ABO-101 / Arbor Biotech - LARVOL DELTA

Arbor Biotechnologies to Present Foundational Data for PH1 and CNS Programs at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting (GlobeNewswire) - "Arbor Biotechnologies...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....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."

P1/2 data • Preclinical • Genetic Disorders • Metabolic Disorders

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 (ASGCT 2025) - "Taken together, these results demonstrate signals of efficacy in mice and NHPs and safety at the genome-wide and organismal level, further supporting the advancement of ABO-101 into the clinic as a potential treatment for PH1. Disease Focus of Abstract:Metabolic Disease"

Metabolic Disorders • Nephrology

Arbor Biotechnologies Announces $73.9 Million Series C Financing to Advance Novel Gene Editing Therapeutics (GlobeNewswire) - "Arbor Biotechnologies...announced the closing of a $73.9 million Series C financing to support the advancement of its pipeline of novel gene editing therapeutics targeting diseases in the liver and central nervous system (CNS)....The proceeds will support clinical development of the company's lead therapeutic candidate, ABO-101, in primary hyperoxaluria type 1 (PH1) and progression to IND/CTA filing of its first-in-class programs, including an RT editing program for a rare liver disease and a program targeting amyotrophic lateral sclerosis (ALS)."

Financing • Amyotrophic Lateral Sclerosis • Rare Diseases

Development of ABO-101, A Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1 (IPNA 2025) - "We found on-target editing to be >90%, compared with off-target editing detected at only 3 sites, all of which were below 0.07% in a highly sensitive assay, suggesting that ABO-101 editing is highly potent and specific to HAO1 . Conclusions Taken together, these results provide in vivo pharmacology data to support a gene editing approach in PH1, enabling the advancement of ABO-101 into the clinic as an investigational therapy for PH1."

Metabolic Disorders • Nephrology • Renal Disease

Development of ABO-101, a Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1 (KIDNEY WEEK 2024) - "Taken together, these results provide in vivo proof of pharmacology for a gene editing approach and support further advancement of ABO-101 towards the clinic as a potential treatment for PH1."

Metabolic Disorders • Nephrology • Renal Disease