Authors
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Ula V. Jurkunas, MD: CALEC Transplant Feasible for Limbal Stem Cell DeficiencyThe associate professor of ophthalmology at Harvard Medical School discussed early results from a phase 1/2 trial from Mass Eye and Ear and partners.
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Raphaël Ognar on Adding New Options to the Oncologic Cell Therapy ArsenalThe cofounder and chief executive officer of NKILT Therapeutics discussed unique advantages of the CIR platform.
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Bone Marrow DNA Sequencing Predicts Acute Lymphoblastic Leukemia Relapse After TisagenlecleucelFindings from the phase 2 ENSIGN and ELIANA studies suggest DNA sequencing predicts ALL relapse.
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Diane Simeone, MD, on Screening Patients for CAR-T Eligibility Before Solid Tumor RelapseSimeone discussed the progress and challenges seen so far in the BASECAMP-1 observational study.
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Francesca Del Bufalo, MD, PhD, on Evaluating GD2-Directed CAR-T in Pediatric NeuroblastomaThe medical doctor and scientist at Bambino Gesù Chidren’s Hospital discussed phase 1/2 clinical trial data she presented at ASGCT’s 2024 Meeting.
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Florian Eichler, MD, on Addressing Unmet Needs in Canavan Disease With Gene TherapyThe neurologist at Massachusetts General Hospital discussed BBP-812, an investigational AAV vector-based gene therapy being evaluated for Canavan disease in a phase 1/2 trial.
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Cell Therapy May Yield Deep Remissions in Multiple MyelomaIde-cel's March 2021 FDA approval marks a significant advance in relapsed/refractory multiple myeloma treatment.
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Bruce Cree, MD, PhD, MAS, on the Importance of Understanding Disease Pathobiology for Novel Treatment ApproachesThe clinical research director of the UCSF Multiple Sclerosis Center discussed the difference between developing targeted therapies for well-understood diseases like SCD and less well-understood diseases like MS.
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Raising Awareness of CAR T Therapy for Mantle Cell Lymphoma: Luhua Wang, MDThe professor from The University of Texas MD Anderson Cancer Center discussed growing awareness of CAR T-cell therapy among community oncologists and patients with mantle cell lymphoma.
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Shankar Musunuri, PhD, on Continuing Research With Gene Therapy in Inherited Retinal DiseasesThe chairman, chief executive officer, and cofounder of Ocugen discussed programs including OCU400 and OCU410.
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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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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.
