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CSL doses first patient with haemophilia B gene therapy Hemgenix

Pharmaceutical Technology

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

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

Pharmaceutical Technology

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research. Securing the supply chain.

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The future of the gene therapy market: driving innovations in biotherapeutics

Pharmaceutical Technology

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient.

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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023. Why is Casgevy a Breakthrough Gene Therapy for Patients with Sickle Cell Disease?

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Forbion European Acquisition and enGene sign merger deal

Pharmaceutical Technology

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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UK boosts life science innovation with £277 million funding

Pharmaceutical Technology

Pharmaron is receiving a £151 million investment in capital to support the growth of operations in Liverpool, quadrupling production capacity for gene therapy and vaccine components. On March 10, the National Health Service provided a grant for building a new cell and gene therapy manufacturing facility in Bristol.

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The new frontiers of pharmaceutical innovation – according to investors ?

Pharmaceutical Technology

Source: GlobalData. In the fourth quarter of 2022, the most recent period for which data is available, GlobalData estimates suggest the value of venture capital investment in pharmaceutical firms worldwide was $7.8 Research – and money – going into even more advanced gene therapies is ramping up. Source: GlobalData.