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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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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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ImmunoLogic, Episode 2: "Charting New Frontiers in CAR T-Cell Safety" With Bruce Levine, PhDIn Episode 2 of ImmunoLogic, Bruce Levine, PhD, discussed the current the risk-benefit-ratio for CAR-T therapy.
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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.
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Arlo-Cel Generates Excitement, Moves Quickly to Phase 3 Studies for Multiple MyelomaArlocabtagene autoleucel shows promise as a potential first in class GPRC5D-targeted CAR T-cell therapy for heavily pretreated multiple myeloma.
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Optogenetic Gene Therapy Well Tolerated For Retinitis PigmentosaInvestigators are evaluating the safety and tolerability of an optogenetic treatment combining a gene therapy and a medical device.
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Henry Kaplan from University of Louisville School of Medicine: Combatting Retinal Degeneration with GlucoseAccording to Henry Kaplan, MD, University of Louisville School of Medicine, "One has to recognize that there are multiple approaches like gene therapy, neuroprotection, stem cell transplantation, and pharmacologic manipulation of other genes really holds the greatest benefit in terms of trying to reverse the inevitable loss of vision."
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Q&A With Jack Reich From Renova Therapeutics: Gene Therapy for Congestive Heart Failure Shows Promise in Early TrialsCongestive heart failure is a serious condition facing people around the world. Early trials of a gene therapy have showed promise to not only help these patients with the condition but also make their hearts healthier.
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Gene Therapy Shows Promise in Neutralizing HIVResearchers safely infuse engineered immune cells in groundbreaking gene therapy study.
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News Reports; March 2007News items reported in this issue: 1) 186-Gene Signature in Cancer Stem Cells Predicts Recurrence 2) National Prostate Cancer Coalition Commences Clinical Trial Education Program 3) Concomitant High-Dose Radiation Therapy Plus Cetuximab Improves Locoregional Control and Reduces Mortality, with No Increase in Radiation Therapy–Associated Toxicity in Patients with Advanced Squamous-Cell Carcinoma of the Head and Neck 4) STAT3 Pathway Inhibitor (Degrasyn) Drug Class Shows Promise in the Treatment of Malignant Brain Tumors
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News Reports; March 2007News items reported in this issue: 1) 186-Gene Signature in Cancer Stem Cells Predicts Recurrence 2) National Prostate Cancer Coalition Commences Clinical Trial Education Program 3) Concomitant High-Dose Radiation Therapy Plus Cetuximab Improves Locoregional Control and Reduces Mortality, with No Increase in Radiation Therapy–Associated Toxicity in Patients with Advanced Squamous-Cell Carcinoma of the Head and Neck 4) STAT3 Pathway Inhibitor (Degrasyn) Drug Class Shows Promise in the Treatment of Malignant Brain Tumors
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Gene Therapy Shows Robust Activity for Duchenne Muscular DystrophyA novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.
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Rare Neurologic Diseases Are Not Truly RareDespite being labeled as rare diseases, a number of neurologic conditions impact more patients than most would believe. The consultant with expertise in ophthalmology, gene therapy, and rare and orphan diseases, chimed in about how these diseases can often be overlooked.
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Ian Miller, MD: Adapting AAV Gene Therapy for SCN1A-Positive EpilepsyThe medical director of the Comprehensive Epilepsy Clinic at Nicklaus Children’s Hospital discusses his early stage gene therapy trial for Dravet syndrome.
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Parkinson and Lewy Body-like Dementia TriggersThe discovery of a possible trigger for PD and Lewy body dementia could provide a new avenue for gene therapy in these neurological conditions.
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CGRPs: New Help in Migraine Prophylaxis?A new class of injectable monoclonal antibodies directed against calcitonin gene related peptide (CGRP) or its receptor may offer hope to migraineurs who have not found effective prophylactic therapy.
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Gene Therapy Ushers in an Era of Hope for Rare DisordersPreclinical trials and success stories suggest that much is riding on vector-based therapies for the treatment of rare neurological conditions.
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Dystrophin Pathway and Treatments Offer Hope for Patients with Duchenne Muscular DystrophyGene therapy has generated excitement as a treatment or even a potential cure for inherited diseases. Among them: Duchenne muscular dystrophy.
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Large Gene Network Required to Predict Response to HER2-Targeted TherapyLajos Pusztai, MD, DPhil, discusses findings that suggest it is unlikely that any single gene can predict response to targeted therapy for patients with HER2-positive breast cancer.
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Steven Buechler Discusses EarlyR Gene Signature in the BIG 1-98 Trial for Breast CancerSteven Buechler, PhD, Department of Applied and Computational Mathematics and Statistics at the University of Notre Dame, discusses independent validation of the EarlyR gene signature in the Breast International Group (BIG) 1-98 study. This randomized, double-blind, phase III trial compared letrozole with tamoxifen as an adjuvant endocrine therapy for postmenopausal women with hormone receptor-positive, early breast cancer
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Sintilimab/Pemetrexed Regimen Significantly Improves PFS in Advanced NSCLCSintilimab injection plus pemetrexed and platinum-based therapy led to a statistically significant improvement in progression-free survival compared with chemotherapy alone as a first-line treatment for patients with locally advanced or metastatic nonsquamous non–small cell lung cancer.
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What's Keeping CAR T-Cell Therapies From the Outpatient Setting?Although these therapies were initially conceived of and developed as inpatient therapies, interest is growing in extending chimeric antigen receptor T-cell therapies to the outpatient setting.
