Usher Syndrome, Type Ib

MILAN, Italy, Sept. 16, 2024 (GLOBE NEWSWIRE) -- 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,” said Prof. Simonelli. “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.” Dr. Jayashree Sahni, CMO of AAVantgarde said, “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.” Prof. Alberto Auricchio, Founder and CSO of AAVantgarde added, “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.” 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 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.” 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. 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:Julia Wilson - JW CommunicationsPhone: +44 (0) 7818 430877Email: juliawilsonuk@gmail.com

MILAN, Italy, May 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 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

DURHAM, N.C., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ATSN-101, the company’s investigational gene therapy being evaluated in an ongoing Phase I/II clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1. RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. If a Biologics License Application for ATSN-101 for the treatment of LCA1 is approved by the FDA, Atsena may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred. “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “The FDA designations that have been granted to ATSN-101 not only emphasize the tremendous need for a treatment for patients with LCA1, but also the potential for our subretinal gene therapy to be a major advance in reversing pediatric blindness.” Positive 12-month safety and efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101 will be presented at the 47th Annual Macula Society Meeting, which is being held February 7-10, 2024, in Palm Springs, CA. ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment. Details of the presentation are as follows: Title: Twelve-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1) Session: Inherited Retinal Dystrophy I (Treatment Trials) Date and Time: Wednesday, February 7, 2024, 6:04 p.m. PST Presenter: Christine Nichols Kay, MD About GUCY2D-associated Leber congenital amaurosis (LCA1) LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1. About Atsena Therapeutics Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 12 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit atsenatx.com. Media Contact: Tony Plohoros 6 Degrees (908) 591-2839 tplohoros@6degreespr.com Business Contact: info@atsenatx.com