John Mascola

🎤 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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Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

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Join us at the Drug Discovery Biology Strategy Meeting this November 12th in Boston to hear from Anitha Krishnan, Ph.D, Executive Director of Research at Aviceda Therapeutics. Anitha brings over a decade of innovative scientific expertise in biochemical, immunological, and molecular biology techniques. Her work is pivotal in gene therapy and cell transplantation studies, targeting ocular diseases such as Glaucoma and Age-related Macular Degeneration. She has made significant strides in understanding immune responses in neurodegeneration and autoimmune diseases, particularly through her research on microglial and macrophages and the Fas-FasL system. Anitha will be delving into the "Opportunities and Risks in the Preclinical Development of Molecules that Do Not Cross React with Rodents," offering invaluable insights from her extensive experience in advancing therapeutic approaches from discovery through to clinical trials. Don't miss the opportunity to gain from her comprehensive expertise and her knack for leading innovative preclinical research. #DrugDiscovery #BiomedicalResearch #OcularDiseases #PreclinicalDevelopment #BostonEvents #LifeSciences #StrategyMeetings

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PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L

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Affini-T’s Ankit Gupta, Senior Director, Head of Gene Editing, will present “AFNT-212: A Non-Viral Gene-Edited TCR-T Cell Therapy” to industry and academic leaders at the 5th Genome Editing Therapeutics Summit in #Boston today. Learn more about it here: https://bit.ly/3BflF2Q. #CRISPR #GeneEditing #DrugDevelopment #TCells

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🔬 New Research Highlight: Amivantamab With TKIs for Advanced NSCLC 🔬 Exciting advancements in lung cancer treatment are on the horizon! Researchers are now exploring the combination of Amivantamab with Tyrosine Kinase Inhibitors (TKIs) for advanced NSCLC patients with ALK, ROS1, or RET alterations for those progressing on current their targeted therapy. 💡 Why is this important? This combination therapy has the potential to enhance treatment outcomes for patients with specific genetic drivers of lung cancer, offering new hope for improved survival and quality of life. 📖 Dive into the details: https://lnkd.in/gEVStAau #ALKPositive #LungCancerAwareness #NSCLC

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DYK Shasqi's CAPAC technology can be used to localize RLTs ⚡ to the tumor? A major pain point for mAb-based radioconjugates is the prolonged exposure and low tumor-to-blood ratio, which causes dose-limiting toxicities. To overcome this, Shasqi separates the radionuclide from the targeting component and reunites the two at the tumor using click chemistry. This enables optimal clearance of the RLT and reduces non-specific toxicities. Shasqi's Jesse McFarland and Tri-Hung Nguyen recently presented a poster on this apprpach at the Gordon Research Conferences, 'Exploring the Power of Radioimmunobiology Via Switching the Radionuclides, Ligands and Immunotargets' in beautiful Maine. Learn more: https://bit.ly/46lz190 #RLTs

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Four hematologic oncology researchers at Dana-Farber have been selected for the American Society of Hematology (ASH) 2024 Scholar Award: Nicoletta Cieri, MD, PhD; Reid Merryman, MD; Erin Parry, MD, PhD; and Zuzana Tothova, MD, PhD. The prestigious ASH Scholar Award provides financial support to fellows and junior faculty as they finish training and become independent researchers.  ”I’m honored to receive this prestigious award,” expresses Cieri, a postdoctoral research fellow in the Catherine Wu, MD, lab. ”My project will focus on establishing a new T-cell-based immune-gene therapy approach for acute myeloid leukemia (AML). In AML, there are few effective immunotherapy targets and, unfortunately, the chances of relapsing after transplant are relatively high. This approach will potentially enable personalized targeting of leukemia-specific targets as a therapy to confront the challenge of disease relapse after stem cell transplantation.” The award, for Merryman, will help him and his team continue research into a bispecific antibody called glofitamab, a chemotherapy-free treatment for follicular lymphoma, a slow-progressing blood cancer. ”Glofitamab activates the immune system to target and kill follicular lymphoma cells,” explains Merryman, an attending physician in the Division of Lymphoma and an assistant professor of medicine at Harvard Medical School (HMS). ”In this clinical trial, we’re hoping to create a new treatment option that does not require chemotherapy. We’re also looking to better understand predictors of response and side effects of this treatment. This award will allow me to continue this important research, which I hope will lead to better treatment options for patients.” Parry will use the award to support her lab’s research into changes in the immune system when follicular lymphoma transforms into a more aggressive lymphoma. ”We seek to define these immune system changes that occur with the transformation,” shares Parry, an attending physician in the Division of Lymphoma and the Center for Chronic Lymphocytic Leukemia, and an instructor of Medicine at HMS. ”Our hope is that our research will lead to the ability to identify patients at risk for transformation and identify potential novel strategies to harness the immune system to treat transformation.” This year, Tuthova, who earned the ASH Scholar Award as a fellow in 2017, received it for the second time. “Receiving this award again is incredibly meaningful and humbling,” expresses Tothova, an investigator in Hematologic Neoplasia at Dana-Farber and an assistant professor of medicine at HMS. The award will support Tothova’s project, which aims to identify ways to prevent RUNX1-mutant familial platelet disorder (RUNX1-FPD) from progressing to myelodysplastic syndrome (MDS) or AML. RUNX1-FPD is an inherited condition in which people with genetic mutations in the RUNX1 gene have a 50% higher lifetime risk of developing MDS or AML.

