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Magazine: CRISPR gene therapies cut through in 2023

Pharmaceutical Technology

If things go as per plan, in a few months, the US Food and Drug Administration (FDA) will deliberate on the first-of-its-kind CRISPR-based gene therapy for sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The post Magazine: CRISPR gene therapies cut through in 2023 appeared first on Pharmaceutical Technology.

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Gene editing: beyond the hype

pharmaphorum

Sangamo CEO Sandy Macrae told us how his company is being cautious about the hype and finding ways to be financially viable in an emerging space. Maybe in 50 years’ time we’ll be using gene editing to lower cholesterol, but it won’t replace statins in anyone but those with life threatening mutations for a long time”.

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New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing

pharmaphorum

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. He said: “These big pharma companies have every type of technology available to them.

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Europe: Where is the drug discovery innovation?

Drug Discovery World

In this article, DDW’s Diana Spencer highlights several prominent hotspots that are taking the lead on advancing European drug discovery and development. The oldest, still active pharmaceutical company worldwide, Merck, started life in 1668 in Darmstadt, Germany. The CDD will sponsor and manage KWF-supported trials. billion ($7.9

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Novartis says £1.8m Zolgensma is model for gene therapy pricing

pharmaphorum

Novartis has said its gene therapy pricing model, involving a steep one-off payment, could be a template for the hundreds of similar treatments that could follow its Zolgensma for Spinal Muscular Atrophy (SMA). . There are around 350 gene therapies in various studies.”. Zolgensma is model for gene therapy pricing appeared first on.

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FDA Approves Gene Therapy Adstiladrin for the Treatment of Bladder Cancer

XTalks

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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FDA head Califf on tighter accelerated approvals and lower drug prices at JP Morgan

Pharmaceutical Technology

FDA director Robert Califf gave his prognosis for the pharma industry at this year’s JP Morgan Healthcare Conference in San Francisco over January 9–12. That leaves three years for pharma companies to prepare. asked panel chair Ipsita Smolinski, MD of consultancy Capitol Street. Cailiff’s answer was circumspect.

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