491833-30-8

Usage

Uses

Used in Gaucher's Disease Treatment:
Genz-123346 is used as a therapeutic agent for the treatment of Gaucher's disease type 1, a lysosomal storage disorder characterized by the accumulation of glycosphingolipids in various organs. It helps in managing the symptoms and improving the quality of life for patients with this condition.
Used in Niemann-Pick Type C Disease Research:
Genz-123346 is used as a potential treatment option for Niemann-Pick type C disease, another lysosomal storage disorder. It is currently under investigation for its efficacy in reducing the accumulation of glycosphingolipids in the brain and other organs, aiming to alleviate the symptoms associated with this condition.
Used in HIV Infection Management:
Genz-123346 is being explored as a potential therapeutic agent for the management of HIV infection. Its effects on glycosphingolipid metabolism may offer new avenues for the treatment of this viral infection, although more research is needed to establish its efficacy and safety in this context.
Used in Drug Delivery Systems:
Genz-123346 is used as an active pharmaceutical ingredient in the development of drug delivery systems, such as nanoparticles, to improve its bioavailability and therapeutic outcomes. These systems aim to enhance the delivery of Genz-123346 to target tissues and cells, potentially increasing its efficacy and reducing side effects.
Medically, Genz-123346 is known as Zavesca and is orally bioavailable. It is generally well tolerated, with possible side effects including diarrhea, flatulence, and weight loss.

Upstream product

• 112-05-0 nonanoic acid 
• 104943-23-9 nonanoic acid N-hydroxysuccinimide ester 
• 491833-28-4 (1R,2R)-2-amino-1-(2,3-dihydrobenzo[β][1,4]dioxin-6-yl)-3-pyrrolidin-1-yl-propan-1-ol 

Relevant academic research and scientific papers

Synthesis of UDP-glucose: N-acylsphingosine glucosyl transferase inhibitors

Disclosed is a novel enantiomeric synthesis ceramide-like inhibitors of UDP-glucose: N-acylsphingosine glucosyltransferase. Also disclosed are novel intermediates formed during the synthesis.

Synthesis and Evaluation of Hybrid Structures Composed of Two Glucosylceramide Synthase Inhibitors

Glucosylceramide metabolism and the enzymes involved have attracted significant interest in medicinal chemistry, because aberrations in the levels of glycolipids that are derived from glucosylceramide are causative in a range of human diseases including lysosomal storage disorders, type2 diabetes, and neurodegenerative diseases. Selective modulation of one of the glycoprocessing enzymes involved in glucosylceramide metabolism - glucosylceramide synthase (GCS), acid glucosylceramidase (GBA1), or neutral glucosylceramidase (GBA2) - is therefore an attractive research objective. In this study we took two established GCS inhibitors, one based on deoxynojirimycin and the other a ceramide analogue, and merged characteristic features to obtain hybrid compounds. The resulting 39-compound library does not contain new GCS inhibitors; however, a potent (200nm) GBA1 inhibitor was identified that has little activity toward GBA2 and might therefore serve as a lead for further biomedical development as a selective GBA1 modulator. Taking the best of both: Two established glucosylceramide synthase (GCS) inhibitors were merged via convergent synthesis to obtain hybrid compounds. Members of this 39-compound library have characteristics of both parent GCS inhibitors. No new GCS inhibitors were established, but a potent (200nm) acid glucosylceramidase (GBA1) inhibitor was identified. This adamantanemethyloxypenanoic acid pyrrolidene-substituted derivative of eliglustat can serve as a lead for further biomedical development of selective GBA1 modulators.