The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize… Read More
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… Read More
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… Read More
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… Read More
The National Institutes of Health has awarded the first phase of a roughly $40m grant to Yale University to advance a novel CRISPR-based gene-editing delivery platform for the targeted treatment of neurogenetic diseases. The two diseases being targeted in the project are Angelman syndrome and H1-4… Read More
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… Read More
A new licensing agreement between Vita Therapeutics and MilliporeSigma (the US and Canada Life Science business of Merck) will see Vita leverage MilliporeSigma’s foundational CRISPR patents to advance its preclinical asset VTA-100 for limb-girdle muscular dystrophy (LGMD2A). VTA-100 utilises this CRISPR genome editing technology to insert a functional copy of… Read More
Listen to this article on the DDW Podcast: 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,… Read More
Listen to this article on the DDW Podcast: Mark Kotter, Founder and CEO of bit.bio, talks with Reece Armstrong about different approaches to synthetic biology and its applications in programming stem cells into mature, functional human cells for drug discovery and the creation of next generation. In the last 10 years,… Read More
Steve Wowk, VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. Some of the most important breakthroughs in pharmaceutical research were adapted from naturally occurring phenomena. The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its… Read More
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… Read More
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… Read More
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 “… Read More
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… Read More
Indee Labs and GenScript have formed a research collaboration with the aim to develop a DNA knock-in kit for Hydropore to enable large transgene inserts above the 5kb limit for AAV. As part of the collaboration, GenScript will provide a variety of single-strand and double-strand (ssDNA and… Read More
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… Read More
