Category GENE THERAPY RARE DISEASES

Wyss Institute researchers demonstrate machine-guided engineering of AAV capsids for gene therapy

High-throughput synthetic biology approach reveals hidden AAV features and could help fast-track future gene therapies Adeno-associated viruses (AAVs) have become the go-to vehicle for delivering therapeutic gene cargo to target tissues for the wave of gene therapies that are in development in academic and biotechnology laboratories. However, natural AAVs do not specifically target diseased cells

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Investment in UK cell and gene therapy manufacturing set to continue

New data released shows cell and gene therapy manufacturing facilities are rapidly becoming operational reflecting job expansion in the sector. Additional 6,000m2 of cell and gene therapy manufacturing space expected to become available within the next 12 months. The Cell and Gene Therapy Catapult (CGT Catapult) published the sixth annual UK cell and gene therapy

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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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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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