Daniela Vecchio

Delpacibart Etedesiran, or AOC 1001 developed by Avidity Biosciences, Inc., receives FDA Breakthrough Therapy Designation for for Treatment of Myotonic Dystrophy Type 1. AOC 1001 consists of a mAb that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting DMPK mRNA. This allows the drug to address the underlying cause of disease by reducing DMPK mRNA, potentially alleviating the spectrum of symptoms that people with DM1 experience. The 2024 Annual Conference of our society is just around the corner, scheduled to take place in Cambridge, MA, USA on Saturday, May 11, 2024. Don’t miss out on this exceptional opportunity to network with experts from both academia and industry, register through the following link https://lnkd.in/ecmFPA4v #antibodydiscovery #immunology #oncology #annualmeeting #annualconference #antibodies #mab #mabs #mrna #aoc  #myotonicdystrophytype1 https://lnkd.in/ggytKY-a

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Biocom California recently invited Sapient to contribute an article for its Lifelines Magazine edition focused on innovations in #genetherapy and #celltherapy. https://lnkd.in/gC9YfQnM While cell- and gene-based treatments are advancing rapidly, spurred by a landmark year of FDA approvals, their development is not without challenges. We discuss how #multiomics for gene and cell therapy can deliver the novel insights needed to realize the incredible therapeutic potential represented in these pipelines today: https://lnkd.in/gC9YfQnM

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New research from the Dana-Farber Cancer Institute has used CRISPR to discover a new potential therapeutic target for the treatment of Acute Myeloid Leukaemia (AML). The researchers used genome-wide CRISPR interference to search for genes involved in AML cell metabolism, and discovered a subset of leukaemic cells that relied on phosphoinositide-3-kinase-gamma (PI3Kγ) signalling for energy production. A PI3Kγ inhibitor already exists – eganelisib – although has not yet been used to treat leukaemia. Here, the researchers effectively shrank AML tumour xenografts with eganelisib, and also found that eganelisib worked even more effectively in combination with the standard AML treatment cytarabine. These findings point towards eganelisib as being an attractive option to improve survival in AML patients, as it is an already existing drug that can be used in combination with cytarabine. The team is now designing a clinical trial to test this. CRISPR is continuing to contribute to major discoveries like this one. At Anthony Nolan, our Immunotherapy Group are investigating potential new cell therapies with the help of technologies like CRISPR. Learn more about how you can work with us: https://lnkd.in/eUX92NSS Read more about the study from the Dana-Farber Cancer Institute: https://lnkd.in/e6CuaMYs #AML #CRISPR

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We're thrilled to announce the publication of groundbreaking research on narazaciclib and its anti-AML activity in preclinical models with specific AML mutations of clinical significance. This collaboration between Hanx Biopharmaceuticals, Crown Bioscience, and other cancer researchers has revealed promising results in targeting and inhibiting key kinases involved in AML. Dive deeper into our findings and discover how our collaborative efforts are contributing to treatments for AML. ➡️ https://bit.ly/3UrLjH7 #OncologyResearch #DrugDevelopment #AcuteMyeloidLeukemia

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Dr. Shahar Alon and his team have diretly measured, inside biopsies, how cancer cells manipulate immune cells at the molecular level. They recently published their exciting findings in the RNA journal. Their research focused on quantifying the molecular changes that occur when an immune cell comes into contact with a tumor cell. This understanding could unlock new insights into cancer progression and immune evasion mechanisms. Dr. Shahar Alon and his team execute these measurements in situ by expansion sequencing. Expansion sequencing has been a game-changer, enabling them to sequence genes with super-resolution right where they are. They systematically examined whether individual immune cells from specific cell types express genes differently when close to individual tumor cells. Their first step was to demonstrate that a dense mapping of genes in situ can be used to segment cell bodies in 3D. This improved their ability to detect cells that are likely in contact. Next, they employed three different computational approaches to detect the molecular changes triggered by proximity: differential expression analysis, tree-based machine learning classifiers, and matrix factorization analysis. The systematic analysis revealed numerous genes in specific cell types whose expression separates immune cells that are proximal to tumor cells from those that are not. Interestingly, they found a significant overlap between the different detection methods. One remarkable finding was that an order of magnitude more genes are triggered by proximity to tumor cells in CD8 T cells than in CD4 T cells T, which aligns with the ability of CD8 T cells to directly bind Major Histocompatibility Complex (MHC) Class I on tumor cells. In conclusion, in situ sequencing of an individual biopsy can detect genes likely involved in immune-tumor cell-cell interactions. This is a significant step forward, which might improve the personalization of immunotherapy treatments. The research was led by students Michal Danino and Tal Goldberg under the guidance of Dr. Shahar Alon and Prof. Gonen Singer from the faculty of engineering. For more insights visit at-https://lnkd.in/dmA-m2CV Bar Ilan Institute for Nanotechnology and Advanced materials (BINA), Bar Ilan University Shahar Alon

