Biotech licenses drug discovery platform from Harvard, Tufts to study complex CNS diseases
Unravel Biosciences has licensed a drug discovery platform from Harvard University and Tufts University to decode and model complex central nervous system (CNS) diseases. Read More
Preclinical data on long-acting tuberculosis drug raises hopes for simplified treatment
A long-acting injectable formulation of rifabutin has delivered high plasma concentrations for 16 weeks in mice, resulting in the prevention of tuberculosis and the clearing of the pathogen from the lungs. Read More
Link between genes, mitochondrial DNA reveals way to tackle cardiovascular disease
The discovery of a link between mitochondria, inflammation, and a pair of genes that help regulate blood cell growth has revealed a potential new target for atherosclerosis therapies. Read More
Health organizations form consortium for gene therapy development
The U.S. Food and Drug Administration, the National Institutes of Health, and other organizations are joining forces to increase the development of gene therapies for rare diseases. Read More
New platform identifies best viral vectors for gene therapy
Researchers have developed a novel computational platform that identifies the best viral vector to deliver a gene therapy to its target. The technology, described October 19 in eLife, promises to speed development of adeno-associated virus-mediated gene therapies against vision loss and other disorders. Read More
Precision Advance aims to improve patient access to cell and gene therapy
Improving patient access to cell and gene therapy treatments is the goal of Precision Advance, an initiative of Precision Medicine Group. Precision Advance provides a set of interconnected services and personnel designed to help bring new therapies to market. Read More
Dialogue with regulators will help bring cell and gene therapies to market
CARLSBAD, CA - Open engagement and frequent communication with regulators is needed throughout the development process for cell and gene therapies to help bring them to market, according to an October 13 session at the Cell & Gene Meeting on the Mesa. Read More
GenSight Biologics to highlight gene therapies at Cell & Gene Meeting
French biotechnology firm GenSight Biologics is highlighting its lenadogene neparvovec (Lumevoq) and GS030 gene therapies at the Cell & Gene Meeting on the Mesa in Carlsbad, CA. Read More
New 4D visualization shows epigenetic processes unfold over time
Scientists have developed a new visualization method to construct 4D models of X chromosome inactivation, lending insight into gene expression and opening new pathways to drug treatments for gene-based disorders and diseases. The findings are being published on October 8 in the Proceedings of the National Academy of Sciences of the United States of America. Read More
'Dimmable' switch for gene therapy shows potential for rare diseases
A new alternative splicing system called Xon can modulate levels of protein expression in gene therapy, much like a dimmer switch. The invention addresses a major shortcoming of most gene therapies, which is the ability to regulate gene expression levels in diseases like spinal muscular atrophy. Read More
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