he Food and Drug Administration has halted a clinical trial of Novartis’ Zolgensma gene therapy due to a safety concern found in an animal study, the company said Wednesday. The hold affects the Novartis (NVS) clinical trial known as STRONG, which was testing a higher dose of Zolgensma administered by spinal injection to older children
Researchers are making great strides toward de-veloping gene-based strategies to treat a variety of inherited neurometabolic diseases characterized by severe neurological involvement. A review of the approaches currently under preclinical or clinical investigation is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Click here to read the full-text article free on the Human
The first ever analysis of all the tweets discussing Crispr gene-editing has revealed how the public feels about the technology. The findings suggest that over a six years public approval of gene-editing has cooled and that most people chatting about the technology are worried. Twitter offers researchers a vast amount of semi-structured data and provides direct access
GE Healthcare Life Sciences is launching KUBio™ box, a fully-integrated, flexible, and adaptable biomanufacturing environment to accelerate the production of viral vector-based gene therapies. The latest addition to the KUBio modular bioprocessing portfolio, KUBio box is expected to speed gene therapies to market and contribute to increased capacity in the viral vector area. Gene therapies
A new report has predicted that gene therapy development will pick up pace but a high price point continues to pose a challenge. Source EPR According to a new report, ongoing collaborations between different industry players and a buildup of real-world evidence establishing safety and efficacy are expected to drive the growth of gene therapies
The International Society for Cell and Gene Therapy has created a group to tackle the rising number of commercial cell bank services that are misleading patients. The International Society for Cell and Gene Therapy (ISCT) has announced the formation of a global consortium to combat the growing number of unproven commercial cell banking services. The group will
Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects. This new technique is called the “prime editing” and is being developed
Source NIH Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates
A more efficient approach to gene therapy that could lower costs and improve patient outcomes has recently been developed by a team from Scripps Research. This work, published on October 17 in the journal Blood, offers a potential alternative to the standard process of delivering gene therapy, which is expensive, time-consuming, and requires many steps to administer healthy
NIH Director Francis Collins describes evolution of existing collaborations and how the two agencies might facilitate development of gene therapy, such as by having a common template for regulatory review. Source Pink Sheet The National Institutes of Health is looking to build on its partnerships with the US Food and Drug Administration to tackle new
