Category GENE THERAPY RARE DISEASES

Novartis’ Zolgensma gene therapy study halted by FDA on animal safety concerns

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

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Progressing toward successful gene-based approaches to inherited neurometabolic diseases

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

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Twitter reveals growing global public anxiety about Crispr gene-editing

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

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New KUBio box for viral vectors boosts gene therapy manufacturing

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

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New report predicts growth of gene therapies for neurology indications

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

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Global consortium formed to combat unproven cell banking services

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

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NIH launches new collaboration with Bill & Melinda Gates Foundation to develop gene-based cures for sickle cell disease and HIV on global scale

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

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FDA approves new breakthrough therapy for cystic fibrosis

Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options Source FDA The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older

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