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CRISPR tagging improves accuracy of model cells grown from stem cells

Scienmag

Tagging produces detailed catalog of transcription factors key to making each cell type Credit: Gersbach Lab, Duke University DURHAM, N.C. – A team of biomedical engineers at Duke University has created a new way to turn stem cells into a desired cell type by mastering the language of gene regulatory networks.

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#news #biotech CRISPR tagging improves accuracy of model cells grown from stem cells

BioTech 365

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: CRISPR tagging improves accuracy of model cells grown from stem cells.A team of biomedical engineers at Duke University has created a new way to turn stem cells … Continue reading →

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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The US Food and Drug Administration (FDA) has approved the first gene therapies for the treatment of sickle cell disease, approving two on the same day. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. The mutation causes red blood cells to develop a crescent or “sickle” shape.

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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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First gene therapy trial for Hunter syndrome opens in Manchester, UK

Drug Discovery World

In the combined Phase I and II clinical trial, five children under one year of age with the condition also known as mucopolysaccharidosis type II (MPS II) will be treated with autologous hematopoietic stem cell (HSC) gene therapy.

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Scientists turn cancer cells into anticancer agents

Drug Discovery World

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity. . Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” .