Authors
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Alfonso Sabater, MD, PhD, on Further Research With Gene Therapy in Eye CareThe associate professor of clinical ophthalmology at University of Miami discussed the first topical application of gene therapy to the eye.
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Alice Zhou, MD, PhD, and Omar Butt, MD, PhD, on Surprising Associations With Neurotoxicity and Neurofilament LightThe medical oncologists at Washington University School of Medicine in St. Louis discussed surprising findings from their retrospective study.
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Dr. Skarbnik on the Optimization of CAR T-Cell Therapy Sequencing in MCLAlan P. Z. Skarbnik, MD, discusses the optimization of treatment sequencing with CAR T-cell therapy in mantle cell lymphoma.
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Using Programmable Genomic Integration to Make Edits Big and SmallJohn Finn, PhD, the chief scientific officer of Tome Biosciences, discussed the company’s pipeline for its new technology.
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The Potential of CAR T-Cell Therapy in Adult ALLEunice Wang, MD, and Bijal Shah, MD, MS, discussed unmet needs for adult patients with relapsed/refractory ALL and reflect on the current treatment landscape.
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Expanding Study Populations of Cell Therapy in Sickle Cell Disease: Michael Grimley, MDThe medical director of Bone Marrow Transplantation and Immune Deficiency at Cincinnati Children's discussed data on ARU-1801 presented at ASH 2021.
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Wenjun "Tia" Li, on Reducing Swelling in Mouse Models of Rheumatoid Arthritis With Gene TherapyThe PhD candidate from University of North Carolina – Chapel Hill discussed preclinical research presented at ASGCT 2023.
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Krystof Bankiewicz, MD, PhD, on GDNF Gene Therapy for Parkinson’s and AADC DeficiencyThe professor at Ohio State University and University of California – San Francisco discussed investigations with different delivery methods of GDNF gene therapy.
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Targeting Hematological Malignancies With Autologous and Allogeneic Cell TherapiesThomas Willemsen, president and chief executive officer, Tessa Therapeutics, discussed the company’s technologies and 2023 milestones.
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Carlos A. Ramos, MD, on Alternatives to T-Cell TherapyThe professor of medicine at Baylor College of Medicine discussed research with NK-T cells and alternatives to αβ T-cells.
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Donovan Decker’s Journey in Gene Therapy and Advocacy for LGMD—Breaking BarriersDonovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, discussed his powerful journey as a patient advocate and gene therapy pioneer, shedding light on challenges and progress in LGMD.
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Stem Cell Therapy Device Restores Insulin Production in T1 Diabetes: James Shapiro, MDThe Director of the Islet Transplant Program at the University of Alberta discussed the implantable VC-02 device's success in type 1 diabetes.
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Anelloviruses, a Potential Alternative to AAV for Gene TherapyChris Wright, MD, PhD, the chief medical officer and head of translational research at Ring Therapeutics, discussed research presented at ASGCT 2024.
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Carrie Miceli, PhD, on the Potential of Needle Biopsy Findings to Improve DMD Gene TherapyThe professor of microbiology, immunology, and molecular genetics at UCLA discussed how a better understanding of treatment impact on a cellular level could help improve future gene therapy approaches.
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Casey Maguire, PhD, on Potential Advantages of Encapsulated AAVsThe associate professor and associate investigator of neurology at Harvard Medical School discussed research confirming proof-of-concept with EV-AAVs.
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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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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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IRD Gene Therapy OCU400’s Phase 1/2 TrialArun Upadhyay, PhD, the chief scientific officer and head of research, development, and Medical at Ocugen, also discussed future plans for the gene therapy, which is now in a phase 3 study.
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CARTITUDE Trials in Multiple MyelomaExperts discussed safety and efficacy data of cilta-cel for R/R MM from the CARTITUDE-1 and CARTITUDE-2 studies.
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Matthew D. Weitzman, PhD, and Matthew Charman, PhD, on Further Investigation of the Viral Reproduction ProcessThe investigators from Children’s Hospital of Philadelphia discussed the follow-up studies they are conducting in light of their recent findings regarding DNA virus replication.
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Luke Roberts, MBBS, PhD, on Challenges in Developing Gene Therapy for Heart FailureThe medical director of clinical development at AskBio discussed setbacks in the phase 1 trial of AB-1002 gene therapy.
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Debora Mazzetti, MS, on Multitargeting MicroRNA in GlioblastomaThe research trainee at Brigham and Women’s Hospital discussed developing RNA constructs to aid with RNA interference.
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CAR T Therapy Yields Remission in Systemic Lupus ErythematosusFive patients with lupus treated with anti-CD19 CAR T-cell therapy are now in remission.
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Zachary Crees, MD, on the Potential of Cell Therapy to Treat or Prevent Viral Infections After HSCTThe assistant clinical director of the Center for Gene and Cellular Immunotherapy at Washington University in St. Louis discussed Atara Biotherapeutics' tabelecleucel and AlloVir’s posoleucel.
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Future Research With NK and CAR T Therapy in Multiple Myeloma: Irene Ghobrial, MDThe director at Dana-Farber Cancer Institute discussed future efforts to prevent the development of multiple myeloma.
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Salvador Rico, MD, PhD, on Developing Gene Regulation Therapy for Dravet SyndromeThe chief medical officer of Encoded Therapeutics overviews the company’s timeline and strategy for its gene regulation program for Dravet syndrome.
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Confirming the Safety of Pfizer's Hemophilia B Gene Therapy BeqvezBen Samelson-Jones, MD, PhD, the associate director of clinical in vivo gene therapy at Children’s Hospital of Philadelphia, discussed follow-up data of up to 6 years with investigations of fidanacogene elaparvovec.
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Strategies to Reverse Multiple Sclerosis Neurodegeneration: Robert Zivadinov, MD, PhDThe director of the Buffalo Neuroimaging Analysis Center and professor of Universtiy of Buffalo discussed avenues of research into reversing neurodegeneration in MS.
