MedPath

Tagged News

Jaya Biosciences Secures Key Asian Patents for Novel Alzheimer's Gene Therapy Targeting Lysosomal Enzyme Defects

Precision Medicine
  • Jaya Biosciences received patent allowances in Japan, China, and Singapore for methods of detecting, preventing, reversing, and treating neurological diseases including Alzheimer's disease.
  • The company's lead therapy JB111 is an experimental CNS-directed AAV9-mediated gene therapy targeting PPT1 haploinsufficiency in Alzheimer's patients.
  • Research from Washington University shows that heterozygous loss-of-function mutations in lysosomal enzyme genes represent significant risk factors for adult-onset neurological diseases.
  • The patents cover major Asian markets where Alzheimer's disease affects over 22 million people combined across Japan, China, and Singapore.

IHC Testing Enables Rapid TP53 Mutation Detection in MDS and AML Patients

Precision Medicine
  • Researchers identified a 7% p53 immunohistochemistry threshold that best correlates with multi-hit TP53 mutations in myelodysplastic syndromes and acute myeloid leukemia patients.
  • The IHC testing provides results within 48-72 hours compared to 2+ weeks for next-generation sequencing, enabling faster treatment decisions for high-risk patients.
  • Higher IHC positivity correlated with worse overall survival outcomes, though false-negative results occurred in all TP53 sole nonsense or frameshift mutations.
  • Early TP53 mutation identification allows clinicians to avoid ineffective intensive chemotherapy and expedite stem cell transplant planning for patients with poor prognosis.

Collection of Blood and/or Bone Marrow for Investigation and Characterization of Normal and Abnormal Cell Growth

NCT01174615Recruiting
University of Massachusetts, Worcester
Posted 9/1/2009

CSIR Launches Nationwide Phenome India Health Cohort Study to Develop Personalized Disease Risk Predictions

Precision Medicine
  • The Council of Scientific and Industrial Research has launched Phenome India, a five-year nationwide health cohort study involving over 10,000 CSIR employees to develop personalized risk prediction scores for communicable and non-communicable diseases.
  • Phase II health camps have been completed at multiple CSIR facilities, collecting comprehensive health data including blood biochemistry, molecular assays, imaging assessments, and lifestyle patterns from participants.
  • The study aims to address India's rising cardio-metabolic disorders such as diabetes, obesity, and fatty liver disease by creating precision healthcare interventions tailored to the Indian population.
  • All collected samples will be stored at CSIR Biobank under strict anonymity protocols, with data analysis using AI and machine learning approaches to advance personalized medicine research.

Expert Calls for Standardized Reflexive Biomarker Testing to Address Treatment Delays in Lung Cancer

Precision Medicine
  • Dr. Robert Kratzke from the University of Minnesota warns that delays in molecular testing for non-small cell lung cancer patients can lead to treatment lags that cost lives.
  • The expert advocates for making reflexive biomarker testing standard of care, requiring pathologists to automatically send lung cancer biopsy samples for genomic testing and immunohistochemistry.
  • Policy changes at the American College of Pathology level and insurer requirements are needed to ensure every NSCLC patient receives comprehensive biomarker testing regardless of geographic location.
  • Real-world data and AI integration into electronic health records could help reduce disparities in access to precision oncology treatments, particularly for underserved populations.

Korean Researchers Develop Precision Nanobody Therapy for Lung Adenocarcinoma with 90% Tumor Reduction

Targeted TherapyPrecision Medicine
  • Researchers at Korea Research Institute of Bioscience and Biotechnology developed the A5 nanobody that specifically targets CD155 protein overexpressed in lung adenocarcinoma cells.
  • The A5-LNP-DOX delivery system achieved 2-3 fold higher drug uptake in cancer cells and demonstrated 70-90% tumor burden reduction in animal models.
  • The nanobody therapy suppressed cancer cell migration and invasion by over 50% while showing no major organ toxicity in preclinical studies.
  • Clinical analysis revealed that patients with high CD155 and PXN expression levels had significantly worse survival outcomes, validating the therapeutic target.

