Source: Pharmaceutical Technology
Source: Pharmaceutical Technology
Image Credit: Shutterstock / Ezume Images
Source: Pharmaceutical Technology
The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. The company is studying EDT-301 in sickle cell disease in a Phase I/II RUBY study (NCT04853576), and is on track to provide a clinical update by mid-2023. Editas had previously announced positive safety and efficacy data with two patients enrolled in the RUBY study, stating that the therapy was well-tolerated and that both patients were successfully engrafted and free of vaso-occlusive events during the follow-up. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4% fetal haemoglobin after five months, per a December 2022 update. Editas has plans to dose 20 patients on this study by the end of this year. EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. This increases the expression of fetal haemoglobin. Editas’ CRISPR therapies represent a different approach to the gene editing technology than the one used by CRISPR Therapeutics, which relies on the CRISPR/Cas9 platform. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Optimise your cell therapy process: a guide to cell thawing
Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer.
This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies.
Source: Pharmaceutical Technology
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