Chiesi Group and Arbor Biotechnologies Announce a Global Strategic Partnership to Develop Novel Rare Disease Gene Editing Programs
- Exclusive collaboration and license agreement for the development and global commercialization of Arbor’s clinical-stage gene editing program, ABO-101 for primary hyperoxaluria type 1 (PH1)
- Multitarget research collaboration and option agreement for Arbor’s innovative gene editing technologies for the development of additional rare liver disease targets
- Arbor eligible to receive up to $115M in upfront and near-term payments, along with $2B in potential development, regulatory and commercial milestones and low double-digit tiered royalties for products licensed under the agreements
PARMA, ITALY & CAMBRIDGE, MA, USA – October 6, 2025 – Chiesi Group, an international research-oriented biopharmaceutical company, together with Arbor Biotechnologies, Inc., a next-generation biotechnology company discovering and developing innovative approaches in genetic medicines, today announced an exclusive global collaboration and license agreement for Arbor’s clinical stage program ABO-101 in PH1 together with a multitarget option agreement for the use of Arbor’s advanced gene editing platform technology to develop novel liver targeted therapies for rare diseases.
At Chiesi Group, these programs are spearheaded by Chiesi Global Rare Diseases, the Group’s dedicated business unit focused on research, development, and commercialization of therapies for rare and ultra-rare conditions.
“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. “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.”
“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. 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,” said Devyn Smith, PhD, CEO of Arbor Biotechnologies.
Under the terms of the transaction, Chiesi will receive exclusive rights to develop and commercialize ABO-101 for PH1 and the option to leverage Arbor’s knockout (KO) and reverse transcriptase (RT) editing technology to advance additional rare disease targets, providing Arbor upfront and near-term payments of up to $115M. In addition, Arbor is eligible to receive up to $2B in total milestone payments and up to low double-digit tiered royalties. Arbor and Chiesi will collaborate on the ongoing Phase 1/2 redePHine (NCT06839235) clinical study of ABO-101 in PH1.
“Genomic medicines offer vast potential to impact the lives of patients around the world, especially those living with rare genetic diseases. 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,” said Dan Ory, MD, Chief Medical Officer of Arbor Biotechnologies.
About Primary Hyperoxaluria Type I (PH1)
Primary hyperoxaluria type 1 is an ultra-rare lifelong genetic disease, where 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 (ESKD) and systemic oxalosis. While two siRNA therapies are available for lifelong treatment, the only cure for PH1 is a dual liver and kidney transplant.
About ABO-101
ABO-101 is a novel, investigational 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 is currently being evaluated for PH1 in the redePHine Phase 1/2 clinical study (NCT06839235). ABO-101 consists of a lipid nanoparticle (LNP), licensed from Acuitas Therapeutics, encapsulating messenger RNA expressing a novel Type V CRISPR Cas12i2 nuclease and an optimized guide RNA which specifically targets the human HAO1 gene. ABO-101 has not been approved for any use by the FDA or any other regulatory agency.
About the redePHine Study
The Phase 1/2 redePHine study is 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 primary hyperoxaluria type 1. The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, pediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring program to comply with local and national requirements. For more information on redePHine (NCT06839235), please visit clinicaltrials.gov.
About Chiesi Group
Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.
With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.
For more information visit: www.chiesi.com
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
For more information visit: www.chiesirarediseases.com.
About Arbor Biotechnologies, Inc.
Arbor Biotechnologies™, a clinical stage, next-generation gene editing company based in Cambridge, MA, is advancing a pipeline of novel gene editing therapeutics to address a wide range of genetic conditions – from the ultra-rare to the most common genetic diseases. The company’s unique suite of optimized gene editors, which is capable of approaches ranging from gene knockout, excisions, reverse transcriptase editing, and large gene insertion, goes beyond the limitations of early editing technologies to unlock access to new gene targets and has fueled a robust pipeline of first-in-class assets focused on diseases of high unmet need. With Arbor’s clinical program, ABO-101 for the treatment of primary hyperoxaluria type 1, the company continues to focus its research and development efforts on genomic diseases of the liver and CNS for which there are no existing functional cures. For more information, please visit: arbor.bio.
Chiesi Global Rare Diseases Media Contact:
Sky Striar
LifeSci Communications
sstriar@nulllifescicomms.com
Arbor Biotechnologies Media Contact:
Peg Rusconi
Deerfield Group
prusconi@nulldeerfieldgroup.com