Researchers from McGill University and collaborating institutions aimed to investigate whether oligonucleotides are a viable drug class to prevent hydrocephalus.

Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.

Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.

Acurastem Inc. has been awarded $7.5 million in grant funding by the California Institute for Regenerative Medicine (CIRM) to support the advancement of lead clinical candidate AS-241 toward first-in-human testing.

Vanda Pharmaceuticals Inc. presented data this week at the annual American Academy of Neurology conference regarding allele-specific antisense oligonucleotides (ASOs) that specifically target the mutant p.A53T allele from the SNCA gene while preserving the expression of the wild-type allele. The mutant allele is associated with increased risk of early-onset Parkinson’s disease (PD) and current ASOs target SNCA regardless of its mutation status.

Quiver Bioscience Inc. has received a strategic investment from the Porta family office (Argentina) to advance the company’s antisense oligonucleotide (ASO) therapeutic program for chromosome 15q duplication (Dup15q) syndrome. The investment will fund preclinical activities, including safety studies for candidate selection as the program advances toward clinical development.

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.

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).

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.