Friday, May 16, 2025

Neurodegenerative and Metabolic Disease Challenges and Solutions at ASGCT 2025

2025-05-15. ASGCT 2025. Neurodegenerative diseases like ALS, frontotemporal dementia, and Friedreich’s ataxia pose significant challenges with limited treatment options. 
Rgenta’s RSwitch technology offers precise control over FXN expression for Friedreich’s ataxia. It minimizes risks like cardiotoxicity through adjustable dosing via oral RDrugs.
This means that instead of a one-time, permanent change to the gene, RSwitch enables doctors to adjust the therapy’s effects in response to the patient’s needs. The gene therapy approach aims to correct the FXN deficiency, but unregulated FXN overexpression can lead to harmful side effects, including cardiotoxicity (damage to the heart).