Two years after Casgevy received commercial approval, only around sixty people with sickle cell disease or thalassemia have been able to benefit from it, due to a technical hurdle that the next generation of treatments will attempt to overcome
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Experimental patients often find themselves in a paradoxical situation: they must be sick enough to qualify for a clinical trial but healthy enough to endure its side effects. They also need the audacity to subject their bodies to protocols whose safety and efficacy remain unproven. For this reason, many describe them as pioneers or even warriors.
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The first Investigational New Drug (IND) application for base-editing technology has been cleared by the Food and Drug Administration. BEAM-101, developed by Beam Therapeutics, is an ex vivo base-editing product candidate, meaning that it uses a modified form of CRISPR capable of making single base changes without double-stranded DNA cleavage.
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CRISPeR FRENZY 