Chiesi Group Enters Strategic Partnership with Arbor Biotechnologies for Rare Disease Gene Editing Programs

Rare Daily Staff

Chiesi Group entered into an exclusive global collaboration and license agreement for Arbor’s clinical-stage program ABO-101 in the ultra-rare liver disease primary hyperoxaluria type 1, as well as a multi-target option agreement for the use of Arbor’s advanced gene-editing platform technology to develop other novel liver-targeted therapies for rare diseases.

Under the terms of the agreement, Chiesi will pay Arbor up to $115 million in upfront and near-term payments. In addition, Arbor is eligible to receive up to $2 billion in total milestone payments and up to low double-digit, tiered royalties. Chiesi will receive exclusive rights to develop and commercialize ABO-101 for primary hyperoxaluria type 1, and the option to leverage Arbor’s knockout and reverse transcriptase editing technology to advance additional rare disease targets. Arbor and Chiesi will collaborate on the ongoing phase 1/2 redePHine clinical study of ABO-101 in PH1.

Primary hyperoxaluria type 1 (PH1) is a lifelong genetic disease in which a mutation in the AGXT gene leads to an enzyme deficiency in the liver, resulting in an overproduction of oxalate by the liver and eventual buildup of oxalate crystals in the kidney and other organ systems. As the disease progresses, it can cause recurring kidney stones, kidney damage, and eventually lead to end-stage kidney disease and systemic oxalosis. While two siRNA therapies are available for lifelong treatment, the only cure for PH1 is a dual liver and kidney transplant.

ABO-101 is an experimental gene-editing medicine designed to be a one-time, liver-directed gene-editing treatment that potentially results in a permanent loss of function of the HAO1 gene in the liver to reduce PH1-associated oxalate production. ABO-101 consists of a lipid nanoparticle, licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel type V CRISPR Cas12i2 nuclease, and an optimized guide RNA that specifically targets the human HAO1 gene.

“This collaboration marks a transformative moment — not just for us, but for the entire rare disease community,” said Giacomo Chiesi, executive vice president, Chiesi Global Rare Diseases. “It reflects our commitment to working toward more comprehensive therapeutic options. Achieving this means looking beyond current approaches and exploring the potential of gene editing.”

Photo: iacomo Chiesi, executive vice president, Chiesi Global Rare Diseases