Genetic research
Investigations into novel gene therapy approaches for cystic fibrosis have highlighted the need to develop treatments for the ultra-rare population with cystic fibrosis who are not candidates for CFTR modulators due to genetic ineligibility or intolerance. Despite the potential of nucleic acid-based therapies (NABTs) to address this unmet need, their
Cancer
Genprex, Inc. has announced that it has received a Notice of Allowance for a patent covering the use of its lead drug candidate, Reqorsa Gene Therapy, in combination with PD-L1 antibodies, such as Tecentriq. This patent will expire in 2037 at the earliest. The company has also been granted patents
Genetic research
Researchers at UMass Chan Medical School have made significant progress in developing a gene therapy for GM2 gangliosidosis diseases, including Tay-Sachs and Sandhoff diseases. A Phase I/II clinical trial successfully demonstrated biochemical correction of the disease with minimal adverse reactions. Trial participants maintained oral feeding for longer periods and
Genetic research
Researchers from the University of Maryland have conducted a study on the intra-arterial (IA) delivery of biologics to the head and neck region in mice. The study found that this method provides a more effective and precise way to deliver biologics compared to systemic infusion. The IA route allows for
Genetic research
Researchers at the National Scientific and Technical Research Council, in collaboration with Instituto de Agrobiotecnologia y Biologia Molecular (IABIMO) and Instituto Nacional de Tecnologia Agropecuaria (INTA), have made significant progress in developing baculoviruses as gene delivery vectors for mammalian systems. According to the study, published in Vaccines, the use of
Genetic research
Researchers at the Chinese Academy of Agricultural Sciences have made a significant discovery in gene therapy, exploring the potential of rotavirus as a versatile oral vaccine vector. The team's findings, published in the journal Microorganisms, highlight the unique characteristics of rotavirus and its capacity to induce mucosal IgA
Cancer research
Researchers at Yale University have made significant progress in the development of a novel cancer gene therapy. According to a recent study published in Science Translational Medicine, a tumor-targeting, cell-penetrating monoclonal antibody, TMAB3, has been found to form stable, noncovalent antibody/RNA complexes that mediate highly specific and functional delivery
Cancer research
Researchers at New York University College of Dentistry have published a new study examining the current applications and future prospects of gene therapy in dentistry. The research highlights the potential of gene therapy in treating various oral diseases, including cancer, cancer-related pain, xerostomia (dry mouth), dental caries, and periodontal disease.
Genetic research
Researchers at the University of California, Davis, are exploring a new way to treat vision loss using gene therapy, thanks to a $3.6 million grant from the National Eye Institute. The innovative approach aims to make gene therapy safer and more effective for patients with serious eye diseases like
Deafness
Researchers at the University of California, Irvine, have made a groundbreaking discovery in treating hereditary deafness through gene therapy. A multicenter clinical trial found that gene therapy is both safe and effective in restoring biological hearing in children and adults. The study, published in Nature Medicine, involved 10 participants aged
Genetic research
At 61, a retinitis pigmentosa patient surnamed Zhao has regained her independence after losing her vision, thanks to a revolutionary gene therapy injection that restored her ability to cook, clean, and walk with ease. The breakthrough discovery of a unique photosensitive protein derived from chickens, known as opsin 5 (cOpn5)
Cancer
Researchers at Duke University have presented new findings in the field of cancer gene therapy, highlighting the potential of attenuated viruses to mediate cancer in situ vaccination. According to the study, published in the journal Microbiology and Molecular Biology Reviews, the use of viruses like poliovirus has shown promise in
