May 3, 2024 — 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. Francesca Simonelli, at the 9th Annual Retinal Cell and Gene Therapy Innovation Summit, being held today in Seattle.
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).
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.
Prof. Francesca Simonelli
Head of Ophthalmology at the University of Campania Luigi Vanvitelli (Naples)
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. 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.
Dr. Natalia Misciattelli
CEO of AAVantgarde
Presentation details:
Title: Design of a Phase 1/2 clinical trial using a dual vector strategy for the treatment of MYOA7-related Usher syndrome (USH1B)
Session Title: Session 2 – Pre-clinical Gene Therapy
Date/Time: May 3, 2024 from 9:35 AM to 9.50 AM PDT
Contact
Dr. Magda Blanco – Head of Corporate Development AAVantgarde
Email: m.blanco@aavantgarde.com