Authors
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Julie Kanter, MD, on Highest Priorities for Sickle Cell After Gene Therapy ApprovalsThe director of the Adult Sickle Cell Clinic and associate professor at University of Alabama Birmingham pointed out access to a sickle cell specialist as one such priority.
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Levi Gray-Rupp, PhD, on Using Integrated Circuit T-Cells Against Solid TumorsThe senior director of immunology at Arsenal Bio discussed advantages of ICTs and the company’s clinical trial in ovarian cancer.
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Mantle Cell Lymphoma Outcomes May Improve With Bispecific CD20/CD19-targeted CAR T-cell TherapiesNirav N. Shah, MD, associate professor at the Medical College of Wisconsin in Milwaukee, discussed the evaluation of LV20.19 CAR in MCL, as well as key efficacy and safety data on the therapy to date.
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Thomas Martin, MD, on Exciting Cell Therapy Options in Multiple MyelomaThe clinical professor of medicine, Helen Diller Family Comprehensive Cancer Center, UCSF, discussed cilta-cel and upcoming cell therapies.
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Stacy Lindborg, PhD, on Addressing Unmet Needs in ALS With Cell TherapyThe co-CEO of BrainStorm Cell Therapeutics spoke about the current standards of care in ALS and the potential of NurOwn to address unmet needs.
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Maria Escolar, MD, on Treating Krabbe Disease With Investigational Gene Therapy FBX-101The chief medical officer of Forge Biologics discussed updated data from the phase 1/2 RESKUE clinical trial that she presented at ASGCT’s 2023 conference.
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Christian Hinrichs, MD, on Improving Solid Tumor Targeting With Engineered T-Cell TherapiesThe chief of cancer immunotherapy at Rutgers Cancer Institute discussed clinical results with different cell therapy approaches.
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Gene Therapy for GM1 Gangliosidosis: Latest UpdatesRalph Laufer, PhD, chief scientific officer, Lysogene, discussed gene therapy programs in GM1 gangliosidosis and Fragile X syndrome.
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Pietro Genovese, PhD, on Using Base-Editing to Protect Healthy Stem Cells from Immunotherapy ToxicityThe principal investigator at the Gene Therapy Program of Dana-Farber/Boston Children’s Cancer and Blood Disorder Center discussed innovative uses of gene editing technology.
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Alexis Thompson, MD, MPH, on the Simultaneous Approvals of Exa-cel and Lovo-cel For SCDThe chief of hematology at Children’s Hospital of Philadelphia discussed dual approval of the 2 gene therapies that coincided with ASH’s 2023 conference.
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Samiah Al-Zaidy, MD, on Gene Therapy for Infantile GM1 GangliosidosisAl-Zaidy, the vice president of clinical development and lead on the GM1 Program at Passage Bio, discussed the results of the phase 1/2 Imagine-01 clinical trial of PBGM01.
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Promising Outlook in Multiple Myeloma with Cilta-Cel: Philippe Moreau, MDPhilippe Moreau, MD, discussed the CARTITUDE-1 trial's promising results with ciltacabtagene autoleucel read out at the 2021 ASCO Annual Meeting.
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Sharon Hesterlee, PhD, on What Attendees Can Look Forward to at MDA’s 2024 ConferenceThe chief research officer of the Muscular Dystrophy Association also went over the highlights from last year’s meeting.
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Michael Kelly, PhD, on Gene Therapy’s Continuing Path to Treating DMDThe chief scientific officer of CureDuchenne discussed challenges to tackle in the field.
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Carlos Moraes, PhD, on Understanding Mitochondrial Mutations for Neurodegenerative DiseasesThe Lichtenstein professor of neurology at University of Miami Miller School of Medicine discussed research his lab is pursuing and its applications.
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Novel Therapies in Development for Dravet SyndromeJoseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.
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Novel Therapies in Development for Dravet SyndromeJoseph Sullivan, MD, and Elaine C. Wirrell, MD, discuss new developments for the treatment of Dravet syndrome beyond seizure control including gene editing and therapy.
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Zevorcabtagene Autoleucel Approved in China for R/R Multiple Myeloma, Phase 2 Study in US OngoingZever-cel is being assessed in the phase 2 LUMMICAR-2 trial (NCT03915184) in North America, previously reporting results of an ORR of 100% among 11 patients.
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Re-Dosing With Gene Therapies in Rare DiseasesTakashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
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Marcus Conant, MD, on Investigating Gene Therapy for HIV SuppressionThe Chief Medical Officer of Addimmune discussed past and future research with the AGT103-T gene therapy.
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Kevin Flanigan, MD, on the Impact of Neuromuscular Disease Gene Therapy ApprovalsThe director of the center for gene therapy at Nationwide Children's Hospital discussed the prospect of gene therapy approvals in Duchenne muscular dystrophy and the associated challenges that remain to be addressed.
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Courtney Young, PhD, on Gene Editing Approaches to Treating Neuromuscular DiseaseThe co-founder and chief executive officer of MyoGene Bio spoke about the company's gene editing therapy for Duchenne muscular dystrophy at MDA’s 2023 conference.
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John A. Charlson, MD, on Afami-Cel’s Upcoming Review for Synovial SarcomaThe associate professor of medicine, Medical College of Wisconsin, discussed advantages of afami-cel and data from the pivotal SPEARHEAD-1 trial.
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Rebecca Cottman, PhD, on Enhancing Cell Therapy Cytotoxicity With a Regulated Gene CircuitThe scientist at Senti Biosciences discussed preclinical research presented at ASGCT 2023.
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Improving Speed and Fidelity of CRISPR/Cas9: Kenneth Johnson, PhD, and David Taylor, PhDThe professors and researchers from The University of Texas at Austin discussed new findings of Cas9 structure and mechanisms.
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Increasing CAR T-cell Use in R/R DLBC Lymphoma: Marin F. Xavier, MD; John M. Burke, MDExperts discuss recommendations to increase the uptake of CAR T-cell therapies into clinical practice and relapsed/refractory diffuse large B-cell lymphoma.
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Jonathan W. Weinsaft, MD, on Working to Bring Novel Therapies to Cardiovascular DiseaseThe chief of cardiology at Weill Cornell Medical College discussed gene therapy research into Friedreich ataxia and more.
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Bart P. Leroy, MD, PhD, on Aligning IRD Gene Therapy Outcome Measures With Activities of Daily LivingThe head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital discussed the need to reevaluate requirements for regulatory approval for ophthalmology gene therapies.
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Tiffany Chen, PhD, on the Role of Preclinical Models in Cell Therapy Research for Autoimmune DiseaseThe vice president of discovery at GentiBio discussed the panel she participated in at the inaugural Cell Therapy for Autoimmune Disease Summit.
