Researchers have used CRISPR-Cas9 to remove CD28 from donor T cells and prevent fratricide before equipping them with ...
Wave Life Sciences has reached alignment with the US Food and Drug Administration on a registrational pathway for WVE-006, a GalNAc-conjugated RNA editing oligonucleotide for alpha-1 antitrypsin ...
Screening ionisable lipids with a gene-editor-sized RNA identified LC-1, an LNP component that retained potency as cargo ...
Incyte will share updated clinical single-cell data characterizing its calreticulin-targeted therapy for myeloproliferative ...
Researchers in Germany have identified a SOX9-responsive regulatory route to IRF6, a major orofacial-cleft risk gene, in ...
The minimal residual disease-positive cohort opens as part of the pivotal-stage development of Soficabtagene Geleucel (Sofi-cel), an off-the-shelf CAR-T cell therapy under evaluation for relapsed or ...
Ole Bock moderated two sessions at the Chinese Society of Cell & Gene Therapy Annual Meeting in Beijing, and CMN received the ...
A UCL-led team has developed a clinical-scale CRISPR-Cas9-AAV6 workflow intended to correct Wiskott-Aldrich syndrome in haematopoietic stem and progenitor cells, reaching 61% knock-in in more than 100 ...
Packing chemical modifications throughout the prime editing guide RNA, rather than only at its ends, raised editing in mouse liver to 67% after a single lipid nanoparticle injection. At a clinically ...
A rare visit to a pharmaceutical R&D facility offers insight into how drug discovery, CRISPR technologies and clinical translation are carried out in practice. The article highlights why greater ...
The US FDA has accepted Intellia Therapeutics’ application for lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) and granted the in vivo CRISPR-Cas therapy priority review. In a company-reported ...
Rationally engineered chimeric attachment sites raise the efficiency with which the serine integrase Bxb1 inserts large DNA cassettes into targeted loci in both human cells and rice. Paired with an ...
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