News

This year’s Nobel Prize in Chemistry has been awarded to David Baker, Demis Hassabis, and John Jumper for their work in the field of protein science. Dr Baker, a professor at the University of Washington, US, has been recognised for his pioneering research in computational protein design. Drs Hassabis and Jumper, both from Google DeepMind, are credited with developing AlphaFold2, an artificial intelligence model that predicts the structure of proteins with great accuracy.

Purespring Therapeutics Ltd, a gene therapy company developing treatments for kidney diseases, has raised £80 million from private investors to bring its lead product into clinical development. This is the latest in a series of transactions among biotech companies in the kidney disease sector but the first for a preclinical gene therapy. Purespring’s product candidate, PS-002, is an adeno-associated viral vector with a gene payload for the treatment of IgA nephropathy (IgAN), a chronic kidney disease primarily affecting young adults. 

A Phase 2 trial of a small molecule drug for patients with mild dementia due to Alzheimer’s disease has been stopped due to lack of efficacy, the developer Sage Therapeutics Inc announced on 8 October. The drug, dalzanemdor (SAGE-718), did not show a statistically significant difference in patients with the disorder, compared with a placebo, on the basis of a widely-used intelligence test. 

Victor Ambros and Gary Ruvkun have been awarded this year’s Nobel Prize in Physiology or Medicine for their discovery of microRNA and its role in post-transcriptional gene regulation. Their work has revealed an entirely new mechanism that controls gene expression, transforming our understanding of how cells regulate the production of proteins essential for their function and development.

A consortium of universities and hospitals, with financial support from the UK government and industry, is to undertake a four-year project to collect data on patients who have received immunotherapy for cancer to establish the benefits and risks of these treatments. The treatments, which include checkpoint inhibitors, have extended the lives of many patients by enabling the body’s immune system to recognise and destroy cancer cells.

AstraZeneca Plc is to pay $100 million upfront and up to $1.92 billion in milestone payments for rights to a pre-clinical lipid-lowering therapy from China. The developer, CSPC Pharmaceutical Group Ltd, is listed on the Hong Kong Stock Exchange and has R&D facilities in both China and the US. The asset being acquired is a small molecule lipoprotein inhibitor which is in pre-clinical development for patients suffering from dyslipidaemia, a disorder involving abnormal levels of lipids in the bloodstream. Dyslipidaemia is a risk factor for cardiovascular diseases.

An Italian biotech company, Aptadir Therapeutic Srl, which is developing a new class of RNA therapies, launched on 24 September with pre-seed funding of $1.6 million and plans to tackle cancer by blocking aberrant DNA methylation. DNA methylation is an essential biological process that enables the expression of genes in humans. But this process can be thrown off balance by external factors such as age or diets, leading to cancer and genetic disorders.

Novo Nordisk A/S is to pay an undisclosed amount of money to Evotec SE to gain access to the German company’s cell therapy technology and manufacturing capacity for its experimental therapies in a number of indications including diabetes. The collaboration, announced on 26 September, is an expansion of the Danish company’s platform technologies which are currently dominated by insulins and peptide therapies for obesity. Novo currently has cell therapy programmes under investigation for chronic heart failure, Parkinson’s disease and Type 1 diabetes.

Enara Bio Ltd, a UK biotech, has raised $32.5 million in Series B financing to build a pipeline of therapies for cancer that target peptide antigens in the dark genome. The financing round was co-led by the new investors Pfizer Ventures and M Ventures whose executives, respectively, Rana Al-Hallaq and Bauke Anninga, have joined the company’s board of directors.

A preclinical project designed to treat Huntington’s disease has received £35 million in Series A financing on early evidence that it can slow or even halt progression of the disease. Huntington’s is an inherited disorder that attacks areas of the brain that help control movement. The funding was awarded to LoQus23 Therapeutics Ltd, a biotech company based in Cambridge UK. The venture capital group Forbion led the round, alongside existing investors SV Health Investors’ Dementia Discovery Fund and the Novartis Venture Fund.