Authors
Latest:
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.
Latest:
Gene Therapy for GM1 Gangliosidosis: Latest UpdatesRalph Laufer, PhD, chief scientific officer, Lysogene, discussed gene therapy programs in GM1 gangliosidosis and Fragile X syndrome.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
Michael Kelly, PhD, on Gene Therapy’s Continuing Path to Treating DMDThe chief scientific officer of CureDuchenne discussed challenges to tackle in the field.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
Rebecca Cottman, PhD, on Enhancing Cell Therapy Cytotoxicity With a Regulated Gene CircuitThe scientist at Senti Biosciences discussed preclinical research presented at ASGCT 2023.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
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.
Latest:
Detecting Translocations in Gene Edited CellsVanee Pho, PhD, the chief product officer at Mission Bio, discussed the company’s Genome Editing Solution.
Latest:
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.
Latest:
Efficient Gene Editing in Monogenic DiseasesAndré Choulika, PhD, chief executive officer and cofounder, Cellectis, discussed the company’s expansion into gene therapies.
