Editas Medicineannounces 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 Therapeuticsalso 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.
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.
Review
Book Review. Editing Humanity: The Crispr Revolution and the New Era of Genome Editing Kevin Davies Pegasus (2020). Review by Natalie Kofler for Nature.
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.
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.