ADLM SmartBrief
Capricor reports positive results for DMD cell therapy | Gene therapy's benefits for patients with hemophilia B may last five years | Promising clinical trial results for gene therapy in children who are deaf
Created for np3kckdy@niepodam.pl | Web Version
 
December 9, 2025
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Lipidomics-based test detects uropathogens
A study published in Clinical Chemistry highlights the effectiveness of a lipidomics-based, culture-free screening test for uropathogens. The lysozyme-enhanced fast lipid analysis technique, or FLAT, improves detection of both gram-negative and gram-positive bacteria directly from urine samples. This method decreases analytical turnaround time by at least 90%, making it suitable for high-throughput clinical laboratories.
Full Story: Clinical Chemistry (12/5)
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Science Briefs
 
Capricor reports positive results for DMD cell therapy
Capricor Therapeutics' cell therapy deramiocel slowed deterioration of upper limb function in patients with Duchenne muscular dystrophy in a Phase 3 trial. Capricor plans to resubmit its application to the FDA.
Full Story: Fierce Biotech (12/3)
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Gene therapy's benefits for patients with hemophilia B may last five years
CSL Behring's Hemgenix, the first gene therapy for hemophilia B, has shown durable benefits five years post-treatment, according to data from the HOPE-B trial published in The New England Journal of Medicine and presented at the annual meeting of the American Society of Hematology. The study found a 90% reduction in annual bleeding rates and sustained factor IX activity levels.
Full Story: Fierce Pharma (12/7)
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Promising clinical trial results for gene therapy in children who are deaf
Gene therapy targeting the otoferlin gene offers hope of restoring natural hearing to children with a rare form of genetic deafness, with Regeneron planning to seek FDA approval by the end of 2025. Clinical trials have shown significant hearing improvements, especially with the company's DB-OTO treatment.
Full Story: Medscape (12/5)
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Gene therapy may benefit children with blood disorders
The gene therapy exagamglogene autotemcel, or exa-cel, was found to provide clinical benefits for children ages 5 to 11 with transfusion-dependent beta-thalassemia and sickle cell disease, according to two Phase 3 studies. All six evaluable children with TDT achieved transfusion independence, and all four evaluable children with SCD were free of severe vaso-occlusive crises for at least a year.
Full Story: MedPage Today (free registration) (12/7)
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Urine test may help diagnose, stage bladder cancer
A study in The Journal of Molecular Diagnostics suggests that analyzing cell-free DNA in urine could help diagnose and stage bladder cancer. A small fragment of the MYC gene was found to have high specificity and predictive value for identifying muscle-invasive bladder cancer.
Full Story: Inside Precision Medicine (12/5)
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Tech Briefs
 
Review examines liquid biopsy biomarkers for CRC
A review in the journal Frontiers in Oncology explores advances in early diagnostic biomarkers for colorectal cancer, including circulating tumor DNA, circulating tumor cells, extracellular vesicles and protein or microbial markers, as well as corresponding liquid biopsy technologies. The review discusses the clinical performance, advantages, limitations and challenges of each biomarker and technology.
Full Story: Frontiers (12/8)
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AI may help improve liquid biopsies for cancer
A narrative review published in Clinica Chimica Acta highlights the potential of AI-assisted liquid biopsies for early cancer detection. The review covers more than 100 studies and emphasizes the role of exosomes, which carry molecular signatures of cancer cells, in a noninvasive method for early detection through blood or urine tests.
Full Story: News Medical (12/2)
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Call for Abstracts
Are you ready to showcase your latest research to a global audience of laboratory medicine professionals? ADLM (formerly AACC) is now accepting poster abstract submissions for ADLM 2026.
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