Usher Syndromes

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

The resulting company will operate under the name Opus Genetics and the Nasdaq ticker symbol (IRD) RALEIGH, N.C., Oct. 25, 2024 /PRNewswire/ -- The RD Fund (Retinal Degeneration Fund) – the venture arm of the Foundation Fighting Blindness – announces the all-stock acquisition of Opus Genetics, Inc. "Opus" (private) by Ocuphire Pharma, Inc., (Nasdaq: OCUP) a clinical-stage ophthalmic biopharmaceutical company. The companies have combined under the name of Opus Genetics to create a transformative biotech company committed to being a leader in the development of gene therapies for the treatment of inherited retinal diseases (IRDs). The combined company is trading on the Nasdaq under the ticker symbol "IRD." The RD Fund launched Opus in the fall of 2021 to develop gene therapies for the treatment of inherited retinal diseases (IRDs). The RD Fund led the seed financing for Opus as the first internally conceived spinout of the Fund to further the Foundation's mission. With the full support of the Foundation's and Fund's Board of Directors, the company was co-founded and initially managed by Ben Yerxa, PhD, Rusty Kelley, PhD, Peter Ginsberg, and Jason Menzo along with its scientific founders, Jean Bennett, MD, PhD and Junwei Sun from the University of Pennsylvania and Eric Pierce, MD, PhD, from Harvard and Massachusetts Eye and Ear. "The initial seed funding allowed Opus to build a pipeline of early-stage preclinical assets, including advancing OPGx-LCA5 into a Phase 1/2 trial where 6-month data demonstrated safety and visual improvement in early onset retinal degeneration," said Rusty Kelley, managing director of the RD fund and a founding director of the company. "We thank our major donors, including those that enabled project-based funding from the Foundation Fighting Blindness, for helping us advance Opus's pipeline while weathering a significant biotech downturn," added Jason Menzo, Opus co-founder and CEO of the Foundation Fighting Blindness. The acquisition of Opus by Ocuphire is a key milestone as it represents the first RD Fund portfolio company to enter the public markets. The milestone further validates the venture philanthropy model of derisking assets - making them more attractive to institutional investors and the public markets. "That a highly skilled and experienced team - including former Opus executives and directors Ben Yerxa, Jean Bennett, and Adrienne Graves - will advance the Opus portfolio with a commitment to being a leader in developing genetic medicines for the treatment of IRDs accelerated our approval of the transaction," said Kelley. About the RD Fund The RD Fund (Retinal Degeneration Fund), the venture investment arm of the Foundation Fighting Blindness, has a dual mission: accelerate the approval of therapies for retinal diseases and provide alternative revenue sources to further the Foundation's mission. Established in 2018, the RD Fund prioritizes investments in companies around the globe developing therapies that are close to clinical testing. For more information, visit RDFund.org. About the Foundation Fighting Blindness Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research. The Foundation has raised more than $915 million toward its mission of accelerating research for preventing, treating, and curing blindness caused by the spectrum of blinding retinal diseases including: retinitis pigmentosa, macular degeneration, and Usher syndrome. Visit FightingBlindness.org for more information. Media Contact: Chris Adams 410-423-0585 [email protected] SOURCE Foundation Fighting Blindness WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

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