Trinity College Dublin
🇮🇪Ireland
Ireland Launches MAGIC-I: First National Genomics Study to Transform Childhood Cancer Care
• Ireland has initiated MAGIC-I (Molecular and Genomic Interrogation of Childhood Cancer – Ireland), the country's first clinical genomics study in pediatric oncology, aiming to optimize treatment through advanced genetic profiling.
• The five-year project will establish processes for comprehensive genomic analysis for all children and adolescents with cancer in Ireland, potentially enabling earlier diagnosis and personalized treatment approaches.
• Developed through collaboration between Systems Biology Ireland, Children's Health Ireland, Precision Oncology Ireland, and industry partners, the study incorporates innovative concepts like digital twins and computational modeling.
PrecivityAD2 Blood Test Enhances Alzheimer's Diagnosis and Treatment Decisions
• The PrecivityAD2 blood test significantly improves diagnostic confidence for Alzheimer's disease, increasing it from 50-60% to over 90% among clinicians.
• Clinical integration of the PrecivityAD2 test leads to a 35% increase in prescriptions for Alzheimer's disease medications for patients with positive results.
• The test demonstrates high concordance (99%) with intended clinical use and maintains a diagnostic accuracy of 90% when compared to cerebrospinal fluid analysis.
• PrecivityAD2's Amyloid Probability Score 2 (APS2) effectively differentiates Alzheimer's disease pathology, aiding in more accurate diagnosis and treatment planning.
Quoin Pharmaceuticals Initiates Clinical Testing of QRX003 in Pediatric Netherton Syndrome Patient
• Quoin Pharmaceuticals has begun clinical testing of QRX003 in a young child with Netherton Syndrome at Children’s Health Ireland in Dublin.
• This assessment marks the first evaluation of QRX003 in a pediatric patient, with plans to expand the study to include additional pediatric subjects in Spain.
• QRX003 is Quoin's lead product, currently in late-stage clinical trials, showing promising results in adult Netherton Syndrome patients with no treatment-related adverse events.
• The trial aims to broaden the data set for QRX003 and potentially lower the eligibility age for treatment, addressing the unmet need for approved therapies for Netherton Syndrome.
Quoin Pharmaceuticals Initiates Clinical Testing of QRX003 in Pediatric Netherton Syndrome Patient
• Quoin Pharmaceuticals has begun clinical testing of QRX003 in a young child with Netherton Syndrome at Children's Health Ireland, marking the first evaluation in a pediatric patient.
• The clinical assessment is led by Dr. Alan Irvine, a leading dermatologist, aiming to broaden the data set for QRX003 and potentially lower the eligibility age for treatment.
• QRX003 is currently in late-stage clinical trials with promising data showing improvement across multiple endpoints and no treatment-related adverse events in older patients.
• Quoin plans to expand the clinical assessment to include additional pediatric subjects in Spain and has opened or plans to open clinical sites in the US, Saudi Arabia, and the UK.
Quoin Pharmaceuticals Initiates Clinical Testing of QRX003 in Pediatric Netherton Syndrome
• Quoin Pharmaceuticals has begun clinical testing of QRX003 in a young child with Netherton Syndrome, a rare genetic disease with no approved treatments.
• The clinical assessment is led by Dr. Alan Irvine, a leading dermatology researcher, aiming to broaden QRX003's data set in younger patients.
• QRX003, a topical lotion, is currently in late-stage clinical trials and has shown promising results in older Netherton Syndrome patients.
• Quoin plans to expand the clinical assessment to include additional pediatric subjects in Spain, furthering the evaluation of QRX003's efficacy.
Quoin Pharmaceuticals Initiates Clinical Testing of QRX003 in Pediatric Netherton Syndrome Patient
• Quoin Pharmaceuticals has begun clinical testing of QRX003 in a young child with Netherton Syndrome at Children’s Health Ireland, marking the first evaluation of the drug in a pediatric patient.
• The clinical assessment is led by Dr. Alan Irvine, a leading researcher in Netherton Syndrome, aiming to broaden the data set for QRX003 and potentially lower the eligibility age for treatment.
• In addition to five open clinical sites in the United States, Quoin plans to open a sixth site in Saudi Arabia and two more in the United Kingdom to expand patient recruitment.
• QRX003, a topical lotion, is being evaluated in late-stage clinical trials as a potential treatment for Netherton Syndrome, with promising early data showing improvement across multiple endpoints.
Artelo Biosciences' ART27.13 Mimics Cannabis Effects to Combat Cancer-Related Weight Loss
• Artelo Biosciences is developing ART27.13, a drug mimicking cannabis effects, to increase appetite in cancer patients without causing psychoactive effects.
• ART27.13 targets CB1 and CB2 receptors to stimulate hunger and prevent muscle wasting, addressing cancer cachexia affecting 60-80% of patients.
• The drug, formulated as a once-daily capsule, is currently in clinical trials (CAReS) to assess its ability to restore appetite and improve quality of life.
• Researchers emphasize ART27.13's potential to improve patient outcomes by focusing on supportive care beyond tumor treatment.
Gene Therapy Advances Offer New Hope for Treating Vision Loss
• Gene therapy is emerging as a promising approach for treating various inherited and acquired retinal diseases, addressing previously untreatable conditions.
• Advancements in viral and nonviral vector delivery systems have improved the safety and efficacy of gene therapies for retinal diseases by limiting immune responses and enhancing targeted gene expression.
• Clinical trials show significant improvements in visual function in patients with retinal dystrophies caused by mutations in the _GUCY2D_ and _RLBP1_ genes following gene therapy.
• Gene therapy is also being explored for acquired retinal diseases like glaucoma and age-related macular degeneration, offering potential for durable treatments and reduced reliance on frequent injections.
Rheumatoid Arthritis: Macrophage Subtype Identified as Potential Target for Early Diagnosis and Treatment
• Researchers have identified a dominant macrophage subtype (CD40+CD206+CD163+) in the inflamed rheumatoid arthritis (RA) synovium, associated with disease activity and treatment response.
• This macrophage subtype is present early in RA development, even before clinical symptoms manifest, suggesting its potential as an early diagnostic biomarker.
• Targeting the pro-inflammatory responses of these macrophages could offer new therapeutic strategies for RA, especially for patients unresponsive to current treatments.
• The study highlights the potential of reprogramming macrophages towards resolution of inflammation as a novel therapeutic approach in rheumatoid arthritis.
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