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Chiesi Group partners with Arbor Biotechnologies to advance gene editing therapies for rare diseases, focusing on primary hyperoxaluria type 1.
The collaboration and license agreement includes the development and global commercialization of ABO-101 for primary hyperoxaluria type 1 and the development of additional rare liver disease targets.
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Chiesi group and Arbor Biotechnologies announced an exclusive global collaboration and license agreement for Arbor’s clinical stage program ABO-101 in primary hyperoxaluria type 1 (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.1
"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.”
Per the terms of the agreement, Chiesi will receive exclusive rights to develop and commercialize ABO-101 for PH1 and the option to leverage Arbor’s knockout and reverse transcriptase editing technologies, aiming to advance additional rare disease targets and providing Arbor upfront and near-term payments upwards of $115 million.1
“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.
“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.
ABO-101 is an investigational gene editing medicine created to be a one-time liver-directed gene editing treatment that can potentially lead to a permanent loss of function of the HAO1 gene in the liver reducing PH1-associated oxalate production.1 ABO-101 is currently undergoing evaluation for PH1 in the redePHine Phase I and Phase II clinical study. ABO-101 consists of a lipid nanoparticle, licensed from Acuitas Therapeutics, encapsulating messenger RNA which expresses Type V CRISPR Cas12i2 nuclease, along with an optimized guide RNA specifically targeting the human HAO1 gene.1 Currently, ABO-101 is not yet approved for any use by the FDA or any other regulatory agency.1
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