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#ICYMI In this on-demand webinar, hear from Robert Bowman, Assistant Professor of Cancer Biology at University of Pennsylvania School of Medicine as he discusses: 💡 How single-cell DNA sequencing provided insights into the mutant-specific alterations involved in leukemic transformation from premalignant clonal hematopoiesis to AML 💡 How single-cell DNA sequencing was leveraged to model sequential mutagenesis and deterministically investigate mechanisms of transformation and oncogenic dependency in the context of clonal evolution 💡 His thoughts and perspective on the importance of building disease models that can recapitulate human disease and how single-cell DNA sequencing can be a useful confirmation tool Watch on-demand anytime ➡ https://lnkd.in/gEv4b7KT #diseasemodels #cancermodels #webinar #singlecell #singlecellanalysis

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Exciting news from the IASLC and Lung Cancer Research Foundation (LCRF)! The IASLC-LCRF Team Science Research Grant has awarded $2.5 million to a global team led by Dr. David Barbie of the Dana-Farber Cancer Institute to advance treatments for oncogene-driven lung cancers. This groundbreaking research focuses on drug-tolerant persister (DTP) cells, which evade standard therapies and drive cancer recurrence. The distinguished research team includes: Aaron Hata, MD, PhD – Massachusetts General Hospital Eric Smith, MD, PhD – Dana-Farber Cancer Institute Pasi Jänne, MD, PhD – Dana-Farber Cancer Institute Shunsuke Kitajima, PhD – Japanese Foundation for Cancer Research Their two-pronged approach includes: - Immune Modulation: Inhibiting a negative immune regulator to unleash the body's natural defenses against DTP cells. - Enhanced CAR T-Cell Therapy: Developing CAR T-cells designed to identify and eliminate DTP cells. These strategies aim to prevent therapy resistance, improve survival rates, and lay the foundation for transformative clinical trials. Lung cancer remains the leading cause of cancer deaths worldwide, with oncogene-driven forms posing unique challenges. This grant underscores the power of international collaboration and innovative science in the fight against this devastating disease. Learn more: https://bit.ly/LCRFGrant24 

