Active Heart, Stroke & Blood NIHR-supported project Diabetes, Hormones & Metabolism

A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants with Primary Hyperoxaluria Type 1 (PH1) (ABO-101)

Funder NIHR (part of DHSC)
Recipient organisation NIHR Wellcome Trust Birmingham Clinical Research Facility
NIHR support Recorded as supported by this research centre
Period Mar 2025 — Nov 2026

In plain English

AI plain-English summary

A single injection of a gene-editing treatment called ABO-101 aims to permanently disable a liver enzyme that drives a rare, devastating kidney disease. People with primary hyperoxaluria type 1 (PH1) produce excess oxalate, which forms kidney stones and deposits that destroy kidney tissue. Without treatment, the disease leads to kidney failure, requiring dialysis or transplant. Current therapies only manage symptoms or slow progression. ABO-101 changes a specific gene in liver cells to stop oxalate production at its source, potentially offering a one-time, lifelong cure. This phase 1/2 trial will test safety, side effects, and whether the treatment reduces oxalate levels in both children and adults. If successful, ABO-101 could eliminate the need for repeated hospital visits, dialysis, or organ transplantation for PH1 patients. The study will also track long-term effects, checking whether oxalate suppression and side-effect profiles remain stable years after treatment. This is an early-stage human trial, so efficacy is not yet proven, but the approach represents a direct application of fundamental gene-editing science to a well-defined genetic target.

View original technical description
This study will test an investigational gene editing treatment called ABO-101, intended to help people with a rare genetic disease called primary hyperoxaluria type 1 (PH1). A gene editing treatment is one that changes a targeted gene forever, with the aim to provide lifelong benefit from a one-time treatment. ABO-101 is an investigational medication because it has not been approved by any health authorities to treat any condition. Research is ongoing to see how safe it is and if it can help people with PH1. PH1 affects both children and adults. People with PH1 produce too much oxalate. Oxalate can combine with calcium, creating kidney stones or deposits in the kidneys and injure them. If left untreated, PH1 leads to kidney failure, and patients need a kidney transplant or dialysis to stay alive. ABO-101 changes a gene in the liver that is responsible for an enzyme involved in the production of oxalate. By changing this gene in the liver, researchers expect that ABO-101 can stop the production of this enzyme and reduce oxalate production forever. This study will try to find out how safe ABO-101 is, by evaluating what side effects ABO-101 causes and how bad they are. The main study endpoint evaluates side effects related to the treatment throughout the study. How effective, meaning that if ABO-101 can reduce oxalate production, it may reduce the amount of kidney stones and kidney injury in participants. What the body does to ABO-101, and how the immune system responds to ABO-101. We will also investigate the long-term effects of ABO-101, by checking how long the side effects of ABO-101 last and if the oxalate production remains low many years after receiving ABO-101.

Researchers

Graham Lipkin (Principal Investigator)

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A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants with Primary Hyperoxaluria Type 1 (PH1)
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