Authors
Latest:
Gene Therapy for APOE4 Homozygous Alzheimer DiseaseR. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
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Devon J. Shedlock, PhD, on Shifting from Autologous to Allogeneic TherapiesThe chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.
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Mesenchymal Stem Cells and Other Upcoming Treatments for Progressive MS: Jeffrey Cohen, MDThe director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.
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Angela Genge, MD, FRCPC, eMBA, on Targeting STMN2 to Rescue Neurons in ALSThe chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.
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Next Steps for Car T-Cell Therapy in Multiple Myeloma: Faith E. Davies, MDThe director of the Clinical Myeloma Program at NYU Langone Health’s Perlmutter Cancer Center discusses the next steps for CAR T-cell therapy in the treatment of patients with relapsed/refractory multiple myeloma.
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Caspian Oliai, MD, MS, on Making Allo-Transplant Safer for Older PatientsThe medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.
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Clinical Implications of CAR T-Cell Therapy in R/R Myeloma: Frits van Rhee, MD, PhDThe professor and director of the Myeloma Center at the University of Arkansas for Medical Sciences discussed the clinical implications of CAR T-cell therapy for patients with relapsed/refractory multiple myeloma.
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Gene Therapy: The Future of Rare Disease TreatmentExperts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
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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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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.
