Authors
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Peter Bross, MD, on the Importance of Transparency and Communication With the FDAThe chief of the oncology branch of the Center for Biologics Evaluation and Research at the FDA discussed how academia and industry can work best with regulatory agencies.
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Adam Cuker, MD, on Higher Efficacy of Gene Therapy Over SOC in Hemophilia BThe associate professor of medicine at University of Pennsylvania discussed new data from the BENEGENE-2 study of fidanacogene elaparvovec.
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ASC Transplant Eligibility in Multiple Myeloma: Elisabet E. Manasanch, MDThe associate professor from The University of Texas MD Anderson Cancer Center discussed eligibility for autologous stem cell transplant in patients with multiple myeloma.
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Optimizing CAR T Therapies for Gastric CancerHong Ma, senior vice president, clinical development, cancer immunotherapy, CARSgen, discussed data from a phase 1 trial of CT041.
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Jacob Appelbaum, MD, PhD, on Investigating SC-DARIC33 and IL-15 for AMLThe senior research fellow at Fred Hutch Cancer Center discussed research trends at the 2023 ASGCT meeting.
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Rita Perlingeiro, PhD, on the Potential of iPS Cell-based Regenerative Medicine in Muscular DystrophyThe Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota spoke about preclinical research with an iPS cell-derived product.
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Adam D. Cohen, MD, on Cilta-Cel for Lenalidomide-Refractory Multiple MyelomaThe director of myeloma immunotherapy at University of Pennsylvania discussed cilta-cel's efficacy in lenalidomide-refractory multiple myeloma.
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Jessica S. Little, MD, on the Risk of Fungal Infection in Patients Receiving HSCT and CAR-TThe transplant infectious diseases physician at Dana-Farber Cancer Institute discussed a case study she presented at the 2024 Tandem Meetings.
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Developing Gene Therapies for AMD: Brandi L. Williams, PhD; Gregory S. Hageman, PhDDirectors from the Moran Eye Center discussed researching gene therapy approaches targeting HTRA1 in AMD.
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Joseph Rossano, MD, on the Potential of Gene Therapy in Danon DiseaseRossano discussed unmet needs for patients with Danon disease and the potential of investigational gene therapy RP-A501.
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Bringing Gene Therapy to Common DiseasesShankar Ramaswamy, MD, the cofounder, chairman, and CEO of Kriya Therapeutics, discussed the company’s goal of bringing gene therapy to a much broader population of patients.
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Liso-Cel Plus Ibrutinib Well-Tolerated in Relapsed/Refractory CLL/SLLUpdated data from the phase 1 TRANSCEND-CLL-004 trial were presented at the International Workshop on CLL.
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Roadblocks Faced by Gene Therapies for Muscular DystrophiesJohn Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, offered insights into the obstacles the clinical community is facing around integrating gene therapies into clinical practice.
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Fiona Freeman, PhD, on Investigating miRNA-29b in Osteosarcoma ModelsThe assistant professor at University College Dublin discussed challenges and different approaches to using microRNA in preclinical models of osteosarcoma.
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Akshay Sharma, MBBS, on Overcoming Challenges for Administration of Ex-Vivo SCD Gene TherapyThe attending physician and assistant member of bone marrow transplantation and cellular therapy at St Jude Children’s Research Hospital also discussed hurdles to accessibility for SCD gene therapy.
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Gene Therapy for Sickle Cell DiseaseExperts discuss the safety profile and cost of gene therapy for the management of sickle cell disease.
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Trying New Approaches to AAV Therapy for GlioblastomaXandra Breakefield, PhD, an investigator of neurology at Mass General Research Institute, discussed research from her grad student, Lisa Nieland, presented at the ASGCT 2024 meeting.
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Brian Kim, MBA, on the Emergence of CRISPR-Based Medicines and the Resurgence of AAVThe CEO of Mission Bio also discussed the company’s plans for collaboration.
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Treatment Options After CAR T Therapy for Large B-Cell LymphomaThe professor of medicine from Duke University School of Medicine discussed treatment approaches for after relapse on CAR T cell therapy or for patients who are ineligible for CAR T therapy.
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The Potential to Apply Immunotherapy DOC1021 to a Broad Array of Tumor TypesLaura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology discussed the broader implications of a positive data readout from a trial for the company’s autologous dendritic cell immunotherapy.
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Chris Bond, PhD, on Allogeneic and Induced Pluripotent Stem Cell Approaches to Cell TherapyThe senior vice president of Research & Early Development at Notch Therapeutics discussed the company’s approach to overcoming the drawbacks of autologous cell therapies.
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Accelerating Therapy Discovery and Approval With AIDavid Dimmock, MBBS, the chief medical officer at Creyon Bio, discussed future applications for the company’s AI-guided discovery platform.
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Neutralizing Antibody Versus Total Antibody Assays in Gene Therapy Development: Key Considerations for Assessing ImmunogenicityViral vectors have become the most common method for delivering gene therapy; however, pre-existing humoral immunogenicity can potentially render a gene therapy ineffective.
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Juliane Gust, MD, PhD, on Evaluating Longterm Neurological Outcomes for CAR-T in Pediatric PatientsThe assistant professor of neurology at University of Washington, Seattle Children's discussed efforts to address a gap in knowledge in the long-term effects of CAR-T, if any, on children’s neurological development.
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Achieving Remission in Lupus With CAR T TherapyTeodora Staeva, PhD, chief scientific officer, Lupus Research Alliance, discussed the potential of the approach in patients with SLE.
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Aneal Khan, MD, MSc, FCCMG, FRCPC, on Evaluating Gene Therapy for Fabry DiseaseThe president of MAGIC Clinic and CEO of Discovery DNA discussed the implications of gene therapy data presented at the 2025 WORLDSymposium.
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Weighing Benefits and Risks of Cell Therapy in Multiple Myeloma: Suzanne Lentzsch, MDThe professor of clinical medicine, director, Multiple Myeloma and Amyloidosis Program, New York-Presbyterian Hospital, discussed utility of cellular therapies in multiple myeloma.
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Tom E. Howard, MD, PhD, on a Gene Editing Approach to Treating Hemophilia AThe clinical professor in the Department of Human Genetics at University of Texas Rio Grande Valley discussed how a personalized gene editing approach may help patients avoid development of FVIII inhibitors.
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Bella Neufeld, PhD, on Addressing Pain Points in AAV Gene Therapy DevelopmentThe director of research and development at Teknova discussed the company’s recent announcement of a new line of products intended to streamline process development in AAV gene therapy manufacturing.
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Nadezhda Omelchenko, MD, on Treating Rare Sarcomas With Autologous Natural Killer Cell TherapyThe research associate at Cancer Center of Southern California in Santa Monica discussed findings from 3 patients treated with a combination therapy that included NK cell therapy SNK01.
