Your missing links are here (6 November 2020)
By: Karen O'Hanlon Cohrt - Nov. 6, 2020
- The European Patent Office has today revoked a patent related to CRISPR gene editing. The revocation was based on a lack of novelty due to an invalid claim to priority. The patent had been held by the Broad Institute and others, and the revocation marks yet another event in the long-standing CRISPR patent battle, which was recently covered here.
- How can gene editing help beat COVID-19? Get up to speed on how CRISPR can help to solve the COVID-19 pandemic in this discussion piece that looks at diagnostic and therapeutic CRISPR approaches to COVID-19.
- Researchers from the Institute of Molecular Biotechnology of the Austrian Academy of Sciences have developed CRISPR-LICHT, a revolutionary technology that allows genetic screens in human tissues such as brain organoids. So far, the technology has revealed a major role for the ER-stress pathway in regulating human brain size. The results were published last week in Science. Two of the researhers behind the work explain the technology in a YouTube presentation.
- NKX3.1 is a gatekeeping tumour suppressor gene and is the most frequently deleted gene in prostate cancer. Using CRISPR/Cas9, researchers have now identified a point mutation in Nkx3.1 that prolongs Nkx3.1 protein half-life and their findings suggest that modulation of Nkx3.1 may exert long-term control over premalignant changes and susceptibility to DNA damage in the prostate.
- PpCas9 is a compact Type II-C Cas9 from Pasteurella pneumotropica that has found to be active in human cells. PpCas9 has a novel PAM requirement compared to the prototypical SpCas9, which may broaden the range of Cas9 applications within biomedicine and biotechnology.
- Editas Medicine announces third quarter 2020 results and program updates. Notably, the company has completed dosing of the first cohort with EDIT-101, the first in vivo CRISPR therapy in the landmark BRILLIANCE trial for Leber congenital amaurosis 10. It’s EDIT-301 therapy for sickle cell disease is also moving forward with IND filing expected for Quarter 4.
- Intellia Therapeutics also announces third quarter 2020 results. The company is developing several CRISPR-based therapeutic programmes within cancer and genetic diseases. Following regulatory approval for a Phase 1 trial, the company is on track to dose its first transthyretin amyloidosis (ATTR) patient with NTLA-2001 by the end of the year. This would be the first clinical trial of a systemically delivered CRISPR/Cas9-based therapy, marking a major milestone for the genome editing field.
- Share for CRISPR Therapeutics, Bluebird Bio and Global Blood Therapeutics saw a boost of about 10 %, 7 %, and 15 % respectively following the sharing of results about developmental treatments for sickle cell anaemia at the 62nd Annual American Society of Hematology meeting this week.
Vision and Opinion
- Note: Danish only. CRISPR Medicine News Journalist Dr. Gorm Palmgren and Danish Professor in Biomedicine and member of Danish Ethics Council Jacob Giehm Mikkelsen took part in a discussion on Denmark’s national radio about CRISPR, its discovery and applications and how it ended up winning the 2020 Nobel Prize in Chemistry. Listen in here.
- Book Review. Editing Humanity: The Crispr Revolution and the New Era of Genome Editing Kevin Davies Pegasus (2020). Review by Natalie Kofler for Nature.
- Literature review covering the therapeutic potential of CRISPR-Cas13 for COVID-19.
- We’ve heard much about SHERLOCK and DETECTR for COVID-19 diagnosis. This timely review compares the two competing technologies, their properties, functions and potential to become the ultimate diagnostic tools in outbreak situations.
- Earlier this week at the 62nd annual American Society of Hematology meeting, CRISPR Therapeutics and Vertex Pharmaceuticals announced promising efficacy data concerning seven patients from two ongoing Phase 1/2 clinical trials for the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe haemoglobinopathies. Read the conference abstract here.
- Taiwan’s Ministry of Science and Technology (MOST) brings together international institutions, including Taipei Veterans General Hospital (TVGH), National Yang-Ming University (NYMU), and the University of California, Los Angeles (UCLA) with the goal to advance CRISPR-Cas gene editing technology to clinical stage for sickle cell anaemia and beta-thalassemia.
- New COVID-19 test under development in Saudi Arabia. iSCAN is a RT-LAMP-coupled CRISPR-Cas12 module capable of rapid, sensitive detection of SARS-CoV-2. The one-pot assay gives results in less than an hour and is compatible with lateral flow detection methods. Original research article published in Virus Research.
- Researchers from Singapore and the US join forces to develop a new quantitative diagnostic workflow based on digital PCR combined with the search function of Cas12a. RApid DIgital Crispr Approach (RADICA) is a one-pot chip-based assay that delivers precise results within 1 hour, and has been validated with synthetic nucleic acids from the SARs-CoV-2 and Epstein Barr Virus genomes. The research results have been shared on the preprint server medRxiv.
- Yet another CRISPR-based test for COVID-19, this time from researchers at Stanford University, US. The new test deploys electric fields to extract and purify nucleic acids from swab samples, and any viral RNA present is converted into DNA via isothermal amplification. Cas12a is used to detect amplified viral RNA. The entire process is cataylsed by the use of electric fields that concentrate all of the assays components into a tiny space that dramatically increasing their likeleood of interacting. The platform can detect COVID-19 in just 30 minutes. Research findings published in PNAS.