A tangle of DNA can look like a knotted ball in the cell nucleus. However, the genetic machinery has a complex and regulated structure. Its long repetitive sequences also seemed to have no function. They were called junk DNA, although they were not. The same happened with proteins and low-complexity domains, disordered chains of amino acids that were poorly understood. Nevertheless, that protein noise has turned into music for the 2025 Lasker Awards. These prizes have recognized the work of scientists who were able to see order in chaos.

The Cystic Fibrosis Foundation has agreed to provide Prime Medicine Inc. an additional investment of up to $24 million to continue the development of a gene editing therapy for people with cystic fibrosis. Prime Medicine uses a gene editing technology called prime editing, which enables a wide range of modifications to the DNA with a high degree of precision.

Launched out of 5AM Ventures’ 4:59 Initiative, Renasant Bio Inc. has secured $54.5 million in seed funding to support development of treatments for autosomal dominant polycystic kidney disease (ADPKD), a genetic disease caused by mutations in the PKD1 and PKD2 genes, which encode polycystin proteins PC1 and PC2 that come together to form a calcium ion channel vital for kidney function.

Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator (CFTR) that mediates Cl and HCO3 anion transport.

The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer.

The map of cystic fibrosis (CF) research is being redrawn in the U.K. as improvements in treatment, and in particular the introduction of CF modulator drugs, mean people with the rare inherited disease are living much longer.

The longstanding ambition of developing an inhaled gene therapy for cystic fibrosis has taken a step forward, with the start of a phase I/II trial of a product using a novel pseudotyped viral vector that it is hoped will circumvent problems encountered in previous studies with other vectors.

Glox Therapeutics Ltd. has been awarded a share of a £3 million (US$3.7 million) collaborative discovery program launched by the Cystic Fibrosis Antimicrobial Resistance (CF AMR) syndicate. The syndicate is a cross-sector initiative driven by Medicines Discovery Catapult, Lifearc and Cystic Fibrosis Trust.