Serena Di Cosimo’s Post

Another week of milestones for #cellandgenetherapy! What were the headlines? ✅The US FDA approves the first engineered cell therapy to treat a solid tumor 🔬New positive data announced for gene therapy in clinical trials to treat Duchenne muscular dystrophy 💉First patient dosed in phase I in vivo mRNA CAR therapy to treat advanced hepatocellular carcinoma Read the full stories below and subscribe to our Sector Vector newsletter for more weekly cell and gene therapy clinical, business, and manufacturing news. 📩 https://lnkd.in/e9H6syK6 The FDA approved Adaptimmune's cell therapy to treat metastatic synovial sarcoma, making it the first engineered cell therapy to treat a solid tumor. It is also the second cell therapy for a solid tumor approved this year. https://lnkd.in/gp68yd6U REGENXBIO Inc. reported new, positive interim safety and efficacy data in the Phase I/II trial of its gene therapy in pediatric patients with Duchenne muscular dystrophy. The study observed a consistently high expression of microdystrophin. https://lnkd.in/ezeGvvFC Myeloid Therapeutics announced the first patient in a Phase 1 study for its in vivo mRNA CAR program to treat advanced hepatocellular carcinoma. (HCC) The dosing represents a significant milestone in bringing advanced novel therapies to people with liver cancer. https://lnkd.in/endr_kFe

Proposing self-amplifying RNA encoding NDGR2 (N-myc downstream-regulated gene 2), a tumor suppressor, as a potential treatment of metastatic and drug-resistant cancers. https://lnkd.in/e58JXbV3

- Superior Natural Killers - Learn the secrets of superior stem cell-derived, Natural Killer cell therapeutics. Glycostem's Dr. Amanda van Vliet and colleagues unravelled key distinctive traits of superior allogeneic NK cells. By integrating functional anti-tumor cytotoxicity, phenotypical, and molecular bulk and single cell RNA-Seq analysis, they identified a multi-factorial gene expression signature supporting the development of more efficacious NK therapies, now published in Molecular Therapy Oncology https://lnkd.in/eyuAwm4Q

Tumor purity - a gene expression-based approach to determining “In this study, we exploited over 6000 samples from 10 breast cancer datasets to systematically quantify the impact of intrinsic and extrinsic factors on cellularity and to generate and validate a Breast Cancer Purity Score (BCPS). BCPS outperformed ESTIMATE in quantifying tumour purity and can be successfully used to adjust for tumour purity variability when extrinsic factors are prevalent. BCPS can also capture treatment-induced changes carrying predictive and prognostic information.” #tumorpurity #geneexpression #breastcancer

Single-cell chromatin accessibility landscapes across eight TCGA primary tumor types reveal cancer type-specific gene regulation. Neural network models reveal accessible chromatin signatures specific to cancer tissue and highlight the role of noncoding somatic mutations in cancer. https://buff.ly/4dQHaoB

Successfully participated in the 40th Society Of Pharmaceutical Sciences and Research INTERNATIONAL Webinar on 'Adopting Genome Editing Technology in Combinating Cancer Disease ' I say thank you Dr.Joseph Bamidele Minari(Speaker of the webinar) Associate Professor & Team lead Genome Editing Research Group , Department of Cell Biology and Genetics, University of Lagos,Akoka,Nigeria for informing "Adopting Genome Editing Technology in Combinating Cancer Disease" 👉Key Points Of these International Webinar - 1)Cancer : It is characterized by uncontrolled cell division. 2)Major categories of cancer : Carcinoma, Sarcoma, Leukemia, Lymphoma & Myeloma, Central Nervous System Cancer. 3)Characteristics of Cancerous Cells : Resisting cell death, Evading growth suppressors, Inducing angiogenesis. 4)Genome Editing : It is a type of genetic engineering which targets sequence in the genome by inserting,deleting or replacing a segment by using molecular scissors. 🤔Why we should adopt genome editing technology in combating cancer disease❓ 1️⃣It will help to reduce the mortality & morbidity associated with cancer. 2️⃣It will help to correct genetic disorders. 3️⃣It will reduce side effect associated with other treatment options. 4️⃣It will promote precision medicine.

