Your missing links are here (25 September 2020)

Top Picks

• CTX001, an autologous CRISPR-engineered haematopoietic stem cell therapy under co-development by CRISPR Therapeutics and Vertex Pharmaceuticals is granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD).
• US researchers use CRISPR to knock out the cystic fibrosis transmembrane conductance regulator (CFTR) gene in human macrophages in order to understand the macrophage defects that are observed in cystic fibrosis. Findings suggest that many aspects of CF macrophage dysfunction are CFTR-dependent and not just reflective of the CF inflammatory milieu in general.
• Tuneable, Biodegradable, Lipid-Like Nanoparticles for In Vivo mRNA Delivery. Read about it in our interview with Yizhou Dong from Ohio State University.

Research

• In a new study published in Nature, US-based researchers describe how they used genome-wide CRISPR screens to identify genes that allow cancer cells to evade destruction by the host cytotoxic T cell responses.
• A new assay can identify CRISPR-Cas9 nickase induced single-strand breaksin vitro and thereby be used to screen for genome-wide off-target sites of prime editors.
• Targeted integration of template DNA after CRISPR genome editing has proved difficult in post-mitotic cardiomyocytes, but Japanese scientists have now shown that this can be achieved by intra-ventricular injection of AAV expressing the gRNA and template DNA.
• Scientists in Korea have fused Cas9 to a cell-penetrating protein from silkworm and demonstrated that this could successfully deliver the CRISPR tool to human cells.
• Ultrasensitive CRISPR protocol based on SHERLOCK platform for field-applicable detection of Plasmodium species in symptomatic and asymptomatic malaria.

Clinical Trials

• Great Ormond Street Hospital for Children NHS Foundation Trust in London are intiitating a new Phase I trial for B-cell Acute Lymphoblastic Leukaemia using CRISPR-engineered healthy donor T cells.
• Children's Hospital of Fudan University, China plans to conduct prospective study to assess new CRISPR-based platform to detect pathogenic Bordetella (bacteria) and drug resistance genes in a single step.

Industry

• Precision BioSciences and Servier Expand CAR T Oncology Development Collaboration with Four New Programmess Targeting Haematological and Solid Tumours.
• Dublin-based ERS Genomics announces agreement with Applied Stemcell, California to commercialise CRISPR gene editing services and reagents.
• New next-gen gene editing companyGraphite Biolaunches in California with $45 Million Series A Financing. The company is founded by pioneers in the gene editing field and already has a solid pipeline focused on targeted DNA integration to precisely insert genetic payloads to treat a number of severe diseases.

COVID-19

• The Drug Controller General of India has approved the country’s first CRISPR-based COVID-19 test for commercial launch. The test, which is commercially known as FELUDA, is commercialised by TATA Sons (India) and is so far the world’s first Cas9-based test for SARS-CoV-2.
• Researchers at University of Connecticut validate clinical feasibility of CRISPR-Cas12a based platform for COVID-19 testing in clinical swab samples. Results published in Nature Communications.

Conferences and Webinars

• Labroots to host its 3^rd Annual CRISPR Virtual Event to include discussions about current CRISPR-based editing, next-generation gene editing tools, precision, and the role of CRISPR in drug discovery and development.