #biologics to the tissue associated with glioblastoma is difficult. This study demonstrates how a #liposome can be transported across the blood–brain barrier (#BBB) and deliver siRNA and CRISPR–Cas9 into the cytoplasm of these target tissue cells. Of exceptional note, liposomes are effective only at tumor sites and not in normal tissues, which improves the safety of combined RNAi and CRISPR–Cas9 therapeutics. https://lnkd.in/eFjQkmWC #CRISPR #LNP #mRNA #Cas9 #DDS #nanoparticles #GBM #research #innovation #engineering #bloodbrainbarrier #Plofsome #polymers #polymernanoparticles #PNP #cancer #medicine #precision #quality

🚀 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

✍️ Biotechnology and genetic engineering are rapidly advancing fields that hold tremendous promise for revolutionizing healthcare and addressing pressing medical challenges. These fields encompass a wide range of technologies and techniques that manipulate biological systems at the molecular level to develop new treatments, therapies, and diagnostic tools. 🧬 One of the most significant contributions of biotechnology and genetic engineering to healthcare is the development of precision medicine. By analyzing individuals' genetic profiles and biomarkers, researchers can tailor medical treatments and therapies to specific genetic predispositions, maximizing effectiveness and minimizing adverse reactions. 🏥 Moreover, biotechnology plays a crucial role in the development of novel therapeutic modalities, such as gene therapy, cell therapy, and immunotherapy. These cutting-edge treatments leverage genetic engineering techniques to target and modulate disease pathways, offering potential cures for previously untreatable conditions like cancer, genetic disorders, and autoimmune diseases. 🦠 Furthermore, biotechnology and genetic engineering have revolutionized diagnostic technologies, enabling the development of faster, more accurate, and less invasive diagnostic tests. Techniques like next-generation sequencing, polymerase chain reaction (PCR), and CRISPR-based diagnostics allow for the early detection of diseases, personalized treatment plans, and monitoring of treatment responses.

📢 Attention cell therapy researchers and professionals! We understand that the journey of working with induced pluripotent stem cells (iPSCs) for clinical applications is fraught with challenges. From maintaining robust cell cultures to ensuring reliable performance under stressful conditions, the path is not easy. Don't worry, the Gibco™ CTS™ StemFlex™ Medium, launched on Friday, Nov 15th, is designed to address these challenges. Gibco CTS StemFlex Medium supports the robust expansion of feeder-free pluripotent stem cells (PSCs) and is optimized for the most demanding clinical production applications, including gene editing, clonal expansion, and single-cell passaging. Key benefits of Gibco CTS StemFlex Medium include: -Robust PSC adherent cell culture for cell therapy -Reliable performance in stressful applications -High clonal expansion efficiency -Consistent expansion of high-quality PSCs -Smooth transition from StemFlex Medium (RUO) to CTS StemFlex Medium for clinical production Elevate your cell therapy research and clinical production with the Gibco CTS StemFlex Medium. Visit our product page to learn more about how this innovative medium can make a difference in your work. Learn more: https://lnkd.in/eSdb8aTk

Revolutionary Potential of iPSC Manufacturing for Off-the-Shelf Cell Therapies: "It feels like where we were 25 years ago with monoclonal antibodies....." Today's RoslinCT's webinar brought together leaders in induced pluripotent stem cell (iPSC) manufacturing to discuss the challenges and opportunities in scaling this technology for allogeneic, off-the-shelf cell therapies. The experts agreed that overcoming manufacturing hurdles for iPSCs will usher in a new era of effective, accessible cell-based treatments. Key points included: 🔹Shift from autologous to allogeneic manufacturing is critical for patient access and scalability (eventually). 🔹Optimizing reprogramming methods like Sendai virus and synthetic mRNA is an important first step. 🔹Gene editing tools like CRISPR allow engineering of hypoimmunogenic iPSC lines by knocking out genes like B2M and CIITA. But we should understand the consequences of such engineering. 🔹Analytical challenges remain in ensuring genetic stability, identity testing, and removing contamination. 🔹Scaling and bioprocessing expertise will be crucial, with bioreactor scales potentially reaching industrial 500L+ batches. 🔹Stakeholder collaboration and global harmonization of standards are needed in this nascent field. The webinar reinforced iPSCs as a powerful platform for revolutionizing regenerative medicine. But translational hurdles must still be overcome through continued R&D, technology transfer, and multi-stakeholder coordination. Take-home-message: Everyone should support efforts in agreeing global standards, having open dialogue with regulators, and the development of bespoke analytics that can be used by many developers. Thanks RoslinCT, Cell & Gene Therapy Insights and all the panelists. https://lnkd.in/eJZhhN2v

📢 Attention cell therapy researchers and professionals! We understand that the journey of working with induced pluripotent stem cells (iPSCs) for clinical applications is fraught with challenges. From maintaining robust cell cultures to ensuring reliable performance under stressful conditions, the path is not easy. Don't worry, the Gibco™ CTS™ StemFlex™ Medium, launched on Friday, Nov 15th, is designed to address these challenges. Gibco CTS StemFlex Medium supports the robust expansion of feeder-free pluripotent stem cells (PSCs) and is optimized for the most demanding clinical production applications, including gene editing, clonal expansion, and single-cell passaging. Key benefits of Gibco CTS StemFlex Medium include: -Robust PSC adherent cell culture for cell therapy -Reliable performance in stressful applications -High clonal expansion efficiency -Consistent expansion of high-quality PSCs -Smooth transition from StemFlex Medium (RUO) to CTS StemFlex Medium for clinical production Elevate your cell therapy research and clinical production with the Gibco CTS StemFlex Medium. Visit our product page to learn more about how this innovative medium can make a difference in your work. Learn more: https://lnkd.in/gNyshWqK