全球首款！神济昌华渐冻症基因疗法SNUG01获FDA孤儿药认定

Company Overview - SineuGene, established at the end of 2021, focuses on gene therapy for neurological diseases, leveraging over a decade of foundational research from Professor Jia Yichang's laboratory at Tsinghua University [6][7] - The company has developed various animal disease models, including fruit flies, mice, and Bama pigs, to identify reliable drug targets [7] - SineuGene has established a platform for AAV screening targeting the central nervous system, aiming to create highly targeted, efficient, and low-immunogenic AAV serotypes [7] Product Development - SNUG01, the first-in-class gene therapy product developed by SineuGene, targets TRIM72 and is the first gene therapy product globally to do so [4] - The therapy utilizes recombinant adeno-associated virus type 9 (rAAV9) for precise delivery of the human TRIM72 gene to neurons via intrathecal injection [4] - Preclinical studies indicate that TRIM72 may protect neurons through multiple mechanisms, potentially delaying the degenerative progression of motor neurons in ALS patients [4] Regulatory Milestones - SNUG01 received Orphan Drug Designation (ODD) from the FDA for the treatment of amyotrophic lateral sclerosis (ALS), which is a significant milestone following its clinical trial approval [3][4] - The ODD status provides several benefits, including a 25% tax credit on clinical research costs, exemption from up to $3 million in Biologics License Application fees, and 7 years of market exclusivity post-approval [3] - This designation is expected to accelerate the development and market entry of SNUG01, ultimately benefiting ALS patients in need of effective treatments [3] Clinical Research - The upcoming I/IIa phase international multicenter registration clinical trial aims to assess the safety, tolerability, and preliminary efficacy of SNUG01 in adult ALS patients [3][4] - The trial is designed to ensure that patients in both China and the U.S. can benefit from the treatment simultaneously [3] Disease Context - ALS is a progressive and fatal neurodegenerative disease affecting both upper and lower motor neurons, leading to muscle weakness and atrophy, with a median survival of only 3-5 years [5] - Currently, there are no curative treatments available, and existing therapies only provide limited disease progression delay [5]