Camp4 Therapeutics Corp. has highlighted new preclinical data for CMP-002, the company’s lead investigational antisense oligonucleotide (ASO) therapeutic candidate for SYNGAP1-related disorder. CMP-002 administration resulted in a statistically significant improvement in seizure phenotypes and parameters in a SYNGAP1 haploinsufficient mouse model.

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.

Vivatides Therapeutics Inc. has closed an oversubscribed $54 million series A financing to advance its work in extrahepatic RNA delivery technologies. Proceeds from the financing will be used to further advance the company’s extrahepatic delivery platform and accelerate pipeline programs.

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).