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STRIVE-02: First-in-Human B7H3 CAR T-Cells for Pediatric Patients With Relapsed/Refractory Solid TumorsThree of the 9 patients who received the infusion demonstrated best overall response of Stable Disease, and investigators determined dose level 2 to be the biologically effective dose.
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Improving Outcomes in Corneal Endothelial Disease With Cell TherapyGreg Kunst, chief executive officer, Aurion Biotech, discussed the potential of cell therapy in treating people with corneal endothelial disease around the world.
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Alessandro Aiuti, MD, PhD, on Durable, Clinically Meaningful Efficacy of Arsa-Cel in Metachromatic LeukodystrophyThe deputy director, clinical research, San Raffaele Telethon Institute for gene therapy, discussed long-term follow-up data of up to 12 years.
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Genetic and Gender Factors Linked to Fuchs Endothelial Corneal Dystrophy in University College London StudyThese findings could support gene therapies targeting CTG18.1 that are currently in development.
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Amit Soni, MD, on Challenges to the Uptake of Gene Therapy for HemophiliaThe medical director of the Center for Inherited Blood Disorders discussed barriers to the widespread adoption of val-rox by the hemophilia A community.
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Mary Jane "MJ" Mulcahey, PhD, OTR/L, CPPC, CLCP, FASIA, on Reevaluating Outcome Measures for Spinal Cord Injury Clinical TrialsThe director of the Center for Outcomes and Measurement in the Jefferson College of Rehabilitation Sciences at Thomas Jefferson University discussed the research she presented at the first Annual SCI Investor Symposium.
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Expanding the Use of CAR T Therapy With shRNAThe chief executive and chief medical officers of Celyad Oncology discussed the company’s future research and plans.
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Potential of mRNA-Based Gene TherapiesJake Becraft, PhD, chief executive officer and cofounder, Strand Therapeutics, discussed the company’s future research and plans.
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Peter A. Merkel, MD, MPH, on the Challenges of Bringing Cell Therapy to Autoimmune DiseaseThe chief of the Division of Rheumatology and professor of medicine and professor of epidemiology at Penn Medicine discussed challenges on the horizon in this rapidly emerging field.
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Fernanda Mesquita, PhD, on Using LAMA2-Exosomes to Improve Cardiomyocyte Survival in StrokeThe research associate at the The Texas Heart Institute discussed preclinical research she presented at AHA’s 2023 Scientific Sessions on MSC-derived exosomes.
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Chiraag Kapadia on Investigating Clonal Hematopoiesis After CAR T-cell TherapyThe MD, PhD candidate at Baylor College of Medicine discussed findings from a new study presented at the 2023 Tandem Meetings.
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Mark Forman, MD, PhD, on Genetic Testing and Further Research in Frontotemporal DementiaThe chief medical officer of Passage Bio discussed research needs in FTD.
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WVU Cancer Institute Cellular Therapy Program Recognized by the Foundation for the Accreditation of Cellular TherapyLauren Veltri, MD Director, WVU Cancer Institute Hematopoietic Malignancy and Cellular Therapy Program, discussed the recent FACT accreditation.
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Eileen Tzng, BA, on Exploring Exosome and RNA-based Treatment Methods for Heart FailureThe life science research professional at Stanford University discussed preclinical research she presented at the American Heart Association’s Scientific Sessions 2023.
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James Gulley, MD, PhD, FACP, on Assessing a Novel TCR Bifunctional Antibody in CPI-Resistant Solid TumorsThe director of the Medical Oncology Service at the National Cancer Institute discussed the START-001 trial.
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CGTLive®’s Weekly Rewind – March 21, 2025Review top news and interview highlights from the week ending March 21, 2025.
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Deborah Phippard, PhD, on the Challenges of Evaluating Gene Therapy for Parkinson DiseaseThe chief scientific officer of Precision for Medicine discussed unique difficulties of designing clinical trials for PD gene therapy candidates.
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Performing In-Depth Analysis of Lentiviral Cell Therapies: Luca Biasco, PhDThe director of research and development at AVROBIO discussed the molecular follow-up of cell therapies.
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Caribou Biosciences' chRDNA Technology May Enable New Treatments for ATTR and Familial HypercholesterolemiaSteve Kanner, PhD, the chief scientific officer of Caribou Biosciences, discussed results from preclinical research evaluating the gene editing approach.
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Paul Wuh-Liang Hwu, MD, PhD, on Questions Remaining With AADC DeficiencyUpstaza has been granted marketing authorization by the European Commission.
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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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Gene Therapy for APOE4 Homozygous Alzheimer DiseaseR. Nolan Townsend, Sandi See Tai, MD, and Kim G. Johnson, MD, discussed Lexeo Therapeutics’ LX1001 gene therapy trial that demonstrated promising safety and biomarker effects in patients with early-stage Alzheimer disease.
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Pat Furlong, BSN, RN, on Progress With Research and Advocacy in Muscular DystrophyThe founding president and chief executive officer of Parent Project Muscular Dystrophy discussed how far the field has come since founding the organization/
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CAR-T KITE-363's Safety Profile in R/R B-Cell LymphomaSaurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine, discussed safety and efficacy data from a phase 1 trial for Kite's CD19/CD20-directed CAR-T.
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Peter Cook, PhD, on Exploring DARIC CAR T-Cells for Autoimmune IndicationsThe senior research scientist at Seattle Children’s Research Institute discussed preclinical work on using dimerizing agent-regulated immune-receptor complex T-cells to target plasma cells.
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Eugenio Galli, MD, PhD, on Reexamining Frailty as a Barrier to CAR-T TreatmentThe hematologist at the Hematology and Stem Cell Transplants Unit of Fondazione Policlinico Universitario A. Gemelli IRCCS in Rome discussed a study on the effect of comorbidities in patients receiving CAR-T.
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For Cell and Gene Therapies, MCDA Holds Promise for Improving Equity in Value AssessmentAssessing the value of novel therapies has been challenging and controversial using existing methods, pointing to the need for continued exploration of new approaches.
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Novel Approaches for the Prevention and Treatment of Inflammatory Bone LossCurrent novel therapeutics for the prevention and treatment of bone loss in patients with inflammatory joint disease target cytokines and other inflammatory mediators. Mesenchymal stem cell therapy is a compelling new treatment currently being studied in clinical trials.
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Trial Initiated to Measure Efficacy of T-Cell Therapy in Secondary Progressive MSOpexa Therapeutics is initiating a phase IIb trial of a novel therapy that targets T-cells in patients with secondary progressive multiple sclerosis.
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Osiris Wins Canadian Approval for World's First Stem Cell DrugOsiris Therapeutics said it has received approval from Health Canada to market its stem cell therapy Prochymal for the treatment of acute graft-vs-host disease in children.
