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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Stem Cell vs Gene Therapy Processes in Sickle Cell DiseaseA discussion of the course of gene therapy compared with stem cell therapy.
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Carsten Bönnemann, MD, on Identifying At-Risk Genotypes for DMD Gene TherapyThe senior investigator from NINDS discussed findings investigating serious AEs across different trials.
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Ongoing Gene Therapy Trials in Stargardt Disease : Prof. Carel HoyngThe professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
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N. Nora Bennani, MD, on Diving Deeper Into T-Cell LymphomasThe assistant professor at Mayo Clinic School of Medicine shared her outlook and predictions on research with T-cell lymphomas.
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Transforming Challenges Into Action for SLC6A1In honor of Rare Disease Day, observed this year on February 28, Amber Freed, the founder of SLC6A1 Connect, shared thoughts on advocacy and efforts to raise awareness for rare genetic disease SLC6A1.
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B-VEC Shows Quality of Life Improvements in Dystrophic Epidermolysis BullosaKrystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.
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Giedre Krenciute, PhD, on Revitalizing T-cells in CAR T-cell TherapyThe assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.
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Pursuing Slow but Steady Gene Therapy DevelopmentMichael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
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Aimee C. Talleur, MD, on the Challenge of Evaluating Late Effects from CAR-T in Pediatric PatientsThe physician from St. Jude Children’s Research Hospital discussed the importance of involvement from patients and families while researching the long-term impact of CAR-T.
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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.
