Genetic research
Genprex, a clinical-stage gene therapy company, has announced positive preclinical research results for its diabetes gene therapy drug candidate, GPX-002, at the 2025 American Diabetes Association (ADA) 85th Scientific Session. The research demonstrated that alpha cells in animal models of T1D underwent transdifferentiation to beta-like cells after being transduced with
Genetic research
Investors who purchased or acquired shares of Rocket Pharmaceuticals between February 27, 2025, and May 26, 2025, may be eligible to participate in a securities class action lawsuit after the FDA placed a clinical hold on the company's Phase 2 pivotal trial for RP-A501, an experimental therapy for
Genetic research
Researchers at the China University of Petroleum (East China) have made a significant breakthrough in cancer gene therapy with the development of an all-in-one nanoplatform for imaging, detection, and therapy. This innovative nanoplatform, which integrates imaging, detection, and treatment capabilities, has shown promising results in targeting and treating cancer cells.
Genetic research
Researchers at Genprex, Inc. made significant strides in diabetes gene therapy by presenting positive preclinical data on GPX-002, a novel therapy for Type 1 and Type 2 diabetes, at the 85th American Diabetes Association Scientific Session in Chicago. The study demonstrated the effectiveness of a non-viral lipid nanoparticle delivery system,
Genetic research
Low-grade epilepsy-associated tumors in pediatric patients can be challenging to treat, with seizures often resistant to medication and resulting in significant morbidity. According to a recent report, a multimodal approach to treatment may improve long-term outcomes and quality of life. The study, conducted by researchers at New York University (NYU)
Genetic research
A recent research review from McGill University has shed light on the efficacy and safety of delandistrogene moxeparvovec in treating Duchenne muscular dystrophy (DMD). The study, financed by the American Academy of Neurology (AAN), evaluated six clinical trials, including four with peer-reviewed data available. The research team found that delandistrogene
Genetic research
Research at Duke University Medical Center has made significant advancements in the field of gene therapy, providing new hope for heart transplant patients. Scientists have developed a method of delivering a muscle-tropic recombinant adeno-associated virus (AAV) to donor hearts during normothermic ex-vivo organ perfusion (EVP) systems. This approach has shown
Genetic research
Roche Holding AG has imposed new dosing restrictions on its gene therapy treatment, ELEVIDYStm, for non-ambulatory Duchenne muscular dystrophy (DMD) patients, following the identification of two fatal cases of acute liver failure (ALF). The company has halted treatment for non-ambulatory patients in both clinical and commercial settings, citing an unfavourable
Genetic research
Researchers at the Mayo Clinic Windland Smith Rice Comprehensive Sudden Cardiac Death Program in Rochester, Minnesota, have made significant progress in the development of gene therapy as a treatment for cardiac arrhythmias. This breakthrough offers a new hope for patients suffering from cardiac arrhythmias, which are a leading cause of
Cancer
Researchers at the Missouri University of Science and Technology have developed a new type of dendrimer gel nanoparticle that targets two key proteins in triple-negative breast cancer (TNBC) cells, urokinase-type plasminogen activator receptor (uPAR) and ribonucleotide reductase R2 (R2). This novel design allows the nanoparticles to deliver the antisense oligonucleotide
Cancer
Researchers from Huazhong University of Science and Technology have developed a novel engineering technology to produce extracellular vesicles, which can be used as a cancer therapy. According to the study published in the Journal of Materials Chemistry B, the new method, based on the autonomous "fusion-exocytosis" process, addresses
Genetic research
A novel gene-based therapy has shown significant promise in providing rapid and sustained pain relief to dogs suffering from osteoarthritis, according to a recent pilot study published in Frontiers in Veterinary Science. The trial, led by CureLab Veterinary, demonstrated that weekly injections of a p62-plasmid gene therapy, ElenaVet, resulted in
