Arbor Biotechnologies, Inc.

Dec. 19, 2024 -- Arbor Biotechnologies™, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). The redePHine Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of ABO-101 in both adult and pediatric patients with PH1. “We are thrilled to advance ABO-101 to the clinic as we believe it has the potential to be a first-in-class treatment for PH1, a rare disease with a high unmet need, and reinforces the promise of our precisely tailored gene editing approach,” said Dan Ory, M.D., Chief Medical Officer of Arbor. “ABO-101 is supported by a strong suite of preclinical data demonstrating specific and durable in vivo editing of HAO1 and corresponding, therapeutically relevant reductions in urinary oxalate in PH1 disease models.” ABO-101 is a liver-targeted gene editing therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1). PH1 is a rare genetic disorder in which enzyme deficiencies in the liver lead to the overproduction and buildup of oxalate, resulting in kidney stones eventually leading to end stage kidney disease and systemic oxalosis. ABO-101 is designed to knock down HAO1 gene expression in the liver, thereby providing durable reduction in oxalate production. “The FDA approval of the ABO-101 IND marks a significant milestone for the hyperoxaluria community. At OHF, we are committed to partnering with industry leaders to advance care, research, and patient-focused drug development for hyperoxaluria. Arbor’s innovative CRISPR-based approach represents a groundbreaking opportunity in genomic medicine, with the potential to transform the lives of patients with PH1,” said Kim Hollander, Executive Director of The Oxalosis and Hyperoxaluria Foundation. “The initiation of this study marks an important milestone both for patients with PH1 and in the growth of our company as we begin advancing our pipeline of first-in-class programs into the clinic,” said Devyn Smith, CEO of Arbor. “There is a tremendous need for innovative approaches to treat genetic conditions – from ultra-rare to the most common genetic diseases – and we’re excited for the potential of our next-generation genetic medicines that can address a vast spectrum of devastating diseases. I applaud the hard work of our team at Arbor for getting us to this stage.” ABO-101 is a novel, investigational gene editing medicine designed to be a one-time treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. Arbor Biotechnologies™, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s lead program, ABO-101 for the treatment of primary hyperoxaluria type 1, progressing into clinical trials, the company continues to focus their research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio. The content above comes from the network. if any infringement, please contact us to modify.

BOSTON and INDIANAPOLIS, Nov. 7, 2024 /PRNewswire/ -- Genezen, a best-in-class gene therapy contract development and manufacturing organization (CDMO), is pleased to announce Dr. Pam Stetkiewicz and Stuart Auerbach have joined its Board of Directors. These board appointments bolster Genezen's strategic growth plan and come at a time of significant momentum for the organization. With over a decade of experience and state-of-the-art viral vector facilities, including the recently acquired 86,000ft2 Lexington, MA site, Genezen partners with innovator companies to develop and manufacture gene and cell therapies – from concept to commercial – on a global scale. Dr. Stetkiewicz brings significant gene therapy drug development and CMC experience to Genezen's Board through her current position as Chief Operating Officer at Arbor Biotechnologies, a next-generation gene editing company combining the promise of CRISPR with advanced computational AI-driven discovery, high throughput screening, and robust protein engineering approaches. Dr. Stetkiewicz previously held senior roles at Flagship Pioneering, Editas Medicine, and Novartis and has a PhD in Immunology from Johns Hopkins University. Dr. Stetkiewicz said, "I am excited to join the Genezen board at this critical junction in their growth and partner with the team to continue to build a best-in-class gene therapy CDMO." Mr. Auerbach brings a proven track record over the course of three decades in partnering with founders and management teams to successfully scale companies. Mr. Auerbach has been a key member of the investment team and member of the board of directors of numerous contract development and manufacturing organizations in both the therapeutics and medical device sectors. He is currently serving as a Senior Advisor at Ampersand Capital Partners, a leading healthcare private equity investor, where he was a General Partner for more than twenty years. Mr. Auerbach has a B.S. in Chemical Engineering from Columbia University and an MBA from Harvard Business School. Mr. Auerbach said, "Genezen has emerged as a leader in