Arbor Biotechnologies to Present Data Supporting Therapeutic Programs in PH1 and ALS, and the Discovery of a Novel Type V Nuclease at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

–   Oral and poster presentations showcase data demonstrating pipeline progress and continued advancement of Arbor’s pipeline programs toward the clinic

–   Oral presentation with data validating aspects of Arbor’s approach to the discovery and development of unique gene editors, outlines the identification and optimization of a novel type V nuclease, ABR-004

–   Chief Technology Officer David R. Cheng to participate in symposium highlighting progress and applications of generative AI for cell and gene therapies

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) — Arbor Biotechnologies^®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced four upcoming presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place May 7-11 in Baltimore, Maryland.

Arbor will present in vivo NHP data supporting clinical 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. In a separate presentation, Arbor will detail data 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 also will present data supporting its end-to-end nuclease development platform in a third presentation outlining the discovery and optimization of a unique, compact nuclease, termed ABR-004. Proof-of-concept data in non-human primates show potent, therapeutically relevant silencing of PCSK9 in vivo with the novel nuclease, signaling opportunities for broader therapeutic applications.

David Cheng, Chief Technology Officer at Arbor, will discuss the significance and applications of recent advances in generative AI on the discovery and development of new genomic medicines during the scientific symposium, The Impact of Generative Artificial Intelligence (AI) on CGT.

Together, the suite of presentations will demonstrate the utility of Arbor’s proprietary discovery and development approach for enabling efficient identification and optimization of novel nucleases. The data also underscores progress in advancing its robust pipeline of genetic medicines toward clinical evaluation.

About Arbor Biotechnologies®
Arbor Biotechnologies is a next-generation gene editing company based in Cambridge, MA. Combining the promise of CRISPR with advanced computational AI-driven discovery, high throughput screening, and robust protein engineering approaches, our co-founders Feng Zhang and David Walt laid the groundwork for our proprietary discovery engine, which has yielded an extensive toolbox of gene editors, far exceeding the number of editors published in the literature to date. We envision a future of gene editing that extends beyond simple knockdowns to include precision writing, precise excisions and large insertions. This affords us the potential to treat a broad spectrum of patients, from ultra-rare to the most common genetic diseases. Guided by a deep understanding of the molecular basis of disease and our access to a unique suite of optimized editors, we are rapidly advancing our discovery-stage programs with an initial focus on genomic diseases of the liver and CNS for which there are no existing functional cures. As we advance toward the clinic with our lead program in primary hyperoxaluria type I, we look to expand our strategic partnerships around in vivo gene editing across multiple therapeutic areas and ex vivo cell therapy programs to broaden the reach of our novel nuclease technology. For more information, please visit: arbor.bio

Media Contact:
Peg Rusconi
Verge Scientific Communications
prusconi@vergescientific.com

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