Authors
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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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Slowing Tumor Growth in Glioblastoma With Novel AAV TherapyLisa Nieland, a PhD candidate from Lieden University Medical Center and Breakefield Lab at Mass Gen, discussed her work presented at the ASGCT 2024 meeting.
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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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Early Signals of Efficacy With Cystic Fibrosis Gene TherapyJennifer L. Taylor-Cousar, MD, MSCS, a professor of internal medicine and pediatrics at National Jewish Health discussed updated data from the phase 1/2 AEROW trial of 4D-710.
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Maria Escolar, MD, on the Importance of Newborn Screening for Treating Krabbe DiseaseThe chief medical officer of Forge Biologics discussed how expanded newborn screening practices may help the company’s gene therapy for Krabbe disease, FBX-101, reach more patients.
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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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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.