the gene therapy CDMO space, and I am thrilled to be working again with this first-class management team. I look forward to working together to grow another highly respected organization supporting many of the world's pre-eminent therapeutics innovators." Steve Favaloro, Chairman and CEO, said, "I am thrilled to welcome Pam and Stuart to our board at an exciting time in Genezen's development. They bring a wealth of manufacturing, drug development, and strategic insight to the board, and I am excited to partner with them to continue to build our organization." About Genezen Genezen is a best-in-class CDMO with over a decade of experience and state-of-the-art viral vector manufacturing facilities. With capabilities to work across vector-modality - from concept to commercial – Genezen helps innovator companies deliver life-saving gene and cell therapies worldwide. With flexible and customer-centric programs, Genezen tailors its partnership-model approach to all sizes and stages of organizations, from academic institutions and seed-funded businesses to large, multinational biopharma companies. For more information about Genezen, please visit genezen.com. For press inquiries, please contact: Katie Kristofic [email protected] SOURCE Genezen WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Oral presentation at ASN on lead program, ABO-101 for primary hyperoxaluria type 1 (PH1), supporting in vivo proof of pharmacology. On-track for year-end IND/CTA filing.Oral presentation at ALS One and a poster presentation at ESGCT on lead ALS program ABO-202, highlighting in vivo reversal of ALS-associated STMN2 aberrant splicing and phenotypic outcomes in human motor neurons.Poster presentation at ESGCT demonstrating targeted gene knock-in and broader applicability of type V nuclease platform for monogenic diseases. CAMBRIDGE, Mass., Oct. 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced oral and poster presentations on pipeline and early discovery programs at upcoming medical and scientific meetings: American Society of Nephrology (ASN) 2024 Kidney Week Meeting, October 23-27 in San Diego, California31st Annual European Society of Gene and Cell Therapy (ESGCT), October 22-25 in Rome, ItalyALS One 7th Annual ALS Research Symposium, November 13-15, Virtual At ASN Kidney Week, Arbor will present in vivo non-human primate (NHP) proof-of-pharmacology data supporting continued development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver. Arbor is on track to file the IND/CTA for ABO-101 in PH1 by year-end. At the ALS One 7th Annual ALS Research Symposium and at ESGCT, Arbor will detail data on its lead ALS program ABO-202, supporting genetic disruption of STMN2, preventing errors in the processing of its mRNA, and elucidating a path towards proof of concept for a therapeutic CRISPR gene editing approach for the treatment of amyotrophic lateral sclerosis (ALS). The company will also showcase novel data at ESGCT demonstrating targeted gene knock-in at an endogenous promoter achieving equivalent or improved knock-in efficiency when compared to type II nucleases. This data supports the efficiency and breadth of applicability of Arbor’s type V nucleases to monogenic diseases. Details for the presentations are as follows: ASN 2024 Kidney Week:Oral Presentation Title: Development of ABO-101, A Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1 Session Date and Time: Thursday, October 24, 2024, 4:30-6:00pm PDT Location: Room 23, Convention Center Presenter: Winston Yan, MD, PhD Abstract Number: TH-OR88 ESGCT 2024:Poster Title: Gene Editing with a Type V CRISPR-Cas Enzyme Disrupting the Aberrant Splicing of STMN2 as a Potential Treatment for ALS Session Date and Time: Poster Session 1, October 22, 7:30-9:00pm CEST Location: Forum, La Nuvola Presenter: Chee Yeun Chung, PhD Abstract Number: P0327 Poster Title: Targeted Knock-In at the Human TTR Locus Using Type V Nucleases Session Date and Time: Poster Session 3, October 24, 2:00-3:30pm CEST Location: Concourse Level 1 and Mezzanine Concourse, La Nuvola Presenter: Whitney J. French, PhD Abstract Number: P0603 ALS One 7th Annual ALS Research Symposium:Oral Presentation Title: Gene Editing with a Type V CRISPR-Cas Enzyme Disrupting the Aberrant Splicing of STMN2 as a Potential Treatment for ALS Session Date and Time: November 14, 3:40-4:00pm EDT Presenter: Priscilla D. Negraes, PhD About Arbor Biotechnologies, Inc.Arbor BiotechnologiesTM, a next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions - from the ultra-rare to the most common genetic diseases. Our unique suite of optimized gene editors goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. We are rapidly advancing our lead program in primary hyperoxaluria type 1 to the clinic and focusing our research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio Media Contact:Peg RusconiDeerfield Groupprusconi@deerfieldgroup.com