Chiesi Group and Arbor Biotechnologies have formed an exclusive partnership and licensing deal for the latter’s investigational gene editing medicine ABO-101 fpr primary hyperoxaluria type 1 (PH1), a rare, lifelong genetic disease.
The companies also agreed to a multitarget option to use Arbor’s gene editing platform tech for the development of new therapies for rare liver conditions.
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Chiesi will gain exclusive rights for the development and commercialisation of ABO-101 to treat PH1. It also receives the option to employ Arbor’s knockout (KO) and reverse transcriptase (RT) editing technologies for further rare disease targets.
Arbor is set to obtain up to $115m in upfront and near-term payments, and could potentially accrue up to $2bn in milestone payments throughout the development process, along with receiving up to low double-digit tiered royalties on sales.
The two companies are also set to collaborate on the therapy’s Phase I/II redePHine clinical trial in those with PH1.
Arbor Biotechnologies CEO Devyn Smith stated: “We’re proud to join forces with Chiesi, a company that shares our deep commitment to improving outcomes for patients with rare and life-threatening diseases.”
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By GlobalDataA specialised division called Chiesi Global Rare Diseases works on the advancement and distribution of treatments for rare and ultra-rare disorders.
Its executive vice-president Giacomo Chiesi stated: “This collaboration marks a transformative moment — not just for us, but for the entire rare disease community. It reflects our commitment to working towards more comprehensive therapeutic options. Achieving this means looking beyond current approaches and exploring the potential of gene editing.”
ABO-101 is designed as one-time liver-directed gene editing therapy to permanently inactivate the hydroxyacid oxidase 1 (HAO1) gene in the liver, minimising oxalate production associated with PH1.
The therapy is yet to secure approval from the US Food and Drug Administration (FDA) or any other regulatory body.
In March 2025, Arbor secured $73.9m funding to advance a gene editing therapeutic pipeline for liver and central nervous system diseases.
