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Cell and gene therapies: why advanced medicines call for specialised logistics

Pharmaceutical Technology

But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space. A new frontier in cancer research.

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FDA grants approval for CSL’s haemophilia B gene therapy

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Modernizing cell culture processes for the next wave of genomic medicine

Pharmaceutical Technology

The field of genomic medicine has reached a true turning point. With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genome 244
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Finding a cure: How cell and gene therapies could revolutionise medicine

Pharmaceutical Technology

Cell and gene therapies are predicted to be the future of medical treatment by providing the body with the means to repair itself and recover from a range of serious conditions and severe diseases. While cell and gene therapies are currently much more expensive than traditional medical treatments, the ultimate savings could be significant.

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Scientists unravel the mystery of genes that are key to brain development

Scienmag

Scientists are starting to understand the precise workings of a type of gene that, unlike other genes, does not code for proteins – the building blocks of life.

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How can long-read nanopore sequencing support gene therapy delivery?

Drug Discovery World

The event will feature experts Daniel Jachimowicz, Senior Bioscience Scientist at AstraZeneca BioPharmaceuticals R&D, Sweden, and Bastian Schiffthaler, Senior Data Scientist at AstraZeneca BioPharmaceuticals R&D, Sweden. The post How can long-read nanopore sequencing support gene therapy delivery?

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Gene therapy cures genetic hearing loss

Drug Discovery World

In the first case of its kind, gene therapy has restored hearing in a patient in the United States. AK-OTOF is a gene therapy being developed for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF).