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The challenges of how CGT can help with cardiac indications are well said. You need to deliver many different cell types to specific location and make them precisely form a comprehensive architecture to function correctly. Any cytokine, chemokine releasing, rejection could kill the program. My vision of how CGT can help with cardiac indication needs to combine with iPSC (from patient w/ or w/o gene correction), cell differentiation and 3D organ printing. Shouldn't be too far away. How do you think CGT can help with cardiac indications? Please share your comments.

https://lnkd.in/gnezZtAu Article title: Dissemine intravasculary coagulation may be the presenting feature for chronic myelomonocytic leukemia: Special case report   Author(s): Itir Sirinoglu Demiriz   Journal: Archives of Hematology Case Reports and Reviews   Journal ISSN: 2640-7914 Abstract: Chronic myelomonocytic leukemia (CMML) is a malignant myeloid stem cell disease accompanied by dysplasia in the context of myeloproliferative disease. Peripheral cytopenias (mainly anemia and thrombocytopenia) and hepatosplenomegaly are common findings. Dramatic leukocytosis can also be seen without transformation to acute myeloid leukemia (AML); in some cases, this leukocytosis is associated with leukostasis and end organ damage. Splenomegaly is present in up to 25 percent of patients with CMML and is often accompanied by hepatomegaly, lymphadenopathy, or nodular cutaneous leukemic infiltrates. #Anemia #AcuteMyeloidLeukemia #LymphAngiogenesis #HematologicalMalignancies #BloodTransfusion #SerumProteinElectrophoresis #HematopoieticStemCellTransplantation #PhotoAcoustics #LymphNodeBiopsy #BCellLymphoma #InvivoFlowCytometry #ChainsofHemoglobin #Lymphoma #HodgkinsLymphoma #Coagulation #Thalassemia #NonHodgkinsDisease #MultipleMyeloma #StemCellsAndHematopoiesis #LeukocytesAndPlatelets #RedCellBiology #HematologicNeoplasia #Immunobiology #BloodAndMarrowTransplantation #TransfusionMedicine #VascularBiology #Hemobiology #Peertechz #PeertechzPublications #OpenAccess #ScientificJournals #PeerReviewedJournals #OpenAccessPublishers

🧬 🔬 CGT has long been the poster child for personalized treatment in oncology and rare diseases, but progress into the use of these modalities has been overlooked in non-oncological conditions, until recently.     A recent article in Nature Reviews Drug Discovery indicates a “growing interest in cell therapies in non-oncology indications such as autoimmune, cardiac and infectious diseases, with gene-modified cell therapy trials in autoimmune disorders increasing by almost fourfold between 2021 and 2023.” (https://lnkd.in/eYzz949a). ❤️ In this article with Drug Target Review, Ibon Garitaonandia, PhD, MBA of CellProthera SAS explores this shift in the context of cardiology, carefully considering the challenges for therapeutic development in this area, as well as the opportunities. Read more, here: https://lnkd.in/e4mt4mhf #Cardiology #CellTherapy #GeneTherapy #AdvancedTherapies #TherapeuticDevelopment

Genenta Science Temferon at #ASGCT American Society of Gene & Cell Therapy    Genenta Science's #Temferon has anti-tumor activity and induces #interferon response among #glioblastoma multiforme patients (#GBM), according to new clinical trial results.    #BernhardGentner, associate professor of immuno-oncology at Lausanne University Hospital, presented new data from a Phase I/IIa Genenta Science-sponsored study of the autologous hematopoietic stem cell-based therapy Temferon in glioblastoma.    In 18 treated patients, interim survival rate at two years was 28 percent, with one patient still alive more than three years post-treatment and without further therapy. Temferon was observed to cross the blood-brain barrier and successfully engraft into the tumor microenvironment, where it triggered local anti-tumor activity via release of IFNα from tumor-infiltrating macrophages. Surprisingly, along with myeloid reprogramming of the tumor microenvironment, treated patients had more CD8 effector cells with gene signatures enriched for anti-tumor neoantigen-reactive T cells, suggesting that GBM is not the "immune wasteland" it has been made out to be.    Furthermore, the investigators noted reduced proliferation in response to treatment, with an increased hypoxia response that likely correlates with decreased angiogenesis observed in histopathological analysis. Gene signatures of apoptosis also stood out, which Gentner said may correlate with areas of necrosis observed via histopathology. Tumors from Temferon-treated patients also saw increased M1-like and decreased M2-like macrophages, which largely reproduced findings from a mouse model of the disease.    The investigators are currently planning a Phase II study and evaluating more possible indications for Temferon treatment. Source: Cancer Cell Therapies at ASGCT24 | Precision Medicine Online

