ABO-101 / Abeona Therap - LARVOL DELTA

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

Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine) (clinicaltrials.gov) - P1/2 | N=23 | Recruiting | Sponsor: Arbor Biotechnologies | Not yet recruiting ➔ Recruiting

Enrollment open • Nephrology

Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine) (clinicaltrials.gov) - P1/2 | N=23 | Not yet recruiting | Sponsor: Arbor Biotechnologies

New P1/2 trial • Nephrology

Advances in CRISPR Gene Editing for Pediatric Nephrology: ABO-101 for Treatment of Primary Hyperoxaluria Type 1 (IPNA 2025) - "Sponsored by Arbor Biotechnologies"

Clinical • Nephrology • Pediatrics

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

Development of ABO-101, a Novel Gene Editing Therapy for Primary Hyperoxaluria Type 1 (ASGCT 2024) - "Here, we describe a gene editing approach to reduce the activity of the protein that makes the precursor of oxalate, thereby reducing oxalate production. We show that our gene editing therapeutic candidate, called ABO-101, is effective and safe in primates, a critical first step toward moving this promising therapeutic candidate for PH1 into clinical trials."

Genetic Disorders • Metabolic Disorders • Nephrology

Safety and Efficacy of ABX-101 in Participants Aged 18 to 50 Years of Age With Moderate to Severe Traumatic Brain Injury (clinicaltrials.gov) - P2a | N=45 | Not yet recruiting | Sponsor: Abalonex, LLC

New P2a trial • CNS Disorders • Vascular Neurology • FAP • GFAP

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 (clinicaltrials.gov) - P=N/A | N=1 | Terminated | Sponsor: Abeona Therapeutics, Inc | N=24 ➔ 1 | Trial completion date: Jun 2026 ➔ Apr 2022 | Recruiting ➔ Terminated | Trial primary completion date: Jun 2026 ➔ Apr 2022; Abeona has decided to discontinue development activities for Product AB0-101 due to a lack of drug supply and for business reasons unrelated to the product safety profile and/or signs of efficacy

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Gene Therapies • Hunter Syndrome • Lysosomal Storage Diseases

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB (clinicaltrials.gov) - P1/2 | N=11 | Terminated | Sponsor: Abeona Therapeutics, Inc | Trial completion date: Oct 2022 ➔ Apr 2022 | Active, not recruiting ➔ Terminated | Trial primary completion date: Oct 2022 ➔ Apr 2022; Abeona has decided to discontinue development activities for Product ABO-101 due to a lack of drug supply and for business reasons unrelated to the product safety profile and/or signs of efficacy

Trial completion date • Trial primary completion date • Trial termination • Gene Therapies

[VIRTUAL] Updated Results of Transpher B, a Multicenter, Single-Dose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB) (ASGCT 2021) - "In summary, intravenous administration of ABO-101 in children with MPS-IIIB was well tolerated and showed a clear biochemical response, including normalization of enzyme activity in plasma up to 6 months, improvements in CNS and systemic biomarkers, and reductions in liver volume. Longer follow-up is required to evaluate neurodevelopmental changes."

Clinical • P1/2 data • CNS Disorders • Gene Therapies • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 (clinicaltrials.gov) - P=N/A; N=24; Recruiting; Sponsor: Abeona Therapeutics, Inc; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Gene Therapies • Hunter Syndrome

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 (clinicaltrials.gov) - P; N=24; Not yet recruiting; Sponsor: Abeona Therapeutics, Inc

Clinical • New trial • Gene Therapies • Hunter Syndrome

A Long-term Follow-up Study of Patients with MPS IIIB from Gene Therapy Clinical Trials Involving the Administration of ABO-101 (rAAV9.CMV.hNAGLU) (clinicaltrialsregister.eu) - P1/2; N=24; Sponsor: Abeona Therapeutics Europe SL.

Clinical • New P1/2 trial