Authors
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Sekar Kethiresan, MD, on Following up VERVE-101 With Next-Generation Editing TherapiesThe cofounder and chief executive officer of Verve Therapeutics discussed the company’s pipeline.
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MDA 2025 as a Mixer for Strategies and CollaborationSharon Hesterlee, PhD, the chief research officer of the Muscular Dystrophy Association discussed the upcoming MDA Clinical and Scientific Conference, which will be held March 16-19, in Dallas, Texas.
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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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Terence R. Flotte, MD, on the Upcoming ASGCT Annual Meeting and Recent Trends in Gene TherapyThe vice president of ASGCT discussed important research that will be represented at the upcoming conference and challenges for the field to address in the future.
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Working to Address Unmet Needs in Alzheimer Disease With Lomecel-B Cell TherapyJoshua M. Hare, MD, the cofounder and chief science officer of Longeveron, discussed working to show potential benefits in a follow-up phase 3 trial.
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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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Overcoming AAV Barriers and Limitations to Treat Duchenne Muscular DystrophyMelissa Spencer, PhD, spoke about the emerging role of adeno-associated virus as a delivery system for gene therapies targeting muscular dystrophies, and the challenbges associated with them.
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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 More Research Needed With HIV TherapiesThe Chief Medical Officer of Addimmune discussed the unmet need with HIV and research the company is conducting.
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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 Integrating Genetic Research into Cardiovascular MedicineThe chief of cardiology at Weill Cornell Medical College discussed the center’s increased focus on genetic research.
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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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Evan Weber, PhD, on Improving CAR T-Cell FitnessThe assistant professor of pediatrics at Children's Hospital of Philadelphia discussed the role of the FOXO1 gene in T-cell persistence and exhaustion.
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Detecting Translocations in Gene Edited CellsVanee Pho, PhD, the chief product officer at Mission Bio, discussed the company’s Genome Editing Solution.
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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.
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Efficient Gene Editing in Monogenic DiseasesAndré Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.
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Stephanie Cherqui, PhD, on Bringing Attention to Cystinosis and Gene TherapyThe associate professor from UC San Diego discussed promising efficacy and safety data from a phase 1/2 trial.
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Genovefa (Zenia) Papanicolaou, MD, on Managing Infectious Diseases in Patients Receiving Stem Cell Transplant or Cell TherapyThe infectious diseases specialist at Memorial Sloan Kettering Cancer Center discussed the session she chaired for the Infectious Diseases Track at Tandem’s 2024 Meeting.
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BASECAMP-1 Trial Identifies Patients With Solid Tumors to Assess Novel CAR T-Cell TherapyJ. Randolph Hecht, MD, discusses the challenges researchers have faced in the development of cellular therapy for patients with solid tumors, and details how A2B530 could potentially overcome some of these obstacles.
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Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314The adjunct clinical assistant professor, ophthalmology, Keck School of Medicine, University of Southern California discussed new data from the ALTITTUDE trial.
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Haydar Frangoul, MD, on Weighing Gene Therapy Against Haploidentical Transplant for Sickle Cell DiseaseThe medical director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute discussed the pros and cons of potentially curative treatment options for SCD.
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Robert J. Hariri, MD, PhD, on the Unique Potential of Chimeric TherapyThe founder, chairman, and CEO of Celularity shared his view on the concept of “chimeric vigor” and the potential benefits arising from it in the cell therapy field.
