Arbor Biotechnologies to Present Preclinical Data for ABO-101 in PH1 at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting

  • Data from oral presentation supported ABO-101 IND and CTA approval for clinical assessment in primary hyperoxaluria type 1 (PH1) in the Phase 1/2 redePHine study in US and UK (NCT06839235)
  • Preclinical data demonstrate highly specific and durable editing of HAO1 with long-lasting reduction of urinary oxalate levels out to one-year post-single dose administration of ABO-101
  • Non-human primate studies confirm preclinical efficacy and tolerability of ABO-101 with efficient editing of HAO1, reduced GO enzyme activity, and no clinical signs or adverse events
  • Progeny studies demonstrate the lack of germline transmission of the intended ABO-101 edit to Hao1 in over 500 pups

CAMBRIDGE, MA – May 13, 2025 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today presents IND- and CTA-enabling data for its lead clinical program, ABO-101 in primary hyperoxaluria type 1 (PH1) at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, Louisiana.

“We are excited to showcase this robust preclinical data package which supported the IND clearance and CTA approval in the UK for our first clinical program” said Dan Ory, M.D., Chief Medical Officer at Arbor. “This data increases our confidence in the potential for ABO-101 to provide a long-term clinically-meaningful treatment for PH1 patients, as we begin enrollment of our Phase 1/2 redePHine clinical study evaluating ABO-101 for the treatment of PH1, a rare genetic disorder with high unmet need”.

In an oral presentation, Arbor showcased data supporting the therapeutic potential of ABO-101 for PH1 and its clinical evaluation in the redePHine clinical study (NCT06839235). The presented data demonstrated durable and efficient dose-dependent in vivo editing of Hao1 in the Agxt KO mouse model of PH1, resulting in a corresponding reduction of approximately 40% in urinary oxalate levels that was sustained out to 1-year post-administration of a single dose of ABO-101. Administration of ABO-101 to juvenile mice resulted in editing and oxalate reduction that persisted into adulthood. An additional study evaluating the potential of treated female mice to transmit the edit to their offspring demonstrated no transmission through the germline in over 500 pups.

Data from non-human primate (NHP) studies confirmed the editing efficiency and tolerability of ABO-101 with no clinical signs or adverse events. Moreover, ABO-101 editing was shown to be highly specific to HAO1 in both primary human hepatocytes (PHH) and human splenic endothelial cells (HSEC) with no off-target editing detected above 0.07% at >1,500 potential sites, in an assay with a 10-fold greater sensitivity than reported industry standards for CRISPR nucleases.

Further details on the off-target assessment of ABO-101 and the editing profile of Arbor’s Type V nucleases will be included in a poster presentation during the Thursday poster session.

 

Details for the oral presentation are as follows:

Oral Presentation Title: ABO-101, a Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1, is Efficacious and Well Tolerated in NHPs and Results in High Fidelity Editing in Primary Hepatocytes

Abstract Number: 44

Session: Gene and Cell Therapy for Metabolic Diseases

Session Date and Time: Tuesday, May 13, 2025, 1:30-3:15 PM CDT

Location: Room 278-282

Presenter: Tia DiTommaso, PhD

 

Details for the poster presentation are as follows:

Poster Title: Type V CRISPR Nuclease Edit Patterns Enable Highly Sensitive Off-target Detection

Abstract Number: 1637

Session: Thursday Poster Reception

Session Date and Time: Thursday, May 15, 2025, 5:30-7:00 PM CDT

Location: Poster Hall, Hall I2

Presenter: Ivan Kristanto

 

About ABO-101

ABO-101 is a novel, investigational gene editing medicine designed to be a one-time liver-directed gene editing treatment that results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). 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. 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.

About Arbor Biotechnologies, Inc.

Arbor Biotechnologies™, a clinical stage, 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, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, 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 its 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
prusconi@nulldeerfieldgroup.com