RNA

Now a key pillar in the early stages of therapeutics development, the limitations of introducing a DNA double strand break mean that workflows are broadening away from CRISPR–Cas as a gene knockout technology and towards platforms that enable genetic modulation and safer genetic alterations. Nicola McCarthy, Horizon Discovery, explains. Navigating from target identification to validation […]

Lisa Osborne, CEO at RANA Healthcare Solutions interviews Daniel Kavanagh, PhD, Senior Scientific Advisor, Gene Therapy at WCG. The Covid-19 pandemic has put vaccine development centre-stage. Never have citizens around the world been so focused on the length of time it takes to develop, test, approve, manufacture and distribute new vaccines. This heightened interest is […]

Michael Smyth is Medical Director at Galapagos UK, responsible for launching innovative therapies in the UK to help change the course of inflammatory disease for patients. Having studied medicine at Liverpool University, one of his personal missions is lifelong learning which is reflected in his role to strive for new treatment options that lead to better […]

Two weeks ago, the Moderna mRNA vaccine became the third vaccine to be approved for emergency use in the UK. With 94% effectiveness, this vaccine is an exciting addition to the COVID-19 relief effort and brings us one step closer to the end of the pandemic. Before 2020, efforts to study mRNA vaccines weren’t at the forefront of […]

Targeting RNA is set to be the next big thing; 85% of human genome transcribed to RNA but only 3% translated into proteins, therefore making it an obvious target. With Risdiplam being approved by the FDA in August, and several major collaborations also being penned throughout 2020, including a $190 million deal between Arrakis and […]

The new CPhI report looks ahead to a post-pandemic 2021 with pharma manufacturing capacity and payment structures shifting as new sourcing strategies emerge. It explores the potential implications in 2021 of impending vaccine approvals, a new administration in the White House and a return to dual supply chain strategies. The report – which summarises expert analysis […]

None of the mutations currently documented in the SARS-CoV-2 virus appear to increase its transmissibility in humans, according to a study led by UCL researchers. The analysis of virus genomes from over 46,000 people with COVID-19 from 99 countries is published in Nature Communications. First and corresponding author Dr Lucy van Dorp (UCL Genetics Institute) […]

PerkinElmer will acquire Horizon Discovery for approximately $383 million (£296 million). The transaction is expected to close in the first quarter of 2021 subject to conditions. The investment will allow PerkinElmer to expand its portfolio of leading, automated life sciences discovery and applied genomics solutions to include gene editing and gene modulation tools. The acquisition […]

By Ibis Sanchez-Serrano and Rathnam Chaguturu Mitigation of COVID-19 pandemic requires a combination of effective epidemiological policies and the discovery/development of powerful drugs against the Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) that causes this disease. This article examines the major initiatives being explored at the public policy level to contain the spread of COVID-19 […]

By Dr Jamie Freeman Base editing is a potentially transformative technology for engineering DNA in cells that has the potential to generate knockouts or correct certain errors or mutations in the DNA for applications such as cell or gene therapies. Compared with currently-available gene editing methodologies such as CRISPR/Cas9, which creates ‘cuts’ in the gene […]