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.
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).
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).
Arnatar Therapeutics Inc.’s ART-4, an upregulating antisense oligonucleotide (ASO) therapeutic candidate for the treatment of Alagille syndrome (ALGS), has been awarded U.S. orphan drug and rare pediatric disease designations by the FDA. Approximately 95% of ALGS cases are caused by haploinsufficient mutations in the Jagged-1 (JAG1) gene, leading to insufficient JAG1 protein levels and impaired liver bile duct development.
Down syndrome (DS) is the most prevalent genetic cause of Alzheimer’s disease (AD). Previous evidence suggests that increased dosage of the amyloid precursor protein (APP) gene plays a crucial role in AD in individuals with Down syndrome (DS-AD), making APP expression a crucial therapeutic target.
The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).
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