Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes loss of vision. Pathogenic variants in proteins involved in RNA splicing are the second most common cause of autosomal dominant RP, with mutations in PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP.
Currently available treatments for chronic hepatitis D virus (HDV) infection rarely result in a cure after a defined treatment period. Researchers from Aligos Therapeutics Inc. hypothesized that antisense oligonucleotides (ASOs) targeting HDV RNAs may inhibit intracellular HDV RNA amplification.
GSK plc has announced a breakthrough in the treatment of chronic hepatitis B, reporting a functional cure rate of 19% across two phase III trials of its antisense oligonucleotide bepirovirsen.
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.
Quiver Bioscience Inc. has received a multi-year grant from the National Institutes of Health (NIH) to advance its lead Nav1.7-targeted antisense oligonucleotide (ASO), QV-2421, through IND-enabling studies and first-in-human trials for chronic neuropathic pain.
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.
