Manufacturers are developing new analytical tools to satisfy increasingly stringent regulatory requirements.
Developers of gene therapies are taking advantage of new vector technologies, incorporating CRISPR systems, and augmenting gene therapy with targeted therapies and immunotherapies.
Rather than inject loose collections of therapeutic cells, implant 3D-bioprinted tissue constructs and gain more control over cell-based precision medicine applications.
Biology’s version of the desktop computing revolution is currently unfolding [with the introduction of] cutting-edge tools. However, biological innovations are much more complicated than working with silicon, requiring more testing and safety precautions. There is a logistical challenge with which biology must contend before it can become a more nimble, high-growth industry.
In one of the first issues of GEN, we published an article about the so-called Cline affair. Martin J. Cline, MD, a professor of medicine at UCLA, achieved notoriety in 1980 when he performed the first gene therapy experiments on human subjects. Cline said the experiments were carried out in Italy and Israel while a request to perform similar experiments at UCLA was still being considered. UCLA eventually denied Cline permission and, amid the controversy surrounding the experiments, forced him to resign his chairmanship at UCLA Medical School. He also lost several research grants. The results of the experiments were reportedly inconclusive.
Studies in rodents by a University of Cambridge-led team demonstrated how delivering two genes simultaneously using a single vector stimulated axonal transport in a humanized tauopathy model of Alzheimer’s disease, and in an experimental glaucoma model. Encouragingly, the results also showed “promising evidence” that the gene therapy might result in improvement in short-term memory in the tauopathy model of dementia. The team said the studies highlight the potential effectiveness of gene therapy for treating complex polygenic conditions that have no single genetic cause.
Scribe—one of a new wave of gene editing biotech companies—focuses on developing CRISPR-based treatments through its genetic modification platform, designed to build and apply its suite of CRISPR technologies designed for therapeutic use.
Gene therapy developers struggle to manufacture trial materials, according to U.K. group behind network of innovation centers intended to help researchers move therapies into the clinic.
In this GEN webinar, sponsored by OXGENE, our panelists—two industry leaders—will discuss what it takes to develop a cell or gene therapy from concept to commercialization, alongside a conversation about innovations that are likely to shape the future of advanced therapy discovery and biomanufacturing.
More recently, the Liu laboratory has described prime editing, which enables the installation of all base substitutions, small insertions, and small deletions by using an RNA rather than a DNA template, as well as the first method for making precise changes to the sequence of mitochondrial DNA.