CAMBRIDGE, Mass., May 02, 2023 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced that three abstracts were selected for poster presentation at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting being held from May 16-20, 2023, in Los Angeles, CA.
“We look forward to presenting results from several of our preclinical studies at ASGCT later this month, which highlight the growing breadth and depth of our editing capabilities, spanning knockdown+ to novel precision editing,” said John Murphy, Ph.D., Chief Scientific Officer of Arbor Biotechnologies. “Among our poster presentations, we will be providing early results with our lead product candidate, ABR-001—a novel type V CRISPR Cas, which we are developing for the treatment of primary hyperoxaluria, or PH. PH is a rare, life-threatening condition that prevents the kidneys from filtering fluids and waste products from the body effectively, often resulting in end-stage renal disease. We are highly encouraged by these preclinical results, which demonstrate that in vivo editing of the HAO1 gene with ABR-001 significantly reduced toxic oxalates in a mouse model of PH Type 1. In addition, we look forward to describing our work on engineering a small nuclease to be a highly active enzyme with therapeutic utility, as well as our ongoing efforts to optimize type V nuclease-based RNA-Templated editors.”
Poster Presentation Details
Abstract Title: Discovery and Engineering of a Miniature CRISPR-Cas Type V-L System
Poster Session Date/Time: Wednesday, May 17, 2023 at 12:00 p.m. PT / 3:00 p.m. ET
Abstract Number: 534
Abstract Title: In Vivo Editing of the HAO1 Gene with ABR-001, a Novel Type V CRISPR Cas, Leads to Significant Reduction in Toxic Oxalate in a Mouse Model of Primary Hyperoxaluria Type 1
Poster Session Date/Time: Friday, May 19, 2023 at 12:00 p.m. PT / 3:00 p.m. ET
Abstract Number: 1611
Abstract Title: RNA-Templated Editing Using Type V CRISPR Effectors
Poster Session Date/Time: Friday, May 19, 2023 at 12:00 p.m. PT / 3:00 p.m. ET
Abstract Number: 1346
All abstracts for the ASGCT Annual Meeting are available on ASGCT’s website.
About Arbor Biotechnologies®
Arbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry to date. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, our proprietary discovery engine is focused on discovering and evolving technologies that enable gene knockout, RT editing, precise excisions and large insertions into endogenous loci. Leveraging our wholly owned nucleases as the chassis for genetic modification, we have the unique ability to work backward from disease pathology and choose the optimal editors or combination of editors for the disease in question, with a focus on areas of high unmet need. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology. For more information, please visit: arbor.bio
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