Gene editing

A Phase Ib trial has shown low-density lipoprotein cholesterol (LDL-C) reductions up to 55% and blood PCSK9 protein reductions up to 84% after a single infusion of Verve Therapeutics’ Verve-101. The trial participants have heterozygous familial hypercholesterolemia (HeFH), a life-threatening inherited disease characterised by lifelong elevations in blood LDL-C and accelerated atherosclerotic cardiovascular disease (ASCVD). […]

DDW’s Diana Spencer meets Dr Stephen Jones, Research Professor and Group Leader at the EMBL Partnership Institute for Genome Editing Technologies at Vilnius University’s Life Sciences Center. DS: Can you tell us what you do and what you’re working on at the moment? SJ: I lead a research group at the EMBL Partnership Institute in […]

bit.bio has launched the first offering from its new product range ioCRISPR-Ready Cells, which allow research and drug discovery scientists to knockout any gene of interest in glutamatergic neurons. CRISPR-Ready ioGlutamatergic Neurons are first-in-class stem-cell derived human neurons containing a constitutively-expressed Cas9 nuclease for gene knockouts and CRISPR screens. The potential applications include studies of […]

While attending Life Science Baltics 2023, Diana Spencer spoke to Monika Paule, CEO and Co-founder of Caszyme, about creating a company based on CRISPR gene editing and some of the challenges of operating in this dynamic area of research. DS: What was the motivation for launching Caszyme and what challenges did you face creating a viable […]

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business.    In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio. Dr. Samulski’s graduate work demonstrated the […]

CRISPR gene editing companies Integrated DNA Technologies (IDT) and Aldevron have inked a new global distribution agreement to expand CRISPR products for cell and gene therapy customers. IDT will stock and sell three additional research use only (RUO) and current good manufacturing practice (cGMP) CRISPR nucleases with unique performance characteristics, manufactured by Aldevron, to provide […]

Rolf Turk, Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing. Years of […]

The latest eBook by DDW ‘The impact of genomics on drug discovery: Creating new opportunities’ is out now. This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects. Download this exclusive eBook to learn more about: If animal genomics can help find […]

This 24-page eBook sponsored by The Jackson Laboratory looks at the key use-cases for genomics and how it is improving drug discovery and development projects. Download this exclusive eBook to learn more about: If animal genomics can help find therapies for human disease Key challenges and drivers of the genomics market How transcriptomics is driving drug discovery […]

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 […]