Usher Syndrome, Type 1B

MILAN, Dec. 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 announces an oral presentation at the FLORetina Annual Meeting, to be held December 5-8 in Florence (Italy). At this conference, safety results at 60 days for the two first subjects with advanced retinitis pigmentosa due to Usher syndrome 1B dosed in the LUCE-1 study will be presented by Prof. Francesca Simonelli, Principal Investigator for this study. This is the first administration of a dual AAV gene therapy in Ophthalmology. Oral Presentations details: Presentation Date/Time: 7 December 2024, 10.50-10.54 h CETPresenter: Prof. Francesca SimonelliSession title: Multicentric longitudinal study in Usher Syndrome IB: disease course and implication for gene therapySession: Inherited retinal diseases treatments About the AAVB-081 programAAVB-081 is an intra-retinal AAV8-based dual hybrid product targeting MYO7A-associated Usher syndrome (USH1B). AAVantgarde’s dual hybrid platform uses two AAV8 vectors, each containing one half of an expression cassette encoding for the Myo7A gene and works at the cell nucleus level, recombining the two halves of the transgene back into a single one within the cell. This technology translates into an efficient recombination that generates therapeutically meaningful protein levels in animal models. About the LUCE-1 TrialLUCE-1 is a Phase 1/2 multicenter, open-label, dose escalation study investigating safety, tolerability and preliminary efficacy of 3 dose levels of dual AAV8.MYO7A (AAVB-081) administered subretinally in subjects with retinitis pigmentosa associated with Usher Syndrome Type 1B. For further information, please refer to: https://clinicaltrials.gov/study/NCT06591793?term=AAvantgarde&rank=2 About Usher syndrome type 1BUsher syndrome type 1B (Usher1B) is an inherited disease that affects the retina and the inner ear. Usher1B is caused by mutations in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kb long and is therefore too large to fit inside a standard AAV vector. Approximately 20,000 patients in the U.S. and E.U. have Usher1B. These children are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. Although there are surgical treatments available to treat deafness in these patients, there are no treatments available to treat progressive vision loss and blindness in these patients. 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: info@avvantgarde.com

MILAN, Oct. 28, 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, is pleased to congratulate Professor Alberto Auricchio, the Company’s Founder and Chief Scientific Officer (CSO), on his appointment as President and Board member of the European Society of Gene and Cell Therapy (ESGCT). The ESGCT Board is composed of researchers and clinicians representing a broad range of European Countries. Members are elected by nomination and vote at the Annual General Meeting. Election to the Board of ESGCT is a great honor and recognizes individuals who have demonstrated outstanding professional achievement and commitment to service. As well as being CSO of AAVantgarde, Professor Auricchio is Scientific Director of TIGEM and Professor of Medical Genetics at University “Federico II” in Naples, Italy. He is co-author of over 140 peer-reviewed publications and inventor of several patents related to the use of viral vectors for gene therapy and has received the Outstanding New Investigator Award of the American Society of Gene Therapy and the International Prize for Scientific Research “Arrigo Recordati”. Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde said, “Congratulations to Alberto on this important and well-deserved recognition of his outstanding scientific contributions to the field of gene therapy. We are privileged and honoured to work with him as Founder and Chief Scientific Officer of AAVantgarde.” About AAVantgarde BioAAVantgarde Bio is a clinical stage, 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 is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Dr. Magda Blanco – Head of Corporate Development, AAVantgardeEmail: info@aavantgarde.com

MILAN, Oct. 08, 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 announces three oral presentations at the European Society of Gene & Cell Therapy 31st Annual Meeting (ESGCT), to be held October 22-25 in Rome. AAVantgarde is validating its platform technology in two inherited retinal diseases with clear unmet need, with Usher syndrome type 1B already in the clinic and Stargardt advancing towards clinical development. At this conference, we will be presenting NHP preclinical data from our Stargardt’s program and the process development, analytics and manufacture of the dual vectors to enable the clinical supply for our programs. In addition, our Scientific Founder, Professor Alberto Auricchio, TIGEM Scientific Director, will give a presentation on expediting AAV-based gene therapies. Oral Presentations details: Presentation Date/Time: Tuesday 22 October 2024, 14.00-16.30 h CESTSession title: ExpEditing AAV gene TherapySession Room: Plenary HallFinal abstract number: INV01 Presentation Date/Time: Tuesday 22 October 2024, 17.00-19.30 h CESTSession title: Safety and expression of intein-based Dual AAV8.ABCA4 in the non-human-primate retinaSession Room: Meeting room 2Final abstract number: OR017 Presentation Date/Time: Friday 25 October 2024, 11.00 – 13.00 h CESTSession title: Production of dual AAV vectors for the delivery of large genesSession Room: Plenary HallFinal abstract number: INV73 About AAVantgardeAAVantgarde Bio is a clinical stage, 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 is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Magda Blanco – Head of Corporate Development AAVantgardeEmail: info@avvantgarde.com