Often cancer becomes aggressive after the first round of therapy fails. However, glioblastoma (GBM) is one of the most innately aggressive high grade brain tumour with overall survival of an year. Extensive pleomorphism or presence of genetically diverse tumour cells within the tumour remained a major bottleneck in GBM drug discovery since last two decades-evident by the fact that are hardly any new drugs unlike other cancers. To mimic genetic diversity of GBM in drug discovery pipeline, clinically relevant GBM cell models are indeed needed. In the early 2013s, we established (as a part of my PhD thesis) one of world's first functionally characterized mesenchymal and proneural cell models derived from human patient tumours. Rigorous in vitro and animal testing of these cell models suggested that we had developed something that did not exist at that time (and perhaps even today) as a useful drug screening tool for GBM. The paper below gives a detailed overview of the research. If your are into GBM drug discovery, feel free to write me to know more on this. https://lnkd.in/gP4hdKp8 Michael Ciesielski #glioblastoma #drugdiscovery #oncology #biotech #vc #invitromodels #3Dmodels

#BrainCancer | #Immunotherapy | #Biomarkers | #Prognosis | multi-#Omics of #RegulatedCellDeath in the #Glioblastoma #TumorMicroEnvironment 🧠 | Pioneering OPEN ACCESS Study Now Breaking Online 👍 | #glioma | #TME | Glioblastoma is a highly aggressive and malignant type of brain cancer that originates from glial cells in the brain, with a median survival time of 15 months and a 5-year survival rate of less than 5%. Regulated cell death (RCD) is the autonomous and orderly cell death under genetic control, controlled by precise signaling pathways and molecularly defined effector mechanisms, modulated by pharmacological or genetic interventions, and plays a key role in maintaining homeostasis of the internal environment. The comprehensive and systemic landscape of the RCD in glioma is not fully investigated and explored. Here, after collecting 18 RCD-related signatures from the opening literature, researchers comprehensively explored the RCD landscape, integrating the multi-omics data, including large-scale bulk data, single-cell level data, glioma cell lines, and proteome level data. They also provided a machine learning framework for screening the potentially therapeutic candidates. Based on bulk and single-cell sequencing samples, they explored RCD-related phenotypes, investigated the profile of the RCD, and developed an RCD gene pair scoring system, named RCD.GP signature, showing a reliable and robust performance in predicting the prognosis of glioblastoma. Using the machine learning framework consisting of Lasso, RSF, XgBoost, Enet, CoxBoost and Boruta, they identified seven RCD genes as potential therapeutic targets in glioma and verified that the SLC43A3 highly expressed in glioma grades and glioma cell lines through qRT-PCR. This study provided comprehensive insights into the RCD roles in glioma, developed a robust RCD gene pair signature for predicting the prognosis of glioma patients, constructed a machine learning framework for screening the core candidates and identified the SLC43A3 as an oncogenic role and a prediction biomarker in glioblastoma. Celentyx Ltd Professor Nicholas Barnes PhD, FBPhS Omar Qureshi Catherine Brady GRAPHICAL ABSTRACT

In the dynamic realm of medicine, we're witnessing promising breakthroughs that are reshaping the landscape of chronic disease treatment. From cardiovascular conditions to neurological disorders, research is unveiling new avenues toward more effective and personalized therapies. 🔬 Gene Therapy for Heart Conditions: Gene therapy is proving to be a game-changer, as evidenced by a recent study led by Wang et al. (2023), showing hopeful outcomes in restoring cardiac function in patients with hypertrophic cardiomyopathy. 🩺 Immunotherapy in Cancer: In the realm of cancer, immunotherapy is making a remarkable difference, offering new hopes for patients with chronic diseases such as metastatic lung cancer. The KEYNOTE-024 trial, cited by Reck et al. (2016), revealed significant improvements in overall survival and tumor response. 🧠 Cellular Therapy in Neurological Disorders: In neurological disorders like Alzheimer's and Parkinson's, cellular therapy is demonstrating extraordinary promises. Studies such as the Fetal Cell Transplantation Study (FCTS), highlighted by Kordower et al. (2022), have shown improvements in motor and cognitive function in Parkinson's disease patients. These advancements are just a glimpse into the fascinating ongoing medical research. We invite those interested in being part of these exciting developments to consider participating in clinical trials and contribute to the advancement of medicine. Join us to stay updated on the latest news and participation opportunities in clinical trials. Together, we can make a real difference in chronic disease treatment. #ChronicDiseases #MedicalResearch #PublicHealth

