Azafaros Receives IND Clearance and FTD from the US FDA for AZ-3102 to Treat Lysosomal Storage Disorders with Neurological Involvement
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
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Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.
