Chiesi Group and Arbor Biotechnologies Partner to Advance Gene Editing Therapies for Rare Liver Diseases

Chiesi Group and Arbor Biotechnologies have announced an exclusive global collaboration and licensing agreement to advance the development of innovative gene editing therapies for rare liver diseases, including the clinical-stage program ABO-101 for primary hyperoxaluria type 1 (PH1). The partnership also includes a multi-target option for Arbor’s advanced gene editing platform, aiming to expand therapeutic possibilities for other rare genetic conditions.

Chiesi Global Rare Diseases, the dedicated business unit of Chiesi Group, will lead the effort on behalf of the company. This strategic alliance underscores Chiesi’s growing investment in genomic medicines and its mission to deliver transformative treatments for patients with rare and ultra-rare diseases.

“This collaboration marks a transformative moment—not just for us, but for the entire rare disease community,” said Giacomo Chiesi, Executive Vice President of Chiesi Global Rare Diseases. “We are proud to partner with Arbor Biotechnologies, a leader in gene editing, to push the boundaries of what’s possible in treating devastating conditions like PH1.”

Arbor Biotechnologies will bring its proprietary knockout (KO) and reverse transcriptase (RT) gene editing technologies to the table. ABO-101, Arbor’s lead candidate, is currently being evaluated in the Phase 1/2 redePHine clinical trial (NCT06839235), which targets PH1, a rare inherited disorder that causes kidney damage due to excessive oxalate production.

“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 of Arbor Biotechnologies. “With Chiesi’s expertise in rare disease innovation and our platform of advanced gene editors, we aim to redefine care for patients living with PH1 and beyond.”

Under the terms of the agreement, Chiesi will hold exclusive global rights to develop and commercialize ABO-101 for PH1. Arbor will receive up to $115 million in upfront and near-term payments and is eligible for up to $2 billion in development, regulatory, and commercial milestones, as well as tiered royalties in the low double digits.

Dr. Dan Ory, Chief Medical Officer of Arbor Biotechnologies, highlighted the importance of the collaboration in accelerating genomic medicine development: “Genomic medicines offer vast potential to impact the lives of patients around the world. We look forward to partnering with Chiesi’s experienced team to bring ABO-101 closer to patients and expand the possibilities of gene editing in liver-targeted rare diseases.”

This collaboration is poised to strengthen both companies’ positions in the rapidly advancing field of genetic medicine, combining Arbor’s next-generation editing technology with Chiesi’s global clinical and commercial expertise in rare disease treatment.