Authors
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Extending Survival in Extensive-Stage Small-Cell Lung CancerIn this phase III trial, investigators assessed the clinical efficacy and safety of durvalumab with or without tremelimumab with etoposide and carboplatin or cisplatin chemotherapy followed by durvalumab with or without tremelimumab maintenance therapy compared with EP alone as first-line treatment in extensive-stage small-cell lung cancer.
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Dr. Lin on CAR T-Cell Therapy–Related Toxicities in Multiple MyelomaYi Lin, MD, PhD, discusses CAR T-cell therapy–related toxicities in multiple myeloma.
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Gary Owens, MS, on the Potential of ARCUS–Mediated Gene Editing in Duchenne Muscular DystrophyThe associate director for gene therapy discovery at Precision Biosciences discussed preclinical research he presented at ASGCT’s 2023 conference.
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Shalini Shenoy, MD, MBBS, on Comparing Treatment Options for Sickle Cell DiseaseThe director of the Stem Cell Transplant & Cellular Therapy Program at St. Louis Children’s Hospital discussed when the choice should be made to transition from symptom management to curative therapies.
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Jennifer Morganroth, MD, MBA, on the Importance of Preparing Clinical Resources for Genetic MedicinesThe neurology resident at Penn Medicine spoke about the preparations taken in the lead up to tofersen’s PDUFA date, which is set for April 25, 2023.
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Amar Kelkar, MD, on Assessing Price and Value for Cell and Gene TherapiesThe stem cell transplantation physician at the Dana-Farber Cancer Institute discussed how to look at the value therapies provide and how to improve the cost-effectiveness of their use.
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The Need for New Therapies in DLBC Lymphoma: Gilles Salles, MDThe lymphoma service chief at Memorial Sloan Kettering Cancer Center discussed the need for new therapies to treat diffuse large B-cell lymphoma.
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Keys to a Successful Cell and Gene Therapy LaunchThe success of cell and gene therapies relies not just on approvability but on affordability and accessibility. Innovative manufacturing, automation, and digital tools are key to expanding patient access and ensuring commercial viability.
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Gene Therapy Will Be a Major Area of Interest for Attendees at MDA’s 2025 ConferencePaul Melmeyer, MPP, the executive vice president of public policy & advocacy at MDA, discussed the growing interest in gene therapy for the annual meeting.
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Magdalena Cichewicz, PhD, on Improving AAV Gene Therapy With Synthetic PromotersThe scientist II at Senti Biosciences discussed the potential of synthetic promoters to improve target specificity in AAV vector-based gene therapies in retinal diseases and beyond.
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Clive Svendsen, PhD, on Unlocking the Potential of Astrocyte Cell TherapyThe director of the Regenerative Medicine Institute at Cedars Sinai discussed research being conducted with CNS10-NPC-GDNF in multiple indications.
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Emilie Aschenbrenner, PharmD, BCOP, on CAR-T Versus Bispecific Antibodies for the Treatment of LBCLThe hematology coordinator for pharmacy at Froedtert and the Medical College of Wisconsin discussed the advantages of each of the 2 modalities in the third and second line settings.
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Delivering Ultracompact CRISPR Systems With AAVLucas Harrington, PhD, the cofounder and chief scientific officer of Mammoth Biosciences, discussed the company’s mouse model research on treating hypertriglyceridemia.
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David Weinstein, MD, on Delivering Gene Therapy Directly to the BrainThe senior vice president of clinical development at Passage Bio discussed intracisternal magna administration of PBGM01.
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Frank Borriello, MD, PhD, on Next Steps for SUPLEXA-101 for Solid TumorsThe scientific cofounder and chief executive officer of Alloplex Biotherapeutics discussed progress of the phase 1 trial and possible future evaluations of the therapy.
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Marty Giedlin, PhD, on Advantages of Allogeneic NK Cell Therapy ApproachesThe senior vice president of Technical Operations at Senti Biosciences discussed the company’s NK cell logic-gating platform and allogeneic approach.
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ImmunoLogic, Episode 4: "Inflammatory Storms and Gentle Balances: Navigating Toxicities in Immunotherapies" With Caroline Diorio, MD, FRCPC, FAAPIn Episode 4 of ImmunoLogic, Caroline Diorio, MD, FRCPC, FAAP, discussed her research on adverse events associated with cell therapy.
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Unraveling the Genetics of GlaucomaDetails enhance knowledge of biological pathways that contribute to disease pathogenesis.
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Unraveling the Genetics of GlaucomaDetails enhance knowledge of biological pathways that contribute to disease pathogenesis.
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Dr. Hwu on the Uptake of Cellular Therapy in OncologyPatrick Hwu, MD, discusses the uptake of cellular therapy in the treatment of patients with cancer.
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Binod Dhakal, MD, on Benefit of Cilta-Cel in Earlier Lines of Multiple Myeloma TreatmentThe associate professor at Medical College of Wisconsin discussed current studies evaluating cilta-cel and ongoing research.
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Stem Cell Therapy for TBI: Masahito Kawabori, MD, PhDThe associate professor at Hokkaido University and investigator of STEMTRA also discussed other efforts in stroke, Alzheimer disease, and Parkinson disease.
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Gene Transfer Therapy Requires Further Investigation Despite Potential Approval, Review SaysAfter a more than 35-year pursuit, the licensing of a gene therapy product for treating hemophilia may be available within 2 years.
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Dr. Hutson on Challenges With Targeted Therapy in Non-Clear Cell RCCThomas Hutson, DO, PharmD, discusses challenges with targeted therapy in non-clear cell renal cell carcinoma.
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The Potential of Gene Transfer Therapy in SMAJohn Brandsema, MD, discusses gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
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Dr. Till on the Potential to Utilize CAR T-Cell Therapy Earlier in MCLBrian Till, MD, discusses the potential to utilize CAR T-cell therapy earlier in the treatment of patients with mantle cell lymphoma.
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Cilta-Cel Demonstrates Durable Responses at 18 Months in Relapsed/Refractory Multiple MyelomaCiltacabtagene autoleucel, an investigational BCMA-directed CAR-T therapy, sustained efficacy and durable responses in heavily pretreated patients with relapsed/refractory multiple myeloma.
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The Role of CAR NK Cells: Competition or Compliment to CAR T-Cell Therapy?New studies into chimeric antigen receptor (CAR)–engineered natural killer (NK) cells has shown promising results, explained Ulrike Köhl, PhD, MD, professor of immune oncology and director of the Institute for Clinical Immunology at the University of Leipzig in Germany.
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Gene Transfer for Heart Failure Shows PromiseIntracoronary gene transfer among heart failure patients increased left ventricular function beyond standard heart failure therapy.
