Chiesi Group and Arbor Biotechnologies announced an exclusive global collaboration and license agreement aimed at developing treatments for rare liver diseases on October 6th. Per the terms of the agreement, Chisei will receive exclusive rights to develop Arbor’s clinical stage program ABO-101, a liver-directed gene editing treatment aimed at treating primary hyperoxaluria type 1, or PH1. The deal also includes a multi-target option agreement for the use of Arbor’s advanced gene editing platform technology to develop additional novel liver-targeted therapies for rare diseases.

According to a company press release, Arbor will receive upfront and near-term payments of up to $115M, as well as up to $2 billion in total milestone payments and up to low double-digit tiered royalties. Arbor and Chiesi will collaborate on the ongoing Phase I/II redePHine clinical study of ABO-101 in PH1, an open-label global multi-center dose escalation study to evaluate safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of a single dose of ABO-101 in participants with PH1.

PH1 is an ultra-rare genetic disease that can cause recurring kidney stones, kidney damage, and eventually lead to end-stage kidney disease and systemic oxalosis. According to the release, two siRNA therapies are available for lifelong treatment, but currently the only cure for PH1 is a dual liver and kidney transplant.

“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,” said Devyn Smith, PhD, CEO, Arbor Biotechnologies, in the release. “Chiesi brings a strong track record in rare disease innovation, combined with our platform of advanced gene editors, we aim to deliver significant solutions that can redefine care for patients living with PH1 and other rare genetic diseases.”

“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,” said Giacomo Chiesi, Executive Vice President, Chiesi Global Rare Diseases, in the release. “While this path holds immense promise, we know there is still a long journey ahead, and much to learn. That’s why we are proud to partner with Arbor Biotechnologies, a leader in the gene editing space with proven expertise in clinical development.  This strategic collaboration brings together complementary strengths and reinforces our unwavering mission: to bring lasting hope to patients and their families through meaningful innovation.”