Authors
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Rigo Garcia, MPH, on Improving DE&I in Rare Disease Health CareThe executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
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Utility of CART22-65s and huCART19 in R/R ALL: Noelle V. Frey, MD, MSCEThe associate professor from Perelman School of Medicine, University of Pennsylvania, discussed co-administered CART22-65s and huCART19 in relapsed/refractory acute lymphoblastic leukemia.
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CAR T-Cell Therapy for Mantle Cell Lymphoma: Tycel Jovelle Phillips, MDThe clinical associate professor from the Rogel Cancer Center at Michigan Medicine discussed the future of CAR T-cell therapy in mantle cell lymphoma patients.
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Sukumar Nagendran, MD, on the Potential of TSHA-102 in Pediatric Patients With Rett SyndromeThe president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.
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Amer Beitinjaneh, MD, MSc, MPH, FACP, on Treating EBV+ PTLD With Tab-celThe associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.
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Richard James, PhD, on the Potential of Engineered Plasma Cells in CancerThe associate professor at the University of Washington, and principal investigator at Seattle Children's Research Institute discussed results from a leukemia mouse model.
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Mitchell Horwitz, MD, on Cost-Effectiveness of Omidubicel for Allo-HCTThe professor of medicine at Duke Cancer Institute discussed uptake and research with the cord blood therapy since its approval.
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Vivien Sheehan, MD, PhD, on Choosing the Right Treatment Option for Patients With Sickle Cell DiseaseThe associate professor of pediatrics at Emory University also discussed the need to empower patients and families to make their own treatment decisions.
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Tackling Storage and Logistical Challenges in Cell TherapyThe chief executive officer of Gamida Cell discussed upcoming research on 2 of their investigational agents, GDA-201 and omidubicel, for hematological malignancies.
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Emma Ciafaloni, MD, on the State of Gene Therapy in Neuromuscular DiseaseThe professor of neurology and pediatrics at University of Rochester Medical Center spoke about her session at MDA’s 2023 conference.
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Determining Order of CAR T Therapy: Elias Jabbour, MD; Jae Park, MDThe oncologists from MD Anderson and Memorial Sloan Kettering Cancer Centers discuss sequencing CAR T-cell therapies and other key therapies in patients with B-cell acute lymphoblastic leukemia.
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Mitigating Adverse Events in CAR T-Cell Therapy: Mounzer Agha, MDThe director of the Mario Lemieux Center for Blood Cancers at UPMC Hillman Cancer Center discussed strategies to manage AEs associated with CAR T therapy.
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Judy Lieberman, MD, PhD, on the Changing Field of RNA-Based TreatmentsThe endowed chair in cellular and molecular medicine at Boston Children’s Hospital discussed the rapid advancements in RNA-based treatments in the past 2 decades and potential advancements that remain on the horizon.
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Evaluating siRNA Therapy Fitusiran in Combination With Antithrombin Modulation for HemophiliaSteven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, discussed findings from the open-label extension of the ATLAS studies at ASH 2024.
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Treating Autoimmune Diseases With Smarter Cell TherapiesKaren Walker, chief technology officer, Kyverna Therapeutics, discussed the company’s CAR T-cell and regulatory T-cell technologies.
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Milan Zdravkovic, MD, PhD, on Using CRISPR-Cas Systems to Target PathogensMilan Zdravkovic, MD, PhD, chief medical officer of SNIPR Biome, discussed the company’s ongoing research on targeting E Coli in the blood with CRISPR-based medication.
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Bristol Myers Squibb and 2seventy bio's CAR-T Ide-Cel Effects Responses in CNS-Involved R/R Multiple MyelomaReal-world data showed ide-cel was active in patients with central nervous system manifestations of multiple myeloma.
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M. Peter Marinkovich, MD, on Bringing RDEB Treatment to the Local Level With B-VECThe associate professor of dermatology at Stanford University discussed how the May 2023 approval of B-VEC may shift the treatment field for RDEB.
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Tami John, MD, on the FDA Approvals of Exa-cel and Lovo-celThe clinical associate professor at Stanford Medicine also discussed ongoing trends in sickle cell disease research.
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Using DNA Nanoplasmids as a Diagnostic Tool in CancerDavid Suhy, PhD, the cofounder and chief scientific officer at Earli, discussed the company’s unique approach to cancer diagnosis.
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David Shyr, MD, on Incorporating AI Into MedicineThe clinical assistant professor at Stanford Medicine discussed potential applications for machine learning in analyzing data in medicine.
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CAR T: A Novel Approach for Mantle Cell LymphomaNew treatment options expand management of relapsed or refractory disease.
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Jacques Galipeau, MD, on Exponential Progress With Cell and Gene TherapyThe Don and Marilyn Anderson Professor of Oncology at University of Wisconsin – Madison discussed the state of cell and gene therapy development.
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Vinaya Murthy, MS, MPH, on Genetic Counseling Services for Rare DisordersAs we learn more about genomics and identify more genes tied to rare disorders, the role of genetic counselors will become even more critical.
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Keith Fargo, PhD, on Potential Treatment Avenues for Charcot-Marie-ToothThe chief scientific officer of the CMT Research Foundation discussed investigative cell and gene therapy approaches for treating CMT.
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Sarepta’s DMD Gene Therapy Elevidys Demonstrates Manageable Safety Across 5-Year TimespanIn pooled data from 156 patients, there were no deaths or study discontinuations.
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Future Goals for CAR-T in Autoimmune DiseaseGeorg Schett, MD, vice president research and chair of internal medicine at the University of Erlangen – Nuremberg, discussed potential upcoming innovations in the field.
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Dr. Ghosh on the Role of Off-the-Shelf CAR T-Cell Therapy in MyelomaMonalisa Ghosh, MD, discusses the role of off-the-shelf CAR T-cell therapy in patients with multiple myeloma.
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Axicabtagene Ciloleucel Improves Outcomes in Relapsed/Refractory Follicular LymphomaA comparative analysis of the ZUMA-5 and SCHOLAR-5 trials revealed improvements in outcomes over currently available therapies.
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Larry Anderson, MD, PhD, on the Evolution of Myeloma Care With Cell TherapyThe professor of internal medicine at UT Southwestern Medical Center shared his perspective on the current landscape of CAR-T cell therapy in the care of patients with myeloma.
