The Nobel Prize-winning modification that prevents the innate immune system from recognizing injected mRNA as foreign and blocking transcription of the protein it encodes has been found on some occasions to cause ribosomal frameshifting.
After five years and 350,000 hours of DNA sequencing, the UK Biobank has opened up access Nov. 30 to the whole genome sequences of half a million people who volunteered to give samples 15 years ago.
Cancer Research UK (CRUK) is worried that rising prices and its total dependence on public donations mean its funding model is becoming unsustainable. The charity is calling on the U.K. government to step in and plug a £1 billion (US$1.23 billion) shortfall it said will open up over the next decade, in order to maintain investment at 2019 levels in real terms.
Freeline Therapeutics plc is being taken private by its founding investor and majority shareholder Syncona Ltd, in an all-cash transaction that values the Nasdaq-listed gene therapy specialist at $28.3 million. Syncona also will provide up to $15 million cash to keep Freeline afloat as it awaits the next tranche of data on its lead program FLT-201, which is positioned as a one-off treatment for the lysosomal storage disorder, Gaucher’s disease.
Bioxodes SA has set the stage for the phase IIa study of its novel anticoagulant in the treatment of intracerebral hemorrhage (ICH), after raising a €12 million (US$13 million) series A. Days before announcing the closure of the round, the first patient in the proof-of-concept study was treated, on Nov. 17, and eight of 10 sites across Belgium are geared up to take part in the trial. The aim is to develop the product, Ir-CPI, as the first injectable antithrombotic that is suitable for use within the first 72 hours of an ICH.
The long-running row over the U.K. voluntary scheme that controls the national drugs budget has been settled, in what the industry is describing as a “tough deal.” Under the scheme there is an annual cap on total sales of branded drugs to the National Health Service, with sales over the agreed limit reimbursed via a levy. In 2022, pharma companies paid back £2 billion (US$2.5 billion) in rebates on total sales of £14 billion. In the new five-year agreement, the allowed annual increase in sales will be 2% in 2024, the same as across the current scheme from 2019 – 2023, but it will then increase to 4% by 2027.
Newco T-Therapeutics Ltd. has raised £48 million (US$59 million) in a series A to advance development of T-cell receptors generated by its transgenic mouse platform for the treatment of solid tumors, autoimmune diseases and infections. In cancer, the specificity of T-Therapeutics’ molecules will overcome shortcomings of immuno-oncology drugs such as checkpoint inhibitors that stimulate a response to some cancer neo-antigens but are unable to recognize cancer-specific self-antigens.
Newco T-Therapeutics Ltd. has raised £48 million (US$59 million) in a series A to advance development of T-cell receptors generated by its transgenic mouse platform for the treatment of solid tumors, autoimmune diseases and infections.
Researchers have demonstrated that inhibiting mitophagy in ‘old’ hematopoietic stem cells (HSCs) completely restored their blood reconstitution capabilities, raising the prospect of addressing the age-related weakening of the immune system that stems from HSCs deteriorating over time.
The German med-tech associations Bvmed and VDGH have hit out at the shortcomings of the EU medical device regulations and are calling for action to improve implementation, predictability and transparency of the system.