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Excited to announce the 2025 Gordon Research Conference (GRC) on Protein Engineering, this second GRC on Protein Engineering will be held at Bryant University in Smithfield, RI from July 27th to August 1st 2025 https://lnkd.in/g-763SeF. please come join us if you would be interested in this exciting field. Different fields have experienced a transformative expansion in research distinction and capabilities. There now exists a strategic opportunity for protein engineering to take the lead and spearhead the integration of this joint effort, reflected by The Nobel Prize in Chemistry 2024. We believe that this GRC conference will create a gateway for the next generation of engineers and researchers, and will be a vehicle toward future development in the midst of an amazing convergence of multiple cutting-edge areas in protein engineering and its applications. Confirmed speakers include the Nobel Laureate Prof. Frances Arnold and distinctive scientists showing below: 1.            Fundamental Principles of Protein Engineering Possu Huang (Stanford University)/Frances Arnold (CalTech, Nobel Laureate)/Huimin Zhao (University of Illinois at Urbana-Champaign) 2.    Biosensors and Imaging Jennifer Prescher (UC Irvine)/Jin zhang (UC San Diego)/Robert Campbell (Tokyo University)/Nathan Shaner (UC San Diego) 3.    Rational Design of New-to Nature Proteins Jiri Damborsky (FNUSA-ICRC & Masaryk University, Czech Republic)/Joelle Pelletier (University of Montreal)/Sílvia Osuna (University of Girona & ICREA, Spain)/Koga Nobuyasu (Osaka Univ, Japan) 4.    AI for Protein engineering Zhongyue John Yang (Vanderbilt University)/ Jennifer Listgarten (UC Berkeley)/Doug Barrick (Johns Hopkins University) 5.    Biocatalysis and Industrial applications Carlos Martinez (Pfzer)/ Stephanie Galanie (Merck)/Zhi Li (National University of Singapore, Singapore)/Brent Dorr (Ginkgo Bioworks) 6.    Protein Engineering for Synthetic Biology Pimchai Chaiyen (Vidyasirimedhi Institute of Science and Technology (VISTEC), Thailand)/Weixin Tang (University of Chicago)/Jay Keasling (University of California, Berkeley) 7.    Directed Evolution for Enzyme Optimization Yang Yang (University of California Santa Barbara)/Leilei Zhu (Tianjin Institute of Industrial Biotechnology, Chinese Academy of Sciences, China)/Miguel Alcalde (Institute of Catalysis, CSIC, Spain)/Chang Liu (University of California, Irvine) 8.    Protein engineering for Immunotherapy and other diseases Anna Wu (Beckman Research Institute, City of Hope)/Xue Gao (University of Pennsylvania)/Bingfei Yu (University of Southern California)/Wilson Wong (Boston University) 9.    Repurposing Protein for Novel Chemistry and Applications Stefan Lutz (Codexis Inc)/Xiongyi Huang (Johns Hopkins University)/Xiaojing Gao (Stanford University)

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In our latest review published on Journal of Experimental Medicine (JEM) | Rockefeller University Press, we explore the evolution of preclinical tumor models focusing on gliomas — among the most challenging and complex tumors. We dive into the history of these models, evaluate their strengths and limitations, and outline an exciting vision for the next generation of models in the current Resolution Revolution era. My deepest gratitude for the visionary guidance of my mentor Howard Fine and the incredible journey we’ve undertaken together. https://lnkd.in/dg5sZUq3

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Exciting updates from Bristol Myers Squibb on #mezigdomide (#CC92480) in combination with tazemetostat and dexamethasone for multiple myeloma! Mezigdomide, a potent CELMoD under investigation, has shown promising preclinical results, being significantly more potent than #iberdomide and other #IMiDs. In the CA057-003 trial (#NCT05372354), 13 patients who had undergone a median of 5 prior lines of therapy received #mezigdomide, #tazemetostat, and #dexamethasone. The overall response rate (ORR) was 53.8%, with higher mezigdomide doses yielding an ORR of 71.4%. The combination demonstrated a manageable safety profile, with grade 3/4 TEAEs occurring in 69.2% of patients. Notably, mezigdomide rapidly degrades Ikaros and Aiolos proteins, inducing apoptosis in multiple myeloma cells and stimulating the immune system. The future of CELMoDs like mezigdomide looks promising as they continue to show high synergy with traditional partners and advanced treatments. Get detailed analysis of the abstract here @ https://lnkd.in/gd-xFFqy Stay tuned for more updates! #MultipleMyeloma #ClinicalTrials #Mezigdomide #BMS #CancerResearch #CELMoDs #healthcare #business #clinicalstudies #clinicaltrials #results #phases #top #abstracts #myeloma #oncology #pharmacautical #hematology

