Alagille syndrome (ALGS) is a rare, multisystem genetic disorder most commonly caused by haploinsufficiency of the JAG1 gene, leading to reduced JAG1 protein function and impaired development of intrahepatic bile ducts. Researchers from Arnatar Therapeutics Inc. described the development of antisense oligonucleotides (ASOs) engineered using their proprietary ACT‑UP1 platform to upregulate endogenous JAG1 expression and thereby address the underlying genetic deficiency.

Scineuro Pharmaceuticals Holdings Ltd. has received a $5 million research grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to accelerate the preclinical development of the company’s novel LRRK2-targeted antisense oligonucleotide (ASO) program, SNP-614, for Parkinson’s disease.

Aperture Therapeutics Inc. has nominated APRTX-001 as a development candidate, with the program now advancing through IND-enabling studies. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of frontotemporal dementia and amyotrophic lateral sclerosis, with potential for indication expansion into Alzheimer’s disease.