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More than a dozen posters and podium talks were presented at the latest #AUA2024 meeting discussing the power of Veracyte, Inc.'s Decipher Prostate and Bladder tests, as well as our and Decipher GRID (Genomics Resource for Intelligent Discovery) transcriptomic database. Among these was a study discussing that patients with low and intermediate risk #ProstateCancer (GGG 1-3 on initial biopsy), the Decipher Score is a predictor of clinically significant upgrading (i.e, increase in GGG) on final radical prostatectomy pathology. Click the link below to read an overview of this presentation: https://lnkd.in/gRvB8GHv

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Ever wonder why some CAR-T cell therapies show better efficacy than others? This week's #RayBioRead is a groundbreaking study published in @Nature focused on the role of tonic signaling in CAR-T cell function. The research, led by Chen, J., Qiu, S., Li, W. et al, identifies positively charged patches (PCPs) on CARs as drivers behind spontaneous CAR activation. By manipulating these PCPs, researchers were able to fine-tune CAR-T cell behavior, enhancing effectiveness and reducing exhaustion. This discovery opens doors for next-gen CAR-T designs. Learn more: https://lnkd.in/gxRguPwx Featured RayBiotech Product: https://lnkd.in/e7NdF3-H

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In the Quantitative Biosciences Institute, UCSF Coronavirus Research Group (QCRG), Nevan Krogan, PhD, built a transdisciplinary, multi-lab, multi-institutional, multi-national, multi-sector open-science consortium in which over a thousand trainees and senior investigators worked together—overcoming virtually every barrier that fragments science and the science community. Read more about the challenges academic research faces when it comes to breaking down silos, and about how facilitating collaboration is the solution, in this Q&A with the UCSF SCHOOL OF MEDICINE Department of Cellular and Molecular Pharmacology faculty member. https://lnkd.in/gg4D39FB

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#Type1cDCs in #PancreaticCancer ☯ | Promotion of Aggressive Disease 👎 | Facilitation of #Immunotherapy 👍 | Krishnan Mahadevan & Co. Posit Combo Therapy of #DendriticCell #Vaccine with #ICB at latest Science Magazine 👏 Inflammation of the pancreas, known as pancreatitis, can influence the emergence and progression of pancreatic cancer. How the body’s immune system responds to pancreatitis versus pancreatic cancer remains unclear. Here* Mahadevan, Allison Dyevoich, PhD, Yang Chen, Bingrui Li, et al. report that pancreatitis leads to the recruitment of a class of immune cells called conventional dendritic cells that can help to protect tissue from self-destruction by T lymphocytes. They found that the coexistence of pancreatitis and pancreatic cancer resulted in an aggressive disease in which conventional dendritic cells influenced a suppressive T cell subset to override the action of T cells that usually destroy cancer. Blocking the suppressor T cells with immunotherapy reduced the growth of pancreatic cancer. Therefore, engineered cancer-specific conventional dendritic cells along with immunotherapy may provide a mechanism to intervene in pancreatic cancer. (Summary, courtesy of Priscilla Kelly, Science Magazine) *https://lnkd.in/eakWNJBw Celentyx Ltd #immunooncology www.celentyx.com Professor Nicholas Barnes PhD, FBPhS Omar Qureshi Catherine Brady FIGURE | Tissue injury and inflammation inform cDC1 vaccine priming of immunotherapy | Pancreatitis recruits activated cDC1s into the pancreas, resulting in T cell suppression (top left). PDAC is characterized by myeloid infiltration, lack of antigen-presenting DCs, and paucity of effector T cells (top right). Pancreatitis associated with PDAC (middle left) and antigen-specific cDC1 vaccine administration (middle right) resulted in effector and memory T cell response in combination with iCBT and eliminated PDAC (bottom) |

