Gene editing

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening.  The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations […]

Harvard investigators have developed a new cell therapy approach to eliminate established tumours and induce long-term immunity.   In this early research, Khalid Shah, Professor of Neurosurgery at Harvard Medical School and Brigham and Women’s Hospital, and colleagues tested the vaccine in an advanced mouse model of brain cancer glioblastoma, with promising results. The findings are […]

Vor Bio, a clinical-stage cell and genome engineering company, has announced initial clinical data from VBP101, its Phase I/IIa multicentre, open-label, first-in-human study of tremtelectogene empogeditemcel or “trem-cel” (formerly VOR33) in patients with acute myeloid leukaemia (AML).   The data observed from the first treated patient support the potential of a trem-cel transplant to be successfully […]

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases […]

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy.  Fukuyama congenital muscular dystrophy (FCMD) is the second most common form of childhood muscular dystrophy in Japan. The disease manifests itself in the form of a severe neuromuscular disorder.  One form of FCMD is caused […]

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month.   Everyone enjoys a good news story and the drug discovery and development sector is no exception. Seeing tangible results of drug efficacy or therapeutics programmes can take years so it was heartening to read in the Guardian1 […]

The fifth CRISPR in Drug Discovery: From Targets to Therapeutics meeting will be held face to face In Oxford on February 7-8, 2023.  The meeting will focus on the application of genome engineering in identification of novel drug targets through large-scale CRISPR based functional genomics studies, developing advanced cellular and in vivo disease models for […]

CRISPR-based knockouts are the gold-standard genetic perturbation method in target identification and validation. However, efficient CRISPR knockout editing and transfection for arrayed screening require specialised skills and infrastructure that your organisation may not have. What if you could receive ready-to-use CRISPR-edited cells without needing to handle, optimise, and transfect your CRISPR library?  In this on-demand […]

Genomic healthcare data is critical to identify disease risk, ancestry, traits and response to medicines and aids in the development of new targeted therapies – precision medicines. DDW’s Megan Thomas observes developments of accessibility in this sector and the potential impact.  The origins  One might argue that this all started getting exciting with the launch […]

Scientists have shown how stomach cancers can develop resistance to a new class of drugs called ATR inhibitors by switching off the activity of two key genes – raising the possibility of outsmarting cancer by predicting drug resistance in advance.  The findings could pave the way for a genetic test to predict which patients are […]