Table of Contents
Around 1 in every million people is born with primary hyperoxaluria type 1, a rare liver-enzyme defect that progressively destroys the kidneys – and for end-stage patients, the standard of care is still a combined liver-kidney transplant.
Arbor Biotechnologies’ CRISPR-based gene editor ABO-101 is being trialled as a one-time in vivo fix, with the redePHine Phase 1/2 study enrolling up to 23 adult and paediatric patients at Mayo Clinic and additional sites in the US, UK and Europe.
Co-founded by CRISPR pioneer Feng Zhang, Arbor is building a proprietary platform of next-generation gene editors aimed at diseases the first wave of CRISPR drugs couldn’t reach – starting with the liver and the brain. Lead candidate ABO-101 is a one-time in vivo therapy designed to knock out the gene responsible for oxalate overproduction; the FDA accepted its IND in December 2024, granted Orphan Drug and Rare Pediatric Disease designations in February 2025, and the first patient was dosed at Mayo Clinic in July 2025. Smith, who took the helm in 2021, has since steered Arbor through a $73.9 million Series C in March 2025 to fund redePHine and the broader pipeline.
At Meeting on the Med 2026, reporting for Onyx, Federico Citterich sat down with Devyn Smith to find out more.
A special thanks to our Onyx Live sponsors:
Cellares - delivering cell therapy manufacturing excellence at scale across a global network of IDMO smart factories https://www.cellares.com/
Comments