Orest Hurko, M.D.

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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We look forward to hearing Christine Rothe, CDO of iOmx, speak at the 6th Annual Macrophage-Directed Therapies Summit in Boston on October 2nd. Christine will provide key insights into IOMX-0675, our highly differentiated, fully human, cross-specific antibody targeting LILRB1 and LILRB2. In the presentation, Christine will discuss how IOMX-0675 effectively repolarizes the tumor microenvironment, resulting in potent tumor cell killing both 𝘪𝘯 𝘷𝘪𝘵𝘳𝘰 and 𝘪𝘯 𝘷𝘪𝘷𝘰. Also, Christine will highlight IOMX-0675's unique binding profile, which selectively antagonizes the immunosuppressive receptors LILRB1 and LILRB2 while sparing the immuno-activating family members. This differentiation results in superior macrophage repolarization and immune cell activation compared to other antibodies. 🔬   📢 Stay tuned for further updates!   Hanson Wade Group #CancerResearch #Macrophages #immunotherapy #ImmunoOncology

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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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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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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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Christian Ramirez Amarilla now at #BITS2024 about how inhibiting METTL3, an important RNA methyltransferase, affects RNA structure, adenosine-to-inosine (A-to-I) editing, and the immune response in leukemia models, providing new insights into potential therapeutic approaches for leukemia. #Bioinformatics #LeukemiaResearch #BITS2024

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New open access policy analysis from Institute for Clinical and Economic Review (ICER) and NEWDIGS Initiative at Tufts Medical Center analyzes the challenges and potential policy options for paying for gene therapies. https://lnkd.in/ert_jyag Gene therapies offer transformative potential but bring three interconnected challenges: determining fair prices, managing clinical uncertainty, and addressing short-term budget impacts. This paper outlines policy reforms and market-based tools to help the US healthcare system overcome these challenges, ensuring more equitable and affordable access to gene therapies as they become more prevalent. #GCT #celltherapy #genetherapy #cellandgenetherapy

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Why Gilead Sciences and Arcellx Anito-cel's data shows Promise in Multiple Myeloma Therapy Innovative Safety Profile: Early clinical data suggest anito-cel offers a reassuring safety advantage, with no reported cases of Parkinsonism, cranial nerve palsies, or Guillain-Barré syndrome—potentially addressing critical quality-of-life concerns. Encouraging Efficacy: With a 97% response rate and a median progression-free survival of 30.2 months in Phase 1, anito-cel demonstrates strong potential as an effective treatment option in heavily pretreated multiple myeloma patients. Cutting-Edge Technology: Anito-cel’s unique D-Domain binding technology may contribute to its promising safety and efficacy, reflecting innovation in CAR-T therapy design. #CellTherapy #MultipleMyeloma #InnovationInHealthcare #CAR_T #advancedtherapies #biotech #biotechnology #lifesciences #Data #ASH2024 https://lnkd.in/eCmJ_vP3

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Genomic newborn screening holds immense potential for saving lives. By enabling early detection of rare diseases, genomic sequencing significantly enhances treatment outcomes and saves lives through timely interventions. In this interview, Director of Prenatal Medical Genetics and Associate Director of Research, Massachusetts General Brigham Personalized Medicine, Boston, Nina Gold delves into her research on genomic newborn sequencing, offering expert insights on the feasibility of implementing this for all newborns and the necessary infrastructure to achieve it. Read the full interview here: https://hubs.la/Q02zYyjK0 #NeonatalCare #RareDiseases #GenomicSequencing

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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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#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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Elevate your Clinical Research journey by joining ACRPM as a FREE member. Unlock exclusive resources and connect with industry leaders. 🔗 #ACRPM #Networking #ClinicalResearch

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CMG publishes new ACMG Therapeutics Bulletin: “Trofinetide approved for children and adults with Rett syndrome (RTT)” in GIM Open, the College’s gold open access journal: https://bit.ly/3zOe6iI #Therapeutics #medicalgenetics #GIMOpen #RettSyndrome #MedicalEducation Rett syndrome (RTT) affects 1 in 10,000 to 23,000 females globally, making it one of the most common genetic causes of intellectual and developmental impairment in females. Trofinetide improved neuronal and synaptic function in study participants with RTT. Participants showed improvement in various behavioral, visual & communication aspects. Clinicians should plan for the most frequent side effects of trofinetide (diarrhea & vomiting) should they occur.

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Myostatin inhibition continues to be a key focal point for researchers of muscle-related health concerns. #longevity #muscleloss #wellness #health #fortetropin #myostatin #follistatin

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Join Catherine Ulich (Senior Field Application Specialist) and Anthony Kayas (Account Manger) at Discovery on Target in Boston next week📍 Discovery on Target highlights advances in current and emerging “hot” targets and technologies, as well as target validation strategies for the discovery and development of novel therapeutic agents ranging from biologics to small molecules. Cathrine will be speaking at 9am on 1st October about Accelerating Clinical Translation: Harnessing Biology and Technology with Predictive Models 💡 You can also stop by Booth #212 to speak to our team and find out more about our models! 👋 #Newcells #DiscoveryonTarget #PredictiveModels

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University of Pennsylvania spinout Interius BioTherapeutics raises $67M in a private equity round. The Philadelphia company is seeking to raise a total of $82.5 million from its Series B round. July 9, 2024 Interius was granted Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) clearance by the Australian Therapeutic Goods Administration (TGA) to commence a first-in-human Phase 1 clinical trial of INT2104, its lead in vivo CAR candidate for treatment of B-cell malignancies. #funding #financing #genetherapy #invivo #CARtherapy #therapeutics #Bcellmalignancies #celltherapy #genetherapy #advancedtherapies #regenerativemedicine #lifescience #lifesciences #biotech #biotechnology #clinicaltrials #seriesfinancing #equityfinancing

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Breaking barriers in ADC development: What’s next? 🔎 Antibody-drug conjugates are no longer just a concept – they’re revolutionizing cancer therapy with precision-targeted treatments. However, their success hinges on robust development strategies. Allucent explores how improved analytical methods, scalable manufacturing, and compliance with global regulatory standards are key to unlocking the full potential of ADCs. 🚀 By understanding the complex interplay of science, strategy, and regulation, biotech companies can navigate the challenges of bringing ADCs from the lab to the clinic. Dive into Allucent’s roadmap for clinical success and learn how ADCs are evolving to meet the unmet needs in oncology. 🔬 Full article below ⬇️ https://lnkd.in/dmUUXcdB #ADCtherapies #oncologyinnovation #cancerresearch #targetedtherapies #precisionmedicine #bispecificADCs #pharmabreakthroughs #drugdevelopment 

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