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.
Cystic fibrosis (CF) is a genetic disorder affecting around 90,000 people worldwide. It is commonly caused by the ΔF508 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which results in a misfolded CFTR protein that is subsequently ubiquitinated and degraded.
Aniridia-associated keratopathy (AAK) is a rare genetic eye condition caused by PAX6 haploinsufficiency, which leads to chronic inflammation, neovascularization and vision loss. Currently, there are limited therapeutic options for the treatment of AAK, which has been linked to limbal stem cell deficiency.
As the first – and so far only – drug to enter clinical testing for the rare neurogenetic disorder Alexander disease, there were some unknowns heading into the readout of the pivotal study testing Ionis Pharmaceuticals Inc.’s zilganersen in children and adults. But the top-line data yielded a clear win for the antisense oligonucleotide candidate, which demonstrated a disease-modifying impact, including statistical significance on the primary endpoint of gait speed as assessed by the 10-Meter Walk Test vs. control at week 61.
Hearing loss is a major global health challenge, affecting over 5% of the population and largely lacking effective biological therapies. Mutations in KCNQ4, which encodes the Kv7.4 potassium channel essential for outer hair cell function, are a leading cause of autosomal dominant, non-syndromic hearing loss (DFNA2).
Acute pancreatitis took center stage as Wall Street took heed of phase III data from Ionis Pharmaceuticals Inc. with olezarsen for severe hypertriglyceridemia (sHTG), while another player in the space, Arrowhead Pharmaceuticals Inc., signed a sizeable deal in a separate therapeutic area with Novartis AG.
The FDA has granted orphan drug designation to Vanda Pharmaceuticals Inc.’s VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV).
A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome.
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