Chiesi Group Bets $115 Million on Arbor Biotechnologies for Potential One-Time Gene Therapy
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Chiesi Global Rare Diseases is making a significant leap into genetic medicine, committing up to $115 million to partner with Arbor Biotechnologies and develop a potential one-time treatment for primary hyperoxaluria type 1 (PH1), a rare and often debilitating inherited disease. This collaboration marks a strategic shift for the Italy-based Chiesi Group, signaling a commitment to curative therapies and expanding beyond its established portfolio of small molecules and enzyme replacement treatments.
Expanding Beyond Traditional Therapies
In the five years since establishing its rare disease division in Boston, Chiesi has secured regulatory approvals for 10 therapies. However, company leadership recognized the need to explore new modalities to address unmet patient needs. “We felt like we were kind of falling behind a little bit by not being able to offer cures for patients,” a senior official stated. “So from our perspective, this is another important tool in the set of solutions that we want to bring in a definitive way to patients in the future.” The partnership with Arbor Biotechnologies, headquartered in Cambridge, Massachusetts, represents that next tool – CRISPR-based gene-editing.
Targeting Primary Hyperoxaluria Type 1 with ABO-101
The core of the deal centers around Arbor’s lead asset, ABO-101, a gene-editing therapy designed to treat PH1. This genetic disorder stems from a deficiency in an enzyme crucial for breaking down oxalate, a compound produced by the liver. The resulting buildup of oxalate leads to kidney stones and, ultimately, can cause end-stage renal disease requiring organ transplantation. However, even a kidney transplant is not a permanent solution, as the underlying liver-based issue continues to produce excess oxalate, eventually damaging the new organ.
Currently approved treatments for PH1, such as Alnylam Pharmaceuticals’ Oxlumo and Novo Nordisk’s Rivfloza, utilize small-interfering RNA to reduce oxalate production. While effective, these therapies require chronic administration – injections every three months or monthly, respectively. ABO-101 offers the promise of a one-time treatment, potentially freeing patients from the burden of lifelong therapy.
In Vivo Gene Editing: A New Frontier
Arbor’s approach to gene editing distinguishes it from earlier CRISPR therapies. Initial applications of CRISPR involved ex vivo editing, where cells are modified in a lab and then infused back into the patient. ABO-101 utilizes in vivo editing, delivering its genetic cargo directly into the patient’s body using a lipid nanoparticle that specifically targets the liver. This innovative delivery system allows the therapy to “knock out” the gene responsible for the oxalate-producing enzyme.
“If you think about one-and-done approaches as a parent, if my child had a chronic disease, I would much prefer to make the disease go away so they can live their life and do what they need to do and not have to have this burden of disease hanging over them for the rest of their lives,” explained Arbor CEO Devyn Smith.
Streamlining Treatment and Expanding Pipeline
Beyond the potential for a durable effect, the Arbor therapy also avoids the need for a conditioning regimen – a common requirement for first-generation gene-editing medicines. This regimen involves using toxic drugs to prepare the patient’s body for treatment, posing significant challenges, particularly for children. Because ABO-101 edits genes directly within the patient, this pre-treatment step is unnecessary.
The broader biotech landscape for in vivo gene-editing includes companies like Editas Medicines, Intellia Therapeutics, Mammoth Biosciences, Precision Biosciences, and Scribe Therapeutics, all of whom have established partnerships. Arbor, while previously focused on ex vivo collaborations, was receptive to a partnership for ABO-101 after an 18-month due diligence process. According to a company release, partnering with Chiesi Global Rare Diseases provides access to expertise and resources crucial for navigating the rare disease space. This collaboration allows Arbor to focus on expanding its pipeline, including three preclinical programs targeting amyotrophic lateral sclerosis (ALS).
Financial Terms and Clinical Development
Chiesi Global Rare Diseases is initiating the alliance with upfront and near-term payments totaling up to $115 million. Arbor Biotechnologies is eligible to receive up to $2 billion in additional milestone payments, plus royalties on future product sales.
ABO-101 is currently undergoing a Phase 1/2 clinical trial with a target enrollment of 23 patients. Arbor will remain the trial sponsor, but Chiesi will collaborate on the study and lead future clinical tests. The