Arbor Biotechnologies to Present Foundational Data for PH1 and CNS Programs at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting
- Oral presentation to include preclinical data supporting the clinical advancement of ABO-101 for the treatment of primary hyperoxaluria type 1
- Four poster presentations collectively demonstrate advancement of company’s differentiated CNS gene editing programs and showcase capabilities of wholly owned gene editing technologies
CAMBRIDGE, MA – April 28, 2025 – Arbor Biotechnologies, Inc., a biotechnology company discovering and developing the next generation of genetic medicines, today announced five upcoming presentations at the 2025 American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting, taking place May 13-17 in New Orleans, Louisiana.
Data presented at ASGCT will showcase Arbor’s progress in applying its nuclease discovery and development capabilities and proprietary gene editing tools to fuel differentiated gene editing programs to address metabolic and CNS diseases with high unmet need, in addition to its continued advancement of novel nucleases for a sustainable platform of gene editing technologies for therapeutic application.
In an oral session, Arbor will showcase data supporting the therapeutic potential of ABO-101 for the treatment of primary hyperoxaluria type 1 (PH1) and its clinical evaluation in the redePHine clinical study (NCT06839235). Presented data will demonstrate robust in vitro and in vivo pre-clinical assessments, in vivo editing efficacy and durability for ABO-101, and rigorous off-target assessment. A separate poster presentation will detail how the unique profile of Arbor’s type V nucleases results in 10-fold greater sensitivity in off target detection relative to the industry standard.
Arbor will also present data demonstrating continued progress of its CNS programs, including the demonstration of in vivo proof-of-concept data supporting the first type V gene editing program for SOD1-ALS as well as the first disclosure of its therapeutic gene editing approach to address Angelman syndrome.
An additional presentation will detail the identification and characterization of novel small reverse transcriptase (RT) editors deliverable via a single adeno-associated virus (AAV) vector, unlocking therapeutic applications in disease relevant tissues beyond the liver.
Details for the presentations 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
Session: Gene and Cell Therapy for Metabolic Diseases
Session Date and Time: Tuesday, May 13, 2024, 1:30-3:15 PM CDT
Location: Room 278-282
Presenter: Tia DiTommaso, PhD
Poster Title: A compact RT editor built from the metagenome
Session: Wednesday Poster Reception
Session Date and Time: Wednesday, May 14, 2024, 5:30-7:00 PM CDT
Location: Poster Hall, Hall I2
Presenter: Dan Brogan, PhD
Poster Title: Disruption of UBE3A-ATS by Gene Editing with a Type V CRISPR-Cas Enzyme as a treatment for Angelman Syndrome
Session: Thursday Poster Reception
Session Date and Time: Thursday, May 15, 2024, 5:30-7:00 PM CDT
Location: Poster Hall, Hall I2
Presenter: Adele Bubnys, PhD
Poster Title: Preclinical development of AAV mediated gene editing for SOD1-ALS using a Type V CRISPR-Cas enzyme
Session: Thursday Poster Reception
Session Date and Time: Thursday, May 15, 2024, 5:30-7:00 PM CDT
Location: Poster Hall, Hall I2
Presenter: Amrutha Pattamatta, PhD
Poster Title: Type V CRISPR nuclease edit patterns enable highly sensitive off-target detection
Session: Thursday Poster Reception
Session Date and Time: Thursday, May 15, 2024, 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