September 16, 2024 — AAVantgarde Bio (AAVantgarde), an Italy-based global biotechnology company, announced that the first subject has been dosed in the LUCE-1 trial, a Phase 1/2 open label clinical trial to assess safety and tolerability of AAVB-081, using the company’s proprietary dual hybrid gene therapy platform to deliver MYO7A protein in subjects with retinitis pigmentosa related to Usher Syndrome Type 1B (USH1B).
This dual-AAV technology for large gene delivery comes from the research of Prof. Alberto Auricchio at TIGEM, an international research institute based in Naples that is owned and managed by the Telethon Foundation. AAVantgarde is a spin-off of TIGEM, and has an exclusive license for the AAV-dual technology for inherited retinal diseases.
The first subretinal gene therapy procedure in the LUCE-1 study was supervised by Prof. Francesca Simonelli, Head of the Ophthalmology Unit at the University Hospital of Campania “Luigi Vanvitelli” (Naples). This hospital is one of 3 clinical sites for the Phase 1/2 trial. Dr. Simonelli is global Chief Investigator at the University Hospital of Campania “Luigi Vanvitelli” that is the lead dosing site for the LUCE-1 study.
I am delighted to be involved as Principal Investigator in this first-in-human Phase 1/2 clinical study of AAV-081 for patients with retinitis pigmentosa related to USH1B. Through this innovative program, we aim to revolutionize our approach to understanding and treating these underserved patients. We are poised to generate robust evidence that will not only advance scientific knowledge, but also directly impact patient care.
Prof. Simonelli.
It is an honour to work closely with Prof. Simonelli in this first-in-human clinical trial and leverage her great expertise in developing gene therapies for ophthalmology indications, with a goal of bringing this potentially novel therapy to the USH1B patient community as expeditiously as possible.
Dr. Jayashree Sahni
CMO of AAVantgarde
It is very exciting to have our dual hybrid technology finally tested clinically in an ophthalmology indication. I am very hopeful that all these years of research prove their value in helping these patients that have such a high unmet need.
Prof. Alberto Auricchio
Founder and CSO of AAVantgarde
We are honoured to have Prof. Simonelli as Principal Investigator for this clinical study aimed at providing hope for 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 extensive experience in this space will be invaluable to the successful development of this novel therapy.
Dr. Natalia Misciattelli
CEO of AAVantgarde