Alberto Auricchio

Alberto Auricchio, M.D., is Scientific Director of the Telethon Institute of Genetics and Medicine (TIGEM) in Pozzuoli, Italy (appointed April 2024), Professor of Medical Genetics at the University of Naples "Federico II," and President of the European Society of Gene & Cell Therapy (ESGCT, elected October 2024). He is Founder and Chief Scientific Officer of AAVantgarde, a biotechnology company developing gene therapies for retinal diseases. Dr. Auricchio is internationally recognized as a European leader in AAV gene therapy for inherited retinal diseases. His research focuses on developing gene therapy approaches for retinal and metabolic diseases using adeno-associated viral vectors. He contributed significantly to the clinical development of Luxturna (voretigene neparvovec), the first FDA-approved gene therapy for humans (2017). Auricchio's team conducted the Italian portion of the RPE65 gene therapy trials in collaboration with Professor Francesca Simonelli at Naples, enrolling five Italian patients with Leber congenital amaurosis. His 2009 Lancet publication demonstrated age-dependent treatment effects, providing critical evidence supporting early pediatric intervention for optimal therapeutic benefit. Auricchio pioneered dual AAV vector systems for large gene delivery, addressing the major technical challenge of AAV packaging capacity limitations (~4.7kb). His laboratory's 2023 Molecular Therapy publication demonstrated that dual AAV8 vectors encoding human MYO7A improved retinal defects in mouse models of Usher syndrome type 1B and established safety in primates. This technology platform extends to Stargardt disease (ABCA4 gene, 7kb) and other large-gene retinal disorders, forming the scientific foundation for AAVantgarde's clinical pipeline. In April 2024, following a global search by an international expert committee, Auricchio succeeded Andrea Ballabio as TIGEM Scientific Director after Ballabio's 30-year tenure. As ESGCT President, he shapes European gene therapy policy and research directions. Auricchio has authored over 150 peer-reviewed publications and holds multiple international patents on AAV gene therapy applications. He received the American Society of Gene & Cell Therapy Outstanding New Investigator Award and the International Prize for Scientific Research "Arrigo Recordati." His ongoing clinical work includes leading a phase I/II trial for mucopolysaccharidosis type VI, scheduled to resume at higher doses in 2026.

Alberto Auricchio医学博士，是意大利Pozzuoli的Telethon遗传与医学研究所(TIGEM)科学主任（2024年4月任命）、那不勒斯"费德里科二世"大学医学遗传学教授、欧洲基因与细胞治疗学会(ESGCT)主席（2024年10月当选）。他是AAVantgarde的创始人兼首席科学官，这是一家开发视网膜疾病基因治疗的生物技术公司。 Auricchio博士国际公认为遗传性视网膜疾病AAV基因治疗的欧洲领导者。他的研究专注于使用腺相关病毒载体开发视网膜和代谢疾病的基因治疗方法。他对Luxturna (voretigene neparvovec)的临床开发做出了重要贡献，这是首个FDA批准用于人类的基因治疗（2017年）。Auricchio团队与那不勒斯的Francesca Simonelli教授合作进行了RPE65基因治疗试验的意大利部分，招募了5名意大利Leber先天性黑蒙症患者。他2009年在Lancet上的出版物证明了年龄依赖性治疗效果，为支持早期儿科干预以获得最佳治疗效果提供了关键证据。 Auricchio开创了用于大基因递送的双AAV载体系统，解决了AAV包装容量限制（约4.7kb）的主要技术挑战。他实验室2023年在Molecular Therapy上的出版物证明，编码人MYO7A的双AAV8载体改善了Usher综合征1B型小鼠模型的视网膜缺陷，并在灵长类动物中建立了安全性。这个技术平台扩展到Stargardt病（ABCA4基因，7kb）和其他大基因视网膜疾病，构成AAVantgarde临床管线的科学基础。 2024年4月，经过国际专家委员会的全球搜索，Auricchio在Andrea Ballabio服务30年后接任TIGEM科学主任。作为ESGCT主席，他塑造欧洲基因治疗政策和研究方向。Auricchio发表了150多篇同行评审出版物，拥有多项AAV基因治疗应用国际专利。他获得了美国基因与细胞治疗学会杰出新研究者奖和"Arrigo Recordati"国际科学研究奖。他正在进行的临床工作包括领导黏多糖贮积症VI型的I/II期试验，计划于2026年以更高剂量恢复。