AAVantgarde announces its innovative clinical study design for its lead program in Usher 1B
2024年5月3日 - 4:00PM
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,”
said Prof. Francesca Simonelli, Head of Ophthalmology at
the University of Campania Luigi Vanvitelli (Naples).
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. 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.”
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
About AAVantgarde
BioAAVantgarde 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. 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
Contact:Magda Blanco – Head of Corporate
Development AAVantgardeEmail: m.blanco@aavantgarde.com