MILAN, Italy, May 03, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announced the presentation of the clinical trial design of the Company’s lead program in Usher 1B by the program’s Principal Investigator, Prof.

意大利米兰，2024年5月3日（环球通讯社）--AAVantgarde Bio（AAVantgarde），一家临床阶段的意大利国际生物技术公司，拥有两个专有的腺相关病毒（AAV）载体平台，用于大基因传递，今天宣布由该项目的首席研究员教授介绍该公司在Usher 1B的领先项目的临床试验设计。

Francesca Simonelli, at the 9th Annual Retinal Cell and Gene Therapy Innovation Summit, being held today in Seattle..

今天在西雅图举行的第九届年度视网膜细胞和基因治疗创新峰会上，弗朗西丝卡·西蒙内利（FrancescaSimonelli）。。

The LUCE-1 clinical study is a first-in-human Phase 1/2 clinical study designed in collaboration with Prof. Simonelli and other leading experts in the field. Leveraging AAVantgarde’s proprietary Dual Hybrid platform, this study aims to provide robust evidence supporting the effectiveness and safety profile of the Company’s lead program, AAVB-081, that addresses the retinitis pigmentosa derived from MYO7A-related Usher syndrome (USH1B)..

LUCE-1临床研究是与Simonelli教授和该领域其他领先专家合作设计的首次人体1/2期临床研究。利用AAVantgarde专有的双杂交平台，这项研究旨在提供强有力的证据支持该公司领先项目AAVB-081的有效性和安全性，该项目解决了由MYO7A相关Usher综合征（USH1B）引起的色素性视网膜炎。。

“I am delighted to be presenting the pre-clinical and clinical activities that paved the way to the design of this first-in-human Phase 1/2 clinical study at the Summit. Through this innovative design, we aim to revolutionize our approach to understanding and treating Usher 1B patients. We are poised to generate robust evidence that will not only advance scientific knowledge, but also directly impact patient care,” said Prof.

“我很高兴在峰会上介绍临床前和临床活动，这些活动为这项首次人体1/2期临床研究的设计铺平了道路。通过这项创新设计，我们的目标是彻底改变我们理解和治疗Usher 1B患者的方法。我们准备产生强有力的证据，不仅可以提高科学知识，而且可以直接影响患者护理，”教授说。

Francesca Simonelli, Head of Ophthalmology at the University of Campania Luigi Vanvitelli (Naples)..

弗朗西斯卡·西蒙内利（FrancescaSimonelli），坎帕尼亚大学（University of Campania LuigiVanvitelli）（那不勒斯）眼科主任。。

Dr. Natalia Misciattelli, CEO of AAVantgarde added “We are honoured to have Prof. Simonelli as Principal Investigator for this first in human Phase 1/2 clinical study aimed at providing hope for underserved USH1B patients that have no therapeutic options to prevent them from losing their sight. Prof.

AAVantgarde首席执行官Natalia Misciattelli博士补充说：“我们很荣幸让Simonelli教授担任这项首次人类1/2期临床研究的首席研究员，该研究旨在为服务不足的USH1B患者提供希望，这些患者没有治疗选择来防止他们失明。

Simonelli is a pioneer in gene therapy in ophthalmology and her valuable experience in this space will greatly help us to the successful development of this novel therapy.”.

西蒙内利是眼科基因治疗的先驱，她在这一领域的宝贵经验将极大地帮助我们成功开发这种新型疗法。”。

Presentation details:

演示详细信息：

Title: Design of a Phase 1/2 clinical trial using a dual vector strategy for the treatment of MYOA7-related Usher syndrome (USH1B)

标题：使用双载体策略设计治疗MYOA7相关Usher综合征（USH1B）的1/2期临床试验

Session Title: Session 2 – Pre-clinical Gene Therapy

课程名称：课程2–临床前基因治疗

Date/Time: May 3, 2024 from 9:35 AM to 9.50 AM PDT

日期/时间：2024年5月3日，太平洋时间上午9:35至上午9:50

About AAVantgarde Bio

关于AAVantgarde Bio

AAVantgarde Bio is a clinical stage, Italian headquartered, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues.

AAVantgarde Bio是一家总部位于意大利的临床阶段国际生物技术公司，该公司开发了两个专有的腺相关病毒（AAV）载体平台，以解决AAV载体的基因治疗货运能力限制。AAVantgarde平台可用于将大基因传递到眼组织和非眼组织。

Co-founded by Professor Alberto Auricchio at TIGEM (Telethon Institute of Genetics and Medicine) in Naples, Italy, and Telethon Foundation, AAVantgarde will initially validate the platform in the clinic in two inherited retinal diseases with clear unmet need. For more information, please visit: www.aavantgarde.com.

由意大利那不勒斯TIGEM（Telethon遗传与医学研究所）的Alberto Auricchio教授和Telethon基金会共同创立，AAVantgarde将在两种遗传性视网膜疾病的临床上初步验证该平台，这些疾病显然没有得到满足。有关更多信息，请访问：www.aavantgarde.com。

Contact:

联系人：

Magda Blanco – Head of Corporate Development AAVantgarde

Magda Blanco–AAVantgarde公司发展主管

Email: m.blanco@aavantgarde.com

电子邮件：m.blanco@aavantgarde.com