Arbor Biotechnologies to Highlight New Preclinical Data Supporting Advancement of Pipeline Programs and Knock-In Applicability of Type V Nucleases at Upcoming Medical and Scientific Meetings

• 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, California
• 31^stAnnual European Society of Gene and Cell Therapy (ESGCT), October 22-25 in Rome, Italy
• ALS One 7^thAnnual 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 7^th 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 7^thAnnual 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 Biotechnologies^TM, 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 Rusconi
Deerfield Group
[email protected]

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