Authors
Latest:
The Future of Sickle Cell Disease’s Landscape of CareMark Walters, MD, a professor in residence for pediatrics at the Sickle Cell Center of Excellence at the University of California discussed gene therapy’s ongoing transformation of the treatment landscape.
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Dr. Dean on the Potential Role of Consolidative CAR T-Cell Therapy in MCLRobert M. Dean, MD, discusses the potential role for consolidative CAR T-cell therapy in mantle cell lymphoma.
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Clinical Implications of Tisagenlecleucel in B-ALL Treatment: Michael R. Grunwald, MD, FACPThe hematologic oncologist at the Levine Cancer Institute discussed the clinical implications of tisagenlecleucel in the treatment of patients with B-cell acute lymphoblastic leukemia.
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Preclinical Models Suggest Promise of CAR-NK Cell Treatment in Several Solid TumorsPrevious research has suggested the efficacy of chimeric antigen receptor natural killer (CAR-NK) cells in hematological malignancies, and more recently, preclinical findings have raised the question of whether the type of treatment could be a viable, potentially more attractive option for solid tumors.
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Examining the Potential of CRISPR Base Editing for Inherited Retinal DiseasesA substantial proportion of patients with inherited retinal disease could be treated with base editing, while therapeutic strategies that focus on common variants could be used to treat a large number of patients with the disease, according to study results.
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Longer-Term JULIET Results Confirm Sustained Response With Tisagenlecleucel in Relapsed/Refractory DLBCLA median 19-month follow-up of the JULIET trial—a single-arm, open-label, multicenter, global, pivotal phase 2 trial of the chimeric antigen receptor-T cell therapy tisagenlecleucel directed against CD19-expressing B cells—has found a 40% complete response and a manageable safety profile in adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL).
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Caring for Children With AADC-d Impacts Parental Ability to Work, Study ShowsThe study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families.
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HIV Latent Reservoir Forms Near the Time of ART Initiation, Researchers FindWhile antiretroviral therapy (ART) can suppress HIV infection, ART cannot completely eradicate HIV, which remains in a latent reservoir in CD4-positive T cells during treatment; discontinuation of ART leads to rapid rebound of the virus. This reservoir forms even when ART is initiated early on in the infection, and while the most widely accepted model of how the reservoir forms involves infection of a CD4-positive T cell as it transitions to a resting state, the dynamics and timing of the reservoir’s formation have been largely unknown.
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Study Identifies Impact of Parkinson Disease Variations on Progression RiskPatients with the GBA mutation of Parkinson disease (PD) were shown to exhibit more severe cognitive decline than patients with idiopathic PD and those with both the GBA and LRRK2 G2019S mutations of PD.
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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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Cost of Care: Outlining the Risks That Go Along With the Benefit of CAR T-Cell TherapyCytokine release syndrome represents a major concern, and source of costs, associated with the life-saving gene therapy.
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Zaia Draws on Decades of Innovation in Infectious Disease for Breakthroughs in Gene TherapyKnown as a gene therapy pioneer, Zaia has spent almost 40 years at City of Hope, in Duarte, California. He was first drawn by the promise of studying cytomegalovirus. Over the decades, his groundbreaking research has encompassed HIV/AIDS, cellular gene transfer therapy, immunotherapy, bispecific antibodies, and now hyperimmune globulin for workers on the frontlines of the coronavirus disease 2019 (COVID-19) pandemic.
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Intra-Articular FX201 Gene Therapy Shows Promise for OsteoarthritisThe therapy was generally well-tolerated and offered substantial pain relief in patients.
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BCMA-Targeted CAR T-Cell Therapy Effective in MM, but With Caveats, Analysis FindsA new meta-analysis finds high rates of efficacy and high rates of toxicity in multiple myeloma patients treated with B-cell maturation agent (BCMA)-targeted chimeric antigen receptor (CAR)- T cells.
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FDA Approves Obe-Cel for Adults With R/R B-Cell Precursor Acute Lymphoblastic LeukemiaThe CAR-T therapy was approved based on the pivotal phase 1b/2 FELIX clinical trial (NCT04404660), which showed good rates of overall complete remission and median duration of remission.
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Opinion: Lifesavers Are Killing With CostsThe market price for gene therapy greatly exceed the costs of the gene editing development and the equipment required for the task.
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EVEREST-1 Trial in Solid Tumors Doses First Patient With A2B530The autologous CAR T-cell therapy A2B530 is being investigated for solid tumors in the multicenter, first-in-human, phase 1/2 EVEREST-1 study.
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Novel Gene Therapy Promising for Sickle Cell DiseaseThe therapy attempts to restore fetal hemoglobin production.
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Gene Therapy for Huntington's Disease Begins Moving from Lab to ClinicNo evidence of adverse events or significant safety concerns has surfaced.
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RGX-314 Gene Therapy for Wet AMD Well Tolerated at 24 WeeksAfter a single dose of RGX gene therapy, the mean change in BCVA was +8 letters in cohort 3 and the average number of injections over the course of 6 months was 1.3 in a phase 1 cohort study.
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CD34+ Cell Therapy Effective for Reducing Angina in NOCAD in Clinical TrialData presented at SCAI 2020 examining a novel cell therapy suggests it could help reduce angina in patients with non-obstructive coronary artery disease.
