Authors
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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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Steven W. Pipe, MD, on Data Continuing to Support Etranacogene Dezaparvovec Over SOC in Hemophilia BThe professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed 3-year follow-up data from the HOPE B trial of the approved gene therapy, branded as Hemgenix.
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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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Liso-cel Shows Survival Benefit in R/R Large B-Cell LymphomaA prespecified interim analysis from the phase 3 TRANSFORM study showed an event-free survival benefit with the second-line therapy.
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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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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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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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MSC Therapy Shows Improvements in Open-Label Extension Trial for Patients With Multiple SclerosisAmong 15 tested patients, who received at least 2 IT injections of the MSC therapy, improvements of between 5% and 18% in 25 feet walking were observed.
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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.
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MeiraGTx announces Fast Track designation by FDA for gene therapy candidate to treat ACHMThe FDA has granted Fast Track designation to MeiraGTx's AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM).
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Emerging Data in Diffuse Large B-Cell LymphomaExperts discussed data from trials in lymphoma presented at ASCO 2021.
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Commentary (Frank): Gene Therapy for Head and Neck CancersLamont et al have presented a very clear and concise review of current gene therapy strategies in the management of squamous cell carcinoma of the head and neck. While the presentation highlighted the most important work to date in this expanding field, it also made reference to some controversies and challenges that we are now facing. With this in mind, I would like to expand on and clarify several points raised by the authors.
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HER2 Overexpression and Paclitaxel Sensitivity in Breast Cancer: Therapeutic ImplicationsOverexpression by the HER2 gene plays a significant role in breast cancer pathogenesis, and the phenomenon is commonly regarded as a predictor of a poor prognosis. HER2 overexpression has been linked to sensitivity and/or resistance to hormone therapy and chemotherapeutic regimens, including CMF (cyclophosphamide, methotrexate, and fluoro-uracil) and anthracyclines. Studies of patients with advanced disease demonstrate that, despite the association of HER2 overexpression with poor prognosis, the odds of HER2-positive patients responding clinically to taxanes were greater than three times those of HER2-negative patients. Further studies in preclinical models used combination therapy for breast cancer cells that overexpress HER2, and the use of agents that interfere with HER2 function plus paclitaxel (Taxol) resulted in significant antitumor effects. [ONCOLOGY 11(Suppl):43-48, 1997]