Colorectal cancer (CRC) is a leading cause of cancer mortality, driven by genetic and environmental factors. Genomics and differential expression analysis offer crucial insights into CRC's molecular mechanisms, aiding in #personalizedmedicine approaches. Our latest use case demonstrates how g.nome® simplifies complex #omics analysis, enabling researchers and clinicians to effortlessly conduct differential gene expression studies. By leveraging publicly available #RNAseq datasets from the Sequence Read Archive (SRA), we uncover novel genetic insights, driving future CRC research and therapeutic strategies. See how we identified potential biomarkers for early detection and prognosis of CRC, spotlighting key genes like KRT23 and ETV4. Our streamlined approach reduces technical barriers, allowing you to quickly derive meaningful insights from complex data. Read the full use case: https://lnkd.in/gkd6tRKT

I would like to give my congratulations to Marije de Rooij, Dennis Remst, Dirk van der Steen, Anne Wouters, Renate Hagedoorn, Michel Kester, Miranda Meeuwsen, Tassilo Wachsmann, Arnoud de Ru, Peter van Veelen, Els Verdegaal, Frederik Frankeburg and Mirjam Heemskerk from the Department of Hematology, Center for Proteomics and Metabolics and Department of Oncology of the Leiden University Medical Center for their work published in the journal Molecular Therapy Oncology titled, “A library of cancer testis specific T cell receptors for T cell receptor gene therapy”. T cell receptor (TCR) gene therapy has a high potential to improve cancer treatment both in a personalized manner but also as an off-the-shelf product. These treatments depend on libraries of TCR that need to be generated, screened and tested for safety. Melanoma-associated antigens (MAGEs) are ideal targets for T cells, because their expression in normal tissue is restricted to testis and placenta. 🔎 In this work the researchers identified seven MAGE-specific TCRs that can be transferred into CD8+ T cells and result in efficient reactivity against different tumour-types, avoiding cross-reactivity. Primary CD8+ T cells were isolated from PBMCs by magnetic-activated cell sorting (MACS). 🔎 Previous clinical trials highlighted the potential risk of cross-reactivity, so extra effort was dedicated to safety screening of these peptides. TCRs recognizing MAGE-A family members with an unsafe expression profile were excluded from further analysis to minimize this risk even further. The identified TCRs can be included in an “off-the-shelf” TCR library that enables treatment of cancer patients with prior generated TCR constructs. Multiple TCRs could then be selected for a personalized and multi-antigen-targeting T-cell therapy. Read the full publication: 👀 If you work could benefit from magnetic cell separation, Miltenyi Biotec offers many options for every application, find more here: https://lnkd.in/d2pKTj6t 👀 Alternatively, feel free to contact us to find out more about how magnetic cell separation can contribute to your research!

Dawood et al. developed a deep-learning pipeline for predicting the drug responses of breast cancer patients using whole slide images (WSIs). The model initially mapped gene expression in cancer cell lines to drug sensitivity, then used this to predict patient drug sensitivity based on their gene expression data. They employed a graph neural network trained on WSIs, where each node represents a patch of the WSI. This network predicted drug sensitivity at both the local (patch) and global (WSI) levels. The model accurately predicted tumor sensitivity for 186 out of 427 drugs. #cancer #histology #cancerresearch https://lnkd.in/g3xSPVt7

🔬 Exciting News: CRISPR Gets FDA Nod for Treating Beta Thalassemia! What does this mean for Cancer?🧬 Last week marked a milestone with the FDA approving CRISPR gene-editing for transfusion-dependent beta thalassemia—the second major U.S. approval for this cutting-edge technology. 🌐👏 🌟 Cancer & Oncology Implications: Beyond blood disorders, CRISPR's success extends to cancer research. Recent trials demonstrate its potential in editing T cells, boosting antitumor immunity, and advancing personalized cancer treatments. A leap forward in precision medicine! 💪🌈 🚀 Revolutionizing Cancer Therapies: Precise T cell editing using CRISPR opens new doors in immunotherapy, enhancing treatment efficacy and paving the way for more targeted approaches. A game-changer in the fight against cancer. 🎯💉 🧬 Next Frontier in Precision Medicine: As we celebrate CRISPR's success, we anticipate its transformative impact on cancer treatment. From refining diagnostics to allogeneic T cell immunotherapy, CRISPR is reshaping healthcare with vast potential applications. 🌟🔍 Kudos to the researchers driving this medical revolution! Here's to a future where CRISPR continues to redefine healthcare. 💙🌐 #CRISPR #PrecisionMedicine #MedicalInnovation #CancerResearch #HealthcareRevolution #GeneEditing https://lnkd.in/gvZqSayh