Authors
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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.
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Subretinal Gene Therapy Improves Vision in Patients with Wet Age-Related Macular DegenerationTreatment with recombinant adeno-associated virus vector gene-therapy was shown to be safe and potentially effective in a small test group of patients with neovascular age-related macular degeneration.
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Taking Small Steps Toward an HIV CureResearchers have had little success in developing an HIV cure, but recent studies involving gene therapy, immune-based therapy, reactivation of the immune system, and "very early" treatment have produced promising results.
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How PCSK9 Moved from Gene to Therapy and What Comes NextIt wasn't long ago that researchers first identified a piece of DNA that regulates cholesterol. This gene has been recently developed into a cholesterol-lowering agent and it's time to address where we go from here.
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Updated CRYSTAL Data Support Effectiveness of Cetuximab (Erbitux) in Wild-Type KRAS Metastatic Colorectal CancerAt the 2010 Gastrointestinal Cancers Symposium, new data from the CRYSTAL trial identified BRAF gene mutations as a poor prognostic indicator in metastatic colorectal cancer (mCRC) but not predictive of response to therapy.
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Ronald Crystal, MD: The Possibilities of Gene Therapy in Neurodegenerative DiseaseThe chairman of the Department of Genetic Medicine at Weill Cornell Medicine detailed the ongoing research in developing gene therapy for patients with Alzheimer disease.
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FDA Approves Omidubicel to Reduce Infection Risk in Patients With Hematologic Malignancies Undergoing Stem Cell TransplantInfection rates in patients receiving omidubicel post-stem cell transplant were about 20% less than those in the control group.
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Zolgensma Gene Therapy Data Manipulation, GRF6019 Phase 2 Results in Mild to Moderate Alzheimer, Mind Moments PodcastNeurology News Network for the week ending August 10, 2019.
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SRP-9001 Improves DMD Disease Trajectory, But Falls Short on Primary End PointIn the phase 3 EMBARK trial, treatment with SRP-9001 improved secondary outcomes of time to rise, microdystrophin expression, and 10-meter walk/run.
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Adjuvant Olaparib Approved for BRCA+, High-Risk Early Breast CancerOlaparib is now the first early-line therapy targeting BRCA-mutations in breast cancer.
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Debating the True Benefit of Adjuvant TKIs in High-Risk RCCAdjuvant therapy with tyrosine kinase inhibitors for patients with high-risk renal cell carcinoma (RCC) who have undergone a nephrectomy may be supported by level IIa evidence from the National Comprehensive Cancer Network guidelines.
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CAR T-Cell Therapy Efficacious at 10 Years in Chronic Lymphocytic LeukemiaCarl June, MD, discussed the longest-term follow-up data on CAR T-cell therapy so far.
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Bispecific CD19/CD22 CAR T Cells Active in Pediatric Relapsed ALLTreatment with CD19/22 chimeric antigen receptor CAR T cells induced a promising response in patients with relapsed/refractory B-cell precursor acute lymphoblastic leukemia.
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More Research Needed to Understand COVID-19 Vaccine Effects in CAR T RecipientsFurther understanding of how COVID-19 vaccinations affect CAR T-cell therapy recipients is needed.
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BCMA/CD19 CAR T Shows Efficacy in Newly Diagnosed Multiple MyelomaUpdated data were presented at the ASH 2022 annual meeting.
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Februrary 2008 Clinical Trial ReportsThe Clinical Trials reported in this issue include: PHASE III: 1) What Factors Predict Prognosis in Patients With Malignant Pleural Mesothelioma? 2) Treatment for Patients With High-Risk Postoperative Breast Cancer PHASE II: 1) Epratuzumab Plus Rituximab Equals Non-Hodgkin's Lymphoma Response 2) Topotecan Added to Twice-Daily Chemoradiation to Treat Limited- Stage, Small-Cell Lung Cancer 3) First-Line Therapy for Patients With Advanced or Metastatic Non–Small-Cell Lung Cancer 4) Radiotherapy for Nasal Squamous Cell Carcinoma Therapy PHASE I/II: 1) A New Combination Approach for Advanced-Stage Germ-Cell Tumors
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Targeting Non-Mutant EGFR: Genetic and Clinical Biomarkers May Help Validate Use of InhibitorsA recent study suggests patients may be selected for therapy based on the number of EGFR gene copies, and evaluated for clinical benefit based on the severity of the rash that often develops.
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Gene Signatures Predict for Radiation Response in Breast CancerRecent studies have demonstrated that somatic mutations of ATM denote greater response to radiation therapy and that gene patterns may help predict the timing of recurrence following radiation.
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Using Polygenic Risk Score to Better Outcomes in Primary Open-Angle GlaucomaCurrent studies are using a polygenic risk score to evaluate the risk and clinical outcomes in primary open-angle glaucoma (POAG).