Oxford BioDynamics' EpiSwitch Biomarkers Show Promise in Pfizer Bladder Cancer Trial

Precision Medicine
  • Oxford BioDynamics' EpiSwitch blood-based biomarkers demonstrated strong correlation with immune profiles of patient tumors in Pfizer's JAVELIN Bladder 100 trial involving 496 bladder cancer patients.
  • The liquid biopsy technology effectively determined tumor immune activity levels in a less invasive manner than traditional tissue biopsies, supporting treatment decision-making.
  • The study validated EpiSwitch as an accurate method for evaluating cancer patient immune responses, building on its existing 94% accuracy in prostate cancer detection compared to standard PSA testing's 55%.
  • Results support expanding EpiSwitch technology beyond prostate cancer to revolutionize cancer detection and monitoring across various cancer types.

Targeted Therapies Transform Biliary Tract Cancer Treatment Landscape with FGFR2 Inhibitors Leading Clinical Advances

Targeted TherapyPrecision MedicineOncology
  • Biliary tract cancer treatment has evolved from traditional cytotoxic chemotherapy to include targeted therapies based on specific molecular alterations, with FGFR2 inhibitors showing particularly promising results.
  • Pemigatinib and futibatinib, selective FGFR inhibitors, have demonstrated significant clinical efficacy with response rates of 35% and 42% respectively in patients with FGFR2 fusions.
  • Comprehensive next-generation sequencing has become the standard approach for identifying actionable molecular alterations, with IDH1/2 mutations, FGFR2 fusions, and ERBB2 alterations being the most clinically significant biomarkers.
  • The rarity of biliary tract cancers presents unique challenges in treatment access, making clinical trials essential for patients with molecular alterations that have FDA-approved therapies in other disease states.

Hydrogels Emerge as Next-Generation Platform for Precision Cancer Drug Delivery

Precision Medicine
  • A comprehensive bibliometric analysis of 4,108 publications reveals hydrogels as a rapidly growing field in cancer drug delivery, with annual publications exceeding 613 in 2024.
  • Injectable hydrogels offer minimally invasive chemotherapy delivery that reduces systemic side effects while improving therapeutic efficacy through localized drug release and enhanced tumor penetration.
  • Emerging research trends focus on immunotherapy applications, immunogenic cell death mechanisms, and multifunctional antimicrobial hydrogels that combine cancer treatment with infection prevention.
  • Current hotspots include carboxymethyl cellulose-based systems, chitosan nanocomposites, and stimulus-responsive hydrogels that enable precise spatiotemporal drug release control.

Comprehensive Genomic Profiling Emerges as Essential Standard for Personalized Cancer Treatment

Precision MedicineTargeted Therapy
  • Comprehensive genomic profiling has become standard practice for non-small cell lung cancer treatment, enabling identification of actionable genomic alterations that guide personalized therapeutic decisions.
  • The integration of both tissue-based testing and plasma-based next-generation sequencing provides complementary molecular profiling capabilities throughout the patient care continuum.
  • Incomplete genomic testing can leave patients without access to potentially beneficial targeted therapies, emphasizing the critical importance of comprehensive molecular profiling for optimal outcomes.
  • Future applications include expanded serial testing for resistance monitoring and potential expansion into earlier cancer stages as precision medicine approaches continue evolving.

Aqtual Raises $31 Million to Advance Novel cfDNA Platform for Rheumatoid Arthritis Therapy Prediction

Precision Medicine
  • Aqtual secured $31 million in Series B funding to advance its active chromatin cell-free DNA platform toward commercialization of a therapy response prediction test for rheumatoid arthritis.
  • The company is actively enrolling patients in the PRIMA-102 clinical trial, which has enrolled over 1,300 RA patients and is expected to complete in Q4 2025.
  • Aqtual's proprietary platform integrates DNA, epigenetic regulation, and protein-DNA binding in a single blood-based assay to quantify tissue-specific gene regulation.
  • Rheumatoid arthritis contributes over $19 billion annually in direct healthcare costs in the U.S., highlighting the significant unmet medical need for personalized treatment approaches.

Prospective, Multi-Center, Observational, Whole Blood Specimen Collection Study in Participants with Rheumatoid Arthritis with Inadequate Response or Intolerance to a DMARD Starting a New BDMARD or TsDMARD Treatment +/- CsDMARD

NCT05936970Recruiting
Aqtual, Inc.
Posted 6/29/2023

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.