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Since 2015, Dana-Farber researchers have been participating in clinical trials that evaluate the use of bispecific antibodies for patients with non-Hodgkin lymphoma. Three bispecific antibody therapies have been FDA approved with more on the horizon. These are exciting new medicines that rally the immune system in a powerful way to fight blood cancers. But rolling out a new type of medicine is a project of its own. The experts at Dana-Farber who have been treating patients with these new medicines are now working on expanding their use. Philippe Armand, MD, PhD, chief of the Lymphoma Program, and Jennifer Crombie, MD, Reid Merryman, MD, and Christine Ryan, MD, physicians and clinical investigators in the Lymphoma Program, are using what they have learned as clinical investigators to run new trials to determine if these medicines could have benefits earlier in treatment, in combination with other therapies, or in other types of lymphoma. “The hope is that these drugs will help improve outcomes for patients with lymphoma,” says Crombie, who has been the principal investigator of a bispecific antibody trial for the past few years. Monoclonal antibodies treat cancer by binding to markers on cancer cells and rallying the immune system to destroy them. Bispecific antibodies are designed to recognize and bind to more than one cell marker. The bispecific antibodies approved for the treatment of B-cell non-Hodgkin lymphoma recognize CD20, a marker known to be on B cells, and CD3, a marker known to be on T cells, which are immune cells that can attack cancer cells. By binding to both, the bispecific antibody pulls the T cells closer to the cancerous B cells, increasing the chances that the T cells will recognize and kill the cancer. “Bispecific antibodies can be a powerful immune-based mechanism because of their dual binding properties,” says Crombie. These medicines are currently approved for use alone in patients with follicular lymphoma or diffuse large-cell lymphoma who have received three lines of treatment and need a new therapeutic option. Some patients may choose to try bispecific antibody therapy after CAR T-cell therapy. Others may opt to try bispecific antibodies before trying, or in lieu of, CAR T-cell therapy. Recommendations will vary depending on the type of lymphoma and other individual factors. These new therapies are administered via infusion or an injection under the skin. However, these immunotherapies can cause side effects that many centers are learning how best to manage. In clinical trials of bispecific antibodies, investigators noted side effects that are like those experienced by patients receiving CAR T-cell therapy. The reactions, however, are often less frequent and less severe. Learn more: https://lnkd.in/e4D8T2GG

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Small molecule drugs could be an affordable alternative to biologics. Katherine Clifford of Tufts University and coauthors compare the health benefits and costs of recent FDA approved small molecule drugs and biologics. Read the full article here: https://lnkd.in/eJdjkycS

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Lake N. Paul, PhD. is in San Diego at the Biotechnology Innovation Organization #BIO2024 conference meeting with colleagues & future clients. Connect with him in person to learn about how, as a Collaborative Research Organization, we are best positioned to support your analytical program. #aav #genetherapy #rarediseases #biophysics #STEM #STEMPhilly #biochemistry #celltherapy #bioanalysisllc #PhillyBusiness #phillyscience

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Are you working on the next generation of kinase inhibitors? Join experts and peers at the 15th Annual Strategies for Targeting Kinases symposium on September 30th in Boston to explore novel chemistries, techniques, and emerging drug modalities for targeting kinases in various diseases. Featured Topics: ▪ Targeting diverse kinase pathways for cancer, inflammation, and more. ▪ Covalent and non-covalent strategies for kinase inhibition. ▪ Utilizing machine learning for kinase inhibitor discovery. ▪ Exploring the "dark kinase" with novel approaches. View the preliminary agenda and register by June 7 for Early Savings! https://lnkd.in/epqYsiQm #kinase #drugdiscovery #targetedtherapy #neurodegeneration #BostonDOT24

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We recently sat down with Dr. George Wang, Vice President of Discovery and Preclinical Services at WuXi Biologics to discuss the importance of looking at developability of an antibody early in the drug discovery process. In the podcast, The Importance of Evaluating Developability in the Antibody Discovery Stage, Dr. Wang defines developability and its significance in achieving scalable production during CMC development, which is essential for large-scale manufacturing before clinical trials. He emphasizes the importance of identifying and addressing developability concerns early in the discovery stage to mitigate risks and ensure cost-effective development. Dr. Wang provides examples where developability studies have helped prevent unforeseen costs and timeline disruptions during biologics discovery and preclinical development phases. He underscores that assessing developability can reveal issues with product aggregation, stability, solubility, or manufacturing efficiencies. https://lnkd.in/g7qY9kXs

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