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🎤 uBriGene Presents Exclusive Sessions at SAPA 2024 @Philly CGT Annual Conference 🎤 🚀 We're thrilled to announce our participation at 2024 @Philly Cell and Gene Therapy Annual Conference with two exclusive sessions aimed at revolutionizing the field: 1️⃣ An Innovative Technology for High Yield and More Stable #CRISPR Guide RNA: Discover how our innovative approach of using in vitro transcription (IVT) in producing guide RNA (#gRNA) that can simplify the process while being more cost-effective and free of organic chemicals. Join us to delve into the future of precision gene editing. 2️⃣ How Could CDMO and Technology Innovations Make CGT Affordable: Dive into the intersection #CDMO of and technological advancements with uBriGene. Learn about our strategies to drive down costs without compromising quality or efficacy. Let's explore how cutting-edge technology can make #CellandGeneTherapy more accessible to all. 2024 @Philly Cell and Gene Therapy Annual Conference brings together the innovators and business leaders to foster collaborations and advance cell and gene therapy in the #GreaterPhiladelphia area. 💡 Join #uBriGene to be at the forefront of innovation and collaboration in the dynamic world of cell and gene therapy and let's make cutting-edge cell and gene technology more accessible!

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Recognizing the signs and symptoms of chronic myeloid leukemia (CML) is essential for effective management. Early detection and understanding of key indicators play a crucial role in creating appropriate treatment strategies and improving outcomes for individuals with CML. Learn more about CML from the American Cancer Society at https://lnkd.in/eZfjAhe2 #CancerResearch #CancerResearchMonth #NCRM24

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Rapid Proteomics - the pre-print: 160 protein profile in under an hour and targeted quant of Ang I and II and several proteins in under 30 minutes from sample collection. https://lnkd.in/ggHgttcu

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Dana-Farber researchers discover new target for potential leukaemia therapy Dana-Farber Cancer Institute scientists have found a new target for leukaemia treatment. They discovered that some leukaemias depend on a complex called PI3Kgamma to survive. Excitingly, an existing drug, eganelisib, can inhibit this complex. Using an existing drug means animal models have already been tested, showing promise when using a combination therapy approach. Clinical trials for this new approach could start soon, offering hope for better leukaemia treatments. https://lnkd.in/g3A4A3ZZ

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Learn about our study characterizing a panel of new AML PDX models! Our research presented at AACR 2024 highlights the establishment of new AML PDX models carrying hotspot mutations. Download the poster now to learn: ✅Methods used to establish the AML PDX models ✅Effect of study treatments on models carrying specific hotspot mutations ✅Which treatments overcame FLT3-TKD mediated resistance in a preclinical setting Download now ➡️ https://bit.ly/49VcSP1 #OncologyResearch #DrugDevelopment #AcuteMyeloidLeukemia

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Shasqi Directors, Jesse McFarland, Director of Chemistry & Bioconjugates and Tri-Hung Nguyen, Senior Director of Translational Sciences, are heading to the Radionuclide Theranostics for the Management of Cancer Gordon Research Conference (GRC) next week. They will be presenting data on how systemic toxicities associated with RLTs can be overcome using our CAPAC technology. Historically, it has been challenging to target RLTs to the tumor using monoclonal antibody-based radioconjugates, due to dose limiting toxicities based on extended systemic exposure and low tumor-to-blood ratio. Our CAPAC platform separates the chelation from the antigen-binding moiety and uses small format binders. This improves on-tumor payload activation and reduces normal tissue exposure, enabling optimal RLT clearance and reducing non-specific toxicity. If you'll be at GRC, stop by our poster to say hi. #RLTs #targetedtherapy #gordonresearchconferences

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From their abstract: G-protein-coupled receptors (GPCRs) are key regulators of human physiology and are the targets of many small-molecule research compounds and therapeutic drugs. While most of these ligands bind to their target GPCR with high affinity, selectivity is often limited at the receptor, tissue and cellular levels. Antibodies have the potential to address these limitations but their properties as GPCR ligands remain poorly characterized. Here, using protein engineering, pharmacological assays and structural studies, we develop maternally selective heavy-chain-only antibody (‘nanobody’) antagonists against the angiotensin II type I receptor and uncover the unusual molecular basis of their receptor antagonism.

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