Usher Syndrome, Type 1B

MILAN, Dec. 09, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), an Italy-based global biotechnology company, presented preliminary safety data on the first two subjects 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). The 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), lead dosing site for the LUCE-1 study. Safety data for two low visual acuity (LVA) subjects (S1 & S2) in the low dose (LD) cohort was presented. The best corrected visual acuity (BCVA) returned to pre-treatment level within 2 weeks in both subjects. All adverse events (AEs) were mild and resolved without further intervention. To date, no dose-limiting toxicities have been observed. The independent Data Safety Monitoring Board has provided recommendation to continue the trial as planned. “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,” said Prof. Simonelli. “These preliminary safety results are very encouraging, and we are progressing with the dose escalation in this study. AAVB-081 shows a favourable safety profile, with no significant immune response and has potential for visual improvement, that I hope will help greatly improving the quality of life of these patients.” Dr. Natalia Misciattelli, CEO of AAVantgarde added “We are honoured to have Prof. Simonelli as Principal Investigator for this clinical study aimed at providing hope for USH1B patients that currently have no therapeutic options to prevent sight loss. We are excited about these preliminary results and look forward to sharing data of our dual AAV platform technology for MYO7A retinitis pigmentosa related to Usher syndrome in relevant forums.” 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 Trial (NCT06591793)LUCE-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. You can find further information on the LUCE-1 study in the link below:LUCE-1 clinical study 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:Dr. Magda Blanco – Head of Corporate DevelopmentEmail: info@aavantgarde.com

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