Authors
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The Potential of Gene Transfer Therapy in SMAJohn Brandsema, MD, discusses gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.
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Dr. Till on the Potential to Utilize CAR T-Cell Therapy Earlier in MCLBrian Till, MD, discusses the potential to utilize CAR T-cell therapy earlier in the treatment of patients with mantle cell lymphoma.
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Cilta-Cel Demonstrates Durable Responses at 18 Months in Relapsed/Refractory Multiple MyelomaCiltacabtagene autoleucel, an investigational BCMA-directed CAR-T therapy, sustained efficacy and durable responses in heavily pretreated patients with relapsed/refractory multiple myeloma.
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The Role of CAR NK Cells: Competition or Compliment to CAR T-Cell Therapy?New studies into chimeric antigen receptor (CAR)–engineered natural killer (NK) cells has shown promising results, explained Ulrike Köhl, PhD, MD, professor of immune oncology and director of the Institute for Clinical Immunology at the University of Leipzig in Germany.
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Gene Transfer for Heart Failure Shows PromiseIntracoronary gene transfer among heart failure patients increased left ventricular function beyond standard heart failure therapy.
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Delays With Payers Mean Some Patients Miss Window for CAR T-Cell Therapy, Findings ShowAjeet Gajra, MD, MBBS, FACP, talks about identifying and removing the barriers for offering CAR T-cell therapy at the community practice level.
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DMD Trial Results Demonstrate Decrease in Creatine Kinase LevelsResults of a Phase 1/2a gene therapy clinical trial in children with Duchenne muscular dystrophy (DMD) revealed a significant decrease in levels of serum creatine kinase, an enzyme biomarker associated with muscle damage caused by DMD.
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Ophthalmology Overview: Gene Therapy Partially Restores Vision in Blind Patient, Presbyopia Therapy, and MoreHighlighting the latest ophthalmology-related news reported across MJH Life Sciences™.
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Scaling the Cell and Gene Therapy Supply Chain for GrowthWhile the cell and gene therapies approved so far are indicated for rare diseases with small patient populations, the successes of chimeric antigen receptor-T (CAR-T) therapies and expanding interest from biopharma stress the need to rapidly scale the supply chain as these therapies move toward commercial availability for more disease states and larger patient populations.
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Selective Inhibitor of FLT3 Allows High-Risk AML Patients to Bridge to Stem Cell TransplantA unique targeted therapy, quizartinib, was able to clear leukemia cells from the bone marrow in more than 33% of patients with an aggressive form of acute myeloid leukemia marked by a mutation in the FLT3 gene.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
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Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
Latest:
Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
Latest:
Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
Latest:
Real-World Economic Value of a 21-Gene Assay in Early-Stage Breast CancerUniversal gene expression profiling of patients with stage II breast cancer resulted in outpatient savings of $11,000 (inclusive of testing costs) within 6 months of initiation of medical therapy.
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Humility and Hope: Evolution of the HIV Pandemic, From ART to Today's Cancer CuresThe connections between cancer and HIV/AIDS became clear relatively early in the HIV/AIDS pandemic and continue to this day. Not only were opportunistic infections present in a majority of HIV-infected patients who met the initial diagnostic criteria for AIDS, but several cancer types were far more prevalent as well. While there is still much to understand before HIV is fully conquered, we have already learned a great deal about the pathobiology of this virus that has helped advanced immune-oncological technologies and led to the development of increasingly effective gene therapy delivery systems.
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Robert Califf, MD, Nominated for FDA CommissionerThe professor of medicine at Duke University School of Medicine previously served as commissioner in 2016.
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Advancements in Gene Therapy for Retinal DiseasesVeeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.
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Luxturna Carries Potential to Correct Blindness, $850k Price TagSpark Therapeutics CEO Jeff Marrazzo reassured patients and the medical community he has an obligation to ensure access to the novel gene therapy.
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FDA Addresses Zolgensma Gene Therapy Data ManipulationThe agency said the gene therapy should remain on the market while it assesses the situation and does not impact their evaluation of data from the human clinical trials.
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MDNN: American Health Trends, the First-Ever Gene Therapy Procedure, and the President's Stance on OpioidsThis week on MDNN: America trends toward healthier outcomes, the first-ever gene therapy procedure was performed, and President Donald Trump declared his stance on opioid traffickers.
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FDA Approves Zolgensma for SMA TreatmentThis is the first gene therapy approved for a devastating condition that leads to permanent ventilation or death for many patients by age 2.
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Human Trials Could Begin for Macular Degeneration Stem Cell TherapyFDA trial approval would make this trial the first ever to test a stem cell-based therapy derived from induced pluripotent stem cells for treating any disease.
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CALD Gene Therapy Granted Breakthrough Therapy DesignationSupported by positive data from an ongoing Phase 2/3 study, bluebird bio’s Lenti-D has been granted Breakthrough Therapy designation by the US FDA for the treatment of patients with cerebral adrenoleukodystrophy.
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CRISPR Gene Editing Treatment for Duchenne Muscular Dystrophy Moves Closer to Clinical TrialsThe latest approach to developing a gene therapy for Duchenne muscular dystrophy shows promising results.
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Rituximab Therapy Correlates with Delayed Progression in Secondary Progressive Multiple SclerosisThe study implies that B-cell depletion by rituximab therapy may be therapeutically beneficial in patients with secondary progressive multiple sclerosis.
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St. Jude Develops Gene Therapy for Severe Combined ImmunodeficiencyResearchers at St. Jude Children’s Research Hospital have developed a new gene therapy that creates fully-functioning immune systems in babies diagnosed with severe combined immunodeficiency, commonly referred to as the “Bubble Boy” disease.
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Breakthrough Designation for Hemophilia TherapyThe FDA gave a gene therapy for bleeding in hemophilia B breakthrough status.
