CRISPR base editing has delivered a landmark n-of-1 gene therapy for an infant with carbamoyl phosphate synthetase 1 (CPS1) deficiency, in a clinical milestone that may redefine how rare genetic diseases are treated. CPS1 deficiency is a mitochondrial disorder caused by a loss-of-function mutation in the CPS1 gene that impairs the urea cycle and leads to life-threatening hyperammonaemia.
A digital twin lung simulation technology, Twinhale, has been used for the first time in idiopathic pulmonary fibrosis (IPF) in a groundbreaking new study. The study by Munich, Germany-based company Ebenbuild, supports the development of a novel inhaled anti-CCN2 (formerly called CTGF) biologic, PRS-220, developed by Pieris Pharmaceuticals - now Pavella Therapeutics Inc.
A study from the Weizmann Institute of Science in Israel has revealed an unexpected immune function of proteasomes, the cellular machinery primarily known for protein degradation. The research, published in Nature on 5 March, demonstrates that proteasomes play a direct role in innate immunity by generating antimicrobial peptides (AMPs) that combat bacterial infections. This discovery not only deepens our understanding of cellular defence mechanisms but also opens new avenues for addressing antibiotic resistance.
AMYRA Biotech AG, a Basel, Switzerland-based company working in digestive health innovation, has published a peer-reviewed literature review in Alimentary Pharmacology & Therapeutics supporting a novel approach to enzyme therapy for coeliac disease. The study underscores the critical role of the intestinal brush border membrane (BBM) in protein digestion and explores the potential benefits of supplementing exopeptidase activity in affected patients.
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.
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.
Professors Katalin Karikó and Drew Weissman have been awarded this year’s Nobel Prize in Physiology or Medicine for their work on nucleoside base modifications that enabled the development of vaccines against Covid-19. Their findings have fundamentally changed our understanding of how mRNA interacts with our immune system according to the Nobel Assembly, which awards the prize.
The Nobel Prize in Chemistry 2023 has been awarded to Moungi Bawendi, Louis Brus and Alexei Ekimov for the discovery and synthesis of quantum dots. Quantum dots are nanoparticles consisting of just a few thousand atoms whose size determines their properties. Their wide-ranging uses include television screens and computer monitors, LED lamps, as well as applications to illuminate tumour tissue in surgery.
The Danish government has appointed the BioInnovation Institute (BII) in Copenhagen as the site for a new project to support entrepreneurs involved in quantum science research. The initiative adds a new dimension to the institute, which was created in 2017 to advance research and enterprise in the life science industry. The institute was started by the Novo Nordisk Foundation, an independent charity which owns Novo Holdings A/S, the majority shareholder of Novo Nordisk A/S. The foundation provided grant funding for the BII until 2021 when it became financially independent.
The Food and Drugs Authority (FDA) of Ghana has approved a vaccine developed by the University of Oxford, UK, to prevent malaria – the first regulatory clearance for the product globally. The vaccine, R21/Matrix-M, is a subunit vaccine made up of a protein secreted by the malaria parasite and fused to a fragment of the hepatitis B virus. It is delivered alongside a plant-based adjuvant that enhances a person’s immune response. The adjuvant was developed by Novavax Inc of the US. The vaccine is being manufactured and scaled up by the Serum Institute of India PvT Ltd.