Join us for exciting developments in the field of Cell and Gene Therapy (CGT) ! This track on NK, TCR, and Innate Killer Cell Therapies aims to solve critical challenges and drive advancements in the following key areas: 1️⃣ Combination Therapies for Enhanced Immunotherapeutic Responses 💡 Objective: Explore how combining cell therapies (e.g., NK, TCR) with traditional treatments can improve immune activation, overcome resistance, and achieve sustained therapeutic responses. 🔍 Focus: Optimizing combination therapies for solid tumors and blood-based cancers. 2️⃣ Cell Therapies for Solid Tumors & Blood-Based Cancers 💡 Objective: Overcome the hurdles in treating solid tumors and hematologic cancers (e.g., leukemia, lymphoma) with cell therapies. 🔍 Focus: Innovating delivery methods, improving cell persistence, and targeting tumor microenvironments for greater effectiveness. 3️⃣ Regulatory, Ethical, and Safety Considerations 💡 Objective: Tackle the regulatory, ethical, and safety challenges in developing cell therapies. 🔍 Focus: Ensuring compliance with regulations, addressing patient safety, and scaling manufacturing processes for widespread use. This track aims to break barriers, expand treatment options, and ensure the safe, effective adoption of cell therapies in clinical practice. #Cell2024 #CellTherapy #GeneTherapy

On this MPS awareness day 2024- Let us empower the need for new therapeutic options for MPS. The development of new therapeutic options for Mucopolysaccharidoses (MPS) is crucial for several reasons: 1. Current Treatments Have Limitations: While treatments like enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) have improved outcomes for some MPS patients, they are not without limitations. ERT requires lifelong administration and may not effectively address all symptoms, while HSCT carries risks and may not be suitable for all patients. 2. Addressing Unmet Needs: Despite advancements in MPS treatment, there are still unmet needs within the patient population. Many individuals continue to experience progressive disease symptoms and complications that are not adequately addressed by existing therapies. New treatment options are needed to target these unmet needs and improve the overall management of MPS. 3. Improving Quality of Life: MPS is a progressive and debilitating condition that can significantly impact patients' quality of life. Symptoms such as skeletal abnormalities, organ enlargement, and cognitive impairment can profoundly affect daily functioning and overall well-being. Developing new therapies that effectively alleviate symptoms and slow disease progression is essential for enhancing the quality of life for MPS patients and their families. 4. Increasing Treatment Accessibility: Access to current MPS treatments may be limited by factors such as cost, availability, and healthcare infrastructure. Developing new therapeutic options, particularly those that are more cost-effective, easier to administer, or applicable to a broader range of patients, can help improve treatment accessibility and reach underserved populations. 5. Advancing Precision Medicine: As our understanding of the genetic and molecular mechanisms underlying MPS continues to grow, there is a growing emphasis on precision medicine approaches tailored to individual patients' genetic profiles and disease characteristics. New therapeutic options, including gene therapy and targeted small molecule therapies, hold promise for personalized treatment strategies that address the specific needs of each MPS patient. To conclude, expanding the treatment toolkit for MPS can provide patients with more effective and personalized care, ultimately leading to better outcomes and a brighter future for those affected by these rare genetic disorders. #MPSAWARNESS #RAREDISEASEAWARENESS

Autologous CAR T cell therapy has transformed hematological malignancy treatment, showcasing the promise of cellular therapies and highlighting areas for improvement. The surge in CAR therapy trials reflects this, with efforts to explore new targets, manufacturing strategies, CAR constructs, patient populations, and effector cells. While many trials use autologous immune cells, allogeneic CAR products are increasingly being tested. The potential advantages of allogeneic therapies have spurred interest in developing techniques to overcome limitations, such as exploring various effector cell types and gene editing techniques. Immunogenicity remains a challenge, particularly for autologous CAR T cell therapies without lymphodepleting chemotherapy. For “off the shelf” cell products, post-infusion immune-modulation may be necessary for long-term persistence. Despite these challenges, experience with allogeneic HCT and solid organ transplantation suggests immunogenicity can be managed. Developing “off the shelf” therapeutic products demands significant resources in development and manufacturing, aiming for a favorable safety and efficacy profile that is accessible and affordable. However, the cost of allogeneic cell products remains uncertain, depending on the genetic modifications required, such as viral transduction and gene-editing. Additionally, the cost will be influenced by the number of cell doses prepared from one production lot, given the high expense of release testing for genetically-modified cell products. Continued investment in optimizing allogeneic strategies is justified by current data, promising that allogeneic cell products could lead to a new era in cell therapy. These advancements hold the potential to make effective therapies more widely available and affordable, overcoming current limitations and significantly improving patient outcomes. #MultipleMyeloma #CAR_TCellTherapy #MedicalResearch #CancerTreatment #InnovativeMedicine #ImmunoOncology #CancerResearch #AllogeneicCAR https://lnkd.in/dWeNhnUU