Alloy Therapeutics Inc. has entered into a collaboration and license agreement with Biogen Inc. for the use of Alloy’s Anticlastic ASO platform to accelerate the development of innovative oligonucleotide therapeutics.
Werner syndrome results from biallelic mutations in the WRN gene on chromosome 8, leading to accelerated aging symptoms. Researchers at Sumitomo Pharma Co. Ltd. have reported the development and characterization of WRN-108, a splice-switching antisense oligonucleotide (ASO) designed to induce exon 27 skipping in WRN transcripts carrying the c.3139-1G>C mutation.
Acurastem Inc. has received a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function. TDP-43 dysfunction is a central biological hallmark of amyotrophic lateral sclerosis (ALS).
Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the Freedom2-DM1 phase II trial, a multiple ascending-dose, randomized, placebo-controlled experiment in myotonic dystrophy type 1 (DM1).
A therapeutic strategy based on alternative splicing of the MECP2 gene could restore protein levels in Rett syndrome, a neurological disorder caused by mutations in that gene. Scientists at Baylor College of Medicine have successfully tested this approach both in vitro in neurons from Rett patients that produce some functional protein, correcting the altered gene expression and improving neuronal functions, and in vivo in mice.
Researchers from the University of Oxford and the Health Research Institute La Fe (Spain) investigated the potential of multigene RNA-based therapeutics in Alzheimer’s disease, aiming to overcome potential compensatory mechanisms and patient heterogeneity.
Metabolic dysfunction-associated steatotic liver disease (MASLD) covers a series of pathogenic conditions including steatosis, inflammation and fibrosis with limited therapeutic options to date. Recent findings have shown upregulation of hepatic murine double minute 2 (MDM2) in patients with MASLD. Additionally, genetic deletion of hepatic MDM2 and its pharmacological inhibition were seen to improve steatosis and fibrosis in MASLD murine models.
Dentatorubral-pallidoluysian atrophy (DRPLA) is a lethal neurodegenerative disorder caused by pathogenic expansion of CAG repeats within the atrophin-1 (ATN1) gene. As DRPLA belongs to the broader class of repeat expansion disorders (RED) that are driven by toxic gain-of-function effects, reduction or elimination of ATN1 expression is predicted to provide therapeutic benefit.
Stoke Therapeutics Inc.’s speeded-up timeline for zorevunersen, the antisense oligonucleotide in development with Biogen Inc. as a first-in-class potential disease-modifying treatment for Dravet syndrome, put the rare, severe form of lifelong epilepsy in the spotlight. The news involved completion of enrollment and a phase III data readout from the Emperor study, as officials said signups of 150 patients are expected in the second quarter of the year, which puts the study on track for data in mid-2027.
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Researchers from Sorbonne University and Hôpital Trousseau developed a targeted approach based on antisense oligonucleotides as a way to improve CF management.
