EN ZH NEWS NEWARK, CA • MAY 17, 2023 AceLink Therapeutics to Present at World Orphan Drug Congress USA (BUSINESS WIRE)–AceLink Therapeutics, Inc., a clinical stage biopharmaceutical company developing the next generation of oral substrate reduction therapies (SRTs) to address significant unmet medical needs and improve the quality of life of patients with inherited disorders …
Monthly Archives: May 2023
AL1311
EN ZH PIPELINE AL1311 is a highly potent NRF2 activator that is being developed for Autosomal Dominant Polycystic Kidney disease (ADPKD). AL1311 is different from other electrophilic and reactive NRF2 activators because it works by disrupting the protein-protein interaction between NRF2 and its regulatory protein Keap1. Non-reactive NRF2 activators have the potential to have better specificity …
AL0804
EN ZH PIPELINE AL0804 is a highly brain penetrant GCS inhibitor being developed for the treatment of neuronopathic glycolipid storage diseases including Gaucher disease (Type II and III), GM2 gangliosidosis (Tay-Sachs and Sandhoff disease), GM1 gangliosidosis, and certain genetic forms of Parkinson’s disease. AL0804 has superior potency and brain penetration compared to other brain penetrant GCS …
AL1211
EN ZH PIPELINE AL1211 is a glucosylceramide synthase (GCS) inhibitor with unique and superior properties to other GCS inhibitors currently approved or in development. AL1211 is more potent and has a better off-target activity profile than other GCS inhibitors. AL1211 also has excellent penetration into the tissues that are affected in Fabry disease like heart and …
GBA PARKINSON’S DISEASE
EN ZH SCIENCE GBA PARKINSON’S DISEASE GBA Parkinson’s disease is arguably the most common genetic form of Parkinson’s disease. Numerous genetic studies have shown that a single mutation in the GBA1 gene (the gene that causes Gaucher disease) can increase your risk of developing Parkinsons’ disease by up to 30-fold. In vitro and in vivo preclinical studies suggest that …
DIABETIC NEPHROPATHY (DN)
EN ZH SCIENCE DIABETIC NEPHROPATHY (DN) Diabetic nephropathy (DN) is a common form of kidney damage that occurs with the progression of metabolic diseases like type I and type II diabetes. If diabetes is not properly treated then damage can occur to the glomeruli, the filtration units of the kidney. This damage can lead to …
POLYCYSTIC KIDNEY DISEASE
EN ZH SCIENCE POLYCYSTIC KIDNEY DISEASE Polycystic kidney disease (PKD) is a genetic disorder in numerous cysts develop primarily within the kidneys, causing the kidneys to enlarge and eventually lose function. There are two main subtypes of PKD: Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease, characterized by the development of …
GANGLIOSIDOSES
EN ZH SCIENCE GANGLIOSIDOSES GM1 Gangliosidosis is a lysosomal storage disorder caused by a mutation in the GLB1 gene. Mutation in GLB1 lead to reduced activity of the lysosomal enzyme beta-galactosidase (bGal). In GM1 gangliosidosis patients, bGal deficiency results in the accumulation of GM1 gangliosides (GM1). GM1 gangliosidosis patients with the most severe bGal deficiency develop severe early onset …
GAUCHER DISEASE
EN ZH SCIENCE GAUCHER DISEASE Gaucher Disease is a lysosomal storage disorder caused by a mutation in the GBA1 gene. Mutation in GBA1 lead to reduced activity of the lysosomal enzyme glucocerebrosidase (GCase). In Gaucher patients, GCAse deficiency results in the accumulation of glucosylceramide glucosylceramide (GL1). Depending on the severity of the GCase deficiency, Gaucher patients can develop severe …
FABRY DISEASE
EN ZH SCIENCE FABRY DISEASE Fabry disease is a lysosomal storage disorder caused by mutations in the GLA gene, leading to reduced activity of the enzyme a-galactosidase A (GalA). In Fabry disease patients, GalA deficiency results in the accumulation of globotriaosylceramide (GL3), leading to a wide range of symptoms from pain, gastrointestinal issues, eye and skin problems to …