Authors
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Handling Neuromuscular Gene Therapy at Real-World SitesNatalie Goedeker, CPNP, the codirector of pediatric neuromuscular clinical research at Washington University in St. Louis, discussed the session she chaired at MDA’s 2025 conference.
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Lowering Barriers to Access for Liso-Cel Based on New DataManali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado, discussed the implications of a large scale analysis of liso-cel recipients.
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Targeting Unmet Needs in Rare Dermatologic Diseases With Gene TherapyMatthew Gantz, president and chief executive officer, Castle Creek Biosciences, discussed FCX-013 for the potential treatment of scleroderma.
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Recognizing and Managing AEs in CAR T-Cell TherapyKelly Garvin, BSN, RN, OCN, discussed methods to communicate the benefits and risks of CAR T-cell therapy to patients and recognize AEs.
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Olivier Danos, PhD, on Enhancing Gene Therapy for DMDThe chief scientific officer at REGENXBIO discussed RGX-202 and the ongoing clinical trial.
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Evaluating Lyell’s CAR-T IMPT-314 in LBCLSarah Larson, MD, the medical director of the Immune Effector Cell Therapy Program at UCLA, discussed initial data from a phase 1/2 trial.
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Overcoming Barriers to TIL Use in Patients With Solid TumorsClosing out their discussion on TIL therapy, Drs Sarnaik and Komanduri identify existing barriers to TIL use in clinical practice and how the field might optimize patient access to these regimens.
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Shahzad Raza, MD, on Future Plans for CAR T-Cell Therapy NXC-201 in R/R Light Chain AmyloidosisThe hematologist/oncologist at the Cleveland Clinic discussed next steps after early promising results were presented at ASCO’s 2025 meeting.
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John Murphy, PhD, on Using Gene Editing to Tackle Primary Hyperoxaluria Type 1The chief scientific officer of Arbor Biotechnologies discussed the company’s preclinical candidate ABO-101.
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Future Plans for Evaluating Spur Therapeutics’ FLT201Reena Sharma, MD, an adult metabolic consultant at Salford Royal Hospital, discussed areas of interest for future study for the Gaucher disease gene therapy product.
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A Review of Autologous Stem Cell Transplantation in AmyloidosisIuliana Vaxman, MD, and Angela Dispenzieri, MD, review eligibility criteria for ASCT in AL amyloidosis, conditioning dosing, efficacy in terms of hematologic and organ response, and future areas of research.
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Sharif Tabebordbar, PhD, on Improving In Vivo Gene Editing for DMDThe cofounder and chief executive officer of Kate Therapeutics discussed research that awarded him an Outstanding New Investigator at the ASGCT 2024 meeting.
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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.
