Authors
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Mathula Thangarajh, MD, PhD, on the Potential of Gene Therapy to Address Cognitive Symptoms in Muscular DystrophyThe child neurologist and neuromuscular physician at Virginia Commonwealth University also discussed the session she chaired at the 2023 MDA Clinical and Scientific Conference.
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Future Research With Stem Cell Therapies in Hematologic MalignanciesThe chief executive and chief medical officers of Vor Biopharma discussed the company’s future research and plans.
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Franck Morschhauser, MD, PhD, on Next Steps for Follicular Lymphoma and Liso-CelThe professor of hematology at University of Lille and chair of LYSA also discussed what the group will be focusing on in 2024.
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Improving Access to Rare Disease CarePam Gavin, executive vice president of NORD, discussed actions the organization is taking to further improve access to advanced care.
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Current Limitations of CAR T-Cell Therapy in Multiple Myeloma: Muhamed Baljevic, MDThe assistant professor from University of Nebraska Medical Center discussed the current limitations of CAR T-cell therapy in multiple myeloma.
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CRISPR-Mediated In Vivo Epigenomic ActivationDaniel Hart, PhD, the senior director and head of technology development at Epic Bio, discussed potential applications of the new technology.
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Selecting AAV Manufacturing PlatformsThe chief scientific officer and chief medical officer of Ultragenyx Gene Therapy discussed the company’s future research in gene therapies.
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Francesca Barone, MD, PhD, on Viral Immunotherapy for Glioma, Prostate CancerThe chief scientific officer at Candel Therapeutics discussed progress in investigations with CAN-3110 and CAN-2409.
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The Role of Established Pharmaceutical Companies in Bringing Cell Therapy to Autoimmune DiseaseDaniela van Eickels, MD, PhD, MPH, the vice president and head of medical affairs for Bristol Myers Squibb’s Cell Therapy Organization, also discussed the company’s planned presentation at ACR Convergence 2024.
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Autologous HSCT's Role in the Mantle Cell Lymphoma Treatment LandscapeAutologous hematopoietic stem-cell transplantation maintained its long-term efficacy over interferon alfa in patients with mantle cell lymphoma.
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ASGCT’s Q4 2024 Landscape Report Indicates Steady Rate of GrowthDavid Barrett, JD, the chief executive officer of ASGCT, broke down the highlights of the organization’s final report for 2024.
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Stephanie Tagliatela on Researching AAV for Lennox-Gastaut, Alzheimer Disease, SCN9a PainThe cofounder and chief scientific officer at Encoded Therapeutics shared preclinical research with the company’s AAV and miRNA platforms.
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John Ligon, MD, on Fertility Outcomes in Patients Who Have Received CAR-TThe assistant professor in the department of pediatrics at the University of Florida College of Medicine discussed also discussed the need to set standard guidelines regarding potential fertility issues for CAR-T treatment.
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David-Alexandre Gros, MD, on Using Tegoprubart to Support Genetically Modified XenotransplantThe chief executive officer of Eledon Pharmaceuticals discussed the company’s investigational monoclonal antibody and its use along with EGenesis porcine kidney transplant product.
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Assessing CAR T-Cell Therapy With Novel Targets for R/R Multiple MyelomaOmar Nadeem, MD, clinical director, Myeloma Cellular Therapies, Dana-Farber Cancer Institute, discussed GPRC5D-targeted CAR-T, BMS-986393.
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Craig M. McDonald, MD, on Safety and Efficacy of DMD Cell TherapyThe principal investigator of the HOPE-2 trial discussed safety and efficacy of Capricor’s CAP-1002.
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Improving Newborn Screening for Spinal Muscular AtrophyCrystal Proud, MD, the director of neurology and neuromuscular medicine at the Children’s Hospital of the King’s Daughters in Norfolk, Virginia, spoke about the enhancements to newborn screening for the disease.
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RGX-314 Gene Therapy Efficacious in Wet AMD: Robert L. Avery, MDThe chief executive officer of California Retina Consultants discussed the positive results of RGX-314 gene therapy in wet AMD.
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Adicet's T-Cell Therapy ADI-100 Fast Tracked by FDA for Refractory Systemic Lupus ErythematosusNotably, the product previously received fast track designation from the FDA for relapsed/refractory class III or class IV lupus nephritis.
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Jennifer Gori, PhD, on the Potential of a Prime Editing Approach in Treating Chronic Granulomatous DiseaseThe vice president of research at Prime Medicine discussed a preclinical study on a novel autologous HSCT treatment that was presented at ASGCT’s 2023 annual meeting.
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Maria Pia Morelli, MD, PhD, on Adapting ctDNA and MRD Negativity Into Solid Tumor Cell Therapy ResearchThe assistant professor at MD Anderson Cancer Center discussed a trial she is leading at MD Anderson with NK cells and cetuximab in colorectal cancer.
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Terese Hammond, MD, on Evolving Treatments for ARDS and More With ImmunotherapyThe director of the CCU/ICU at Saint John’s Health Center discussed a possible more collaborative approach to treating patients in the future.
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Robert Wenham, MD, MS, FACS, On Assessing FSHR-CAR-T Therapy in Ovarian CancerThe chair of gynecologic oncology at Moffitt Cancer Center gave an overview of a phase 1 trial being conducted at Moffitt Cancer Center.
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Yara Abdou, MD, on the Advantages of CAR Macrophages in Solid TumorsThe assistant professor of oncology at UNC School of Medicine discussed the first-in-human study being conducted with CT-0508.
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Uttam Rao, MD, MBA, on Comparing Conditioning Regimens for CAR-TThe transplant physician at St. David's South Austin Medical Center of the Sarah Cannon Transplant and Cell Therapy Network discussed a real-world study comparing bendamustine against fludarabine and cyclophosphamide.
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Brett Kopelan, MA, and Heather Fullmer on the Potential Impact of Gene Therapy on Epidermolysis Bullosa’s Treatment LandscapeIn observance of Epidermolysis Bullosa Awareness Week, the debra of America representatives discussed unmet needs for the patient population and the approval of the first EB gene therapy.
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TJ Cradick, PhD, on A Potential Cure for HIV With EBT-101The chief scientific officer of Excision BioTherapeutics discussed the phase 1/2 clinical trial of the CRISPR gene editing therapy.
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Future Research With Car T-Cell Therapy in Multiple Myeloma: Adam Sperling, MD, PhDThe physician at the Dana-Farber Cancer Institute and instructor in medicine at Harvard Medical School discussed future research with CAR T-cell therapy in multiple myeloma.
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Rachel Lynn, PhD, on Reprogramming Cell Therapies to Combat ExhaustionThe associate director of research at Lyell Immunopharma discussed the company’s programs and newest technologies aimed at solid tumors.
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Using Single Cell Sequencing for Gene Editing Quality ControlAnjali Pradhan, MS, the chief product officer at Mission Bio discussed the company’s Genome Editing Solution.
