Cancer & Disease Research
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
Next-generation gene therapy points to base editing
Base editing has emerged as a new player in the gene therapy arena, with rapid advances in technology and evidence of proof of concept in rare genetic disease and beyond. Read More
'Minibrains' grown from stem cells replicate brain's electrical activity
Researchers have grown 3D human brain tissue from stem cells that replicates the patterns of electrical activity found in the brains of patients with Rett syndrome. These "minibrain organoids" can be used to investigate the underlying causes of neurological disease and screen and test potential drug candidates, according to research published on August 23 in Nature Neuroscience. Read More
McMaster, Sartorius partner on improving biomanufacturing
Sartorius Stedim Biotech and McMaster University, which is located in Hamilton, Ontario, Canada, plan to collaborate on an effort to improve advanced manufacturing processes for antibody and virus-based treatments for diseases like COVID-19 and cancer. Read More
Alternative gene therapies could be effective in treating some rare diseases
Bacteria-mediated genetic transfer has emerged as an alternative gene therapy (AGT) for the treatment of some rare diseases, such as phenylketonuria (PKU). This type of therapy is advantageous because it is easily regulated through established protein expression systems. One company, Synlogic, has developed an AGT candidate that uses a bacterial vector to treat PKU. Read More
Conferences
Building Biology in 3D Hybrid Symposium
October 26-27
La Jolla, California United States
Northeast Regional Laboratory Staff and Core Directors (NERLSCD) 2021
November 3-5
Portsmouth, New Hampshire United States
CPhI Worldwide 2021
November 9-11
Milan, Lombardia Italy
Society for Immunotherapy of Cancer (SITC) Annual Meeting
November 10-14
Washington, DC, District of Columbia United States
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