Shots:
The company received an IND clearance along with an FTD from the US FDA for AZ-3102 to treat GM1/GM2 gangliosidoses & NP-C
The P-II clinical trial (RAINBOW) evaluates the safety, tolerability, PK & PD of AZ-3102 in GM2 & NP-C patients aged 12 to 20yrs. for a duration of 12wks. across the world. The study will also assess the impact AZ-3102 on potential disease biomarkers
AZ-3102 is a brain-penetrant azasugar that is developed with a dual mode of action inhibiting two key enzymes which modulate the metabolism of glycosphingolipids
Ref: Businesswire | Image: Azafaros
Related News:- Takeda Enters into a License Agreement with Selecta to Develop Gene Therapies for the Treatment of Lysosomal Storage Disorders
