A Landmark Collaboration in Gene Therapy
Italy’s Chiesi Group and U.S.-based Arbor Biotechnologies have entered a global research and licensing collaboration valued at more than $2 billion. The agreement, announced on October 6, focuses on the development and commercialization of Arbor’s gene therapy candidate ABO-101 for the rare liver disease primary hyperoxaluria type 1 (PH1).
Terms of the Agreement
Under the terms of the deal, Chiesi will provide $115 million in upfront and near-term payments for the rights to develop and commercialize ABO-101. The agreement also includes up to $2 billion in potential development, regulatory, and commercial milestone payments, along with low double-digit tiered royalties on future product sales. In addition, Chiesi will have the option to use Arbor’s proprietary editing technologies in developing other therapies for rare diseases.
Targeting a Rare Liver Disorder
Arbor’s ABO-101 is being studied as a one-time gene editing treatment for PH1, an inherited liver disorder caused by a mutation in the AGXT gene. This mutation leads to a deficiency in an enzyme responsible for breaking down oxalate, resulting in the buildup of oxalate crystals that can cause kidney stones, kidney damage, and, in severe cases, kidney failure. PH1 accounts for around 80% of primary hyperoxaluria cases, which occur in fewer than three patients per one million people.
ABO-101 uses CRISPR-based technology to target a different gene, HAO1, which codes for glycolate oxidase—an enzyme involved in oxalate production. By disrupting this gene, the therapy is designed to suppress oxalate buildup in the liver. In preclinical research presented earlier this year, a single dose of ABO-101 reduced urinary oxalate levels in mice by 40% over a one-year period.
Clinical Trials and Future Development
Arbor has already begun clinical testing of ABO-101 in the ongoing phase 1/2 redePHine study, which aims to enroll 23 participants. The trial, which dosed its first patient on July 30, has a primary completion date projected for March 2029. Under the collaboration, Chiesi will join Arbor in supporting this clinical program.
In a statement, Giacomo Chiesi, executive vice president of Chiesi Global Rare Diseases, called the Arbor agreement “a transformative moment” for both the company and the broader rare disease community. He said the companies would look “beyond current approaches and [will explore] the potential of gene editing” to develop more comprehensive therapeutic options for patients with rare diseases.
Arbor Chief Medical Officer Dan Ory, M.D., stated, “We look forward to partnering with Chiesi’s experienced and committed team to help accelerate ABO-101 in the clinic and advance development of liver-targeted gene editing therapeutics for patients with PH1 and other rare diseases.”
The only existing curative treatment for PH1 is a combined liver and kidney transplant. Through this agreement, Chiesi gains exclusive global rights to develop and commercialize ABO-101 and access to Arbor’s editing platforms for future programs in rare diseases. Arbor remains eligible to receive milestone and royalty payments as development progresses.
