Gene editing

Cell coding company bit.bio has launched a new disease model cell offering enabling scientists to commission disease-relevant mutation of interest.   The company’s new Custom ioDisease Model Cells offering lets scientists choose the disease-relevant mutation they’re interested in. The disease-relevant mutation is engineered into bit.bio’s human ioWild Type cells using CRISPR/Cas9 gene editing.  As disease model cells come […]

A new approach that delivers a ‘one-two punch’ to help T cells attack solid tumours is the focus of a preclinical study by researchers from the Perelman School of Medicine at the University of Pennsylvania. The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times […]

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. Published in Molecular Cell, the study explains the rules determining to what extent genes respond to CRISPR activation, ensuring that future research can be […]

This is the latest episode of the free DDW podcast, “Mass spectrometry and CRISPR are pillars of drug discovery”. It covers two articles written for Volume 23, Issue 1 – Winter 2021/22 of DDW. They are called “How ASMS offers more than seeing what sticks” and “CRISPR screening in the third dimension”. In the first […]

The US Food and Drug Administration (FDA) has cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE).  Intellia is a clinical-stage genome editing company focused on developing potentially curative therapies using CRISPR-based technologies.   The decision will enable the company to include the United States in the global […]

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study.  The study found that four out of five patients diagnosed with Sanfilippo have continued to gain cognitive skills in line with development in healthy children after being given the […]

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 […]