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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. billion, compared to $19.9

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The use of base editing in stem-cell based therapies

Drug Discovery World

To avoid these histocompatibility challenges, early clinical trials often opt for an autologous graft-based approach 8,9 to subvert the need for prolonged immunosuppressive treatment and streamline the grafting process. Regenerative iPSC-derived therapies are not just limited to ophthalmic diseases.