John Murphy, PhD, and Tia DiTommaso, PhD, on Preclinical Evidence for PH1 Gene Therapy

“We showed that we can see a really durable effect with a single administration of ABO-101 out to a year, and also in juvenile mice, through their development into adults.”

Arbor Biotechnologies recently began the clinical evaluation of ABO-101, a lipid nanoparticle (LNP)-delivered Cas12i2-based gene editing therapy intended to treat primary hyperoxaluria type 1 (PH1), a rare genetic disease caused by a mutation in the AGXT gene. Following the clearance of the investigational new drug (IND) application by the FDA for the phase 1/2 redePHine clinical trial (NCT06839235), Arbor presented some of the preclinical data leading to the IND application’s clearance at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, held May 13 to 17, 2025, in New Orleans, Louisiana.

At the conference, CGTLive® spoke with Tia DiTommaso, PhD, the senior director of preclinical pharmacology Arbor Biotechnologies, who presented the data, and John Murphy, PhD, the chief scientific officer of Arbor Biotechnologies, about the company’s work and the findings presented.

Murphy briefly highlighted Arbor’s broader central nervous system (CNS) pipeline, including genomic medicine approaches for amyotrophic lateral sclerosis and Angelman syndrome. DiTommaso then discussed the key points of her presentation, which was centered on ABO-101's preclinical data. She pointed out that the therapy demonstrated strong target specificity in primary cells and substantial reduction of oxalate levels in mice lasting up to a year with a single dose in adult mice and into adulthood for juevinile mice. DiTommaso noted that studies in nonhuman primates (NHPs) also showed promising safety and tolerability.

Both DiTommaso and Murphy emphasized the importance of the durability and safety data seen so far. Looking ahead, Murphy noted that learnings from the program will guide Arbor’s future activities in the CNS field and beyond. Notably, Arbor announced the dosing of the first patient in redePHine in July 2025.

Click here to view more coverage of the 2025 ASGCT Annual Meeting.

REFERENCE
1. Arbor Biotechnologies announces first patient dosed at mayo clinic in the redephine phase 1/2 study of ABO-101, an investigational gene editing treatment for primary hyperoxaluria type 1. News release. Arbor Biotechnologies, Inc. July 30, 2025. Accessed September 2, 2025. https://arbor.bio/arbor-biotechnologies-announces-first-patient-dosed-at-mayo-clinic-in-the-redephine-phase-1-2-study-of-abo-101-an-investigational-gene-editing-treatment-for-primary-hyperoxaluria-type-1/