Authors
Latest:
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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Potential Upcoming Treatments for Duchenne Muscular DystrophyJohn Brandsema, MD, a pediatric neurologist in the Division of Neurology at Children’s Hospital of Philadelphia, discussed several still-investigational drugs and therapies for DMD.
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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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The Importance of Single Cell Analysis to the Field of Cell and Gene TherapyBrian Kim, MBA, the chief executive officer of Mission Bio, discussed the company’s Tapestri platform for single cell sequencing.
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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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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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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.
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STRIVE-02: First-in-Human B7H3 CAR T-Cells for Pediatric Patients With Relapsed/Refractory Solid TumorsThree of the 9 patients who received the infusion demonstrated best overall response of Stable Disease, and investigators determined dose level 2 to be the biologically effective dose.
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Improving Outcomes in Corneal Endothelial Disease With Cell TherapyGreg Kunst, chief executive officer, Aurion Biotech, discussed the potential of cell therapy in treating people with corneal endothelial disease around the world.
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Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic LeukodystrophyThe deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.
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Amit Soni, MD, on Challenges to the Uptake of Gene Therapy for HemophiliaThe medical director of the Center for Inherited Blood Disorders discussed barriers to the widespread adoption of val-rox by the hemophilia A community.
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Mary Jane "MJ" Mulcahey, PhD, OTR/L, CPPC, CLCP, FASIA, on Reevaluating Outcome Measures for Spinal Cord Injury Clinical TrialsThe director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University discussed the research she presented at the first Annual SCI Investor Symposium.
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Expanding the Use of CAR T Therapy With shRNAThe chief executive and chief medical officers of Celyad Oncology discussed the company’s future research and plans.
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Potential of mRNA-Based Gene TherapiesJake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s future research and plans.
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Peter A. Merkel, MD, MPH, on the Challenges of Bringing Cell Therapy to Autoimmune DiseaseThe chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed challenges on the horizon in this rapidly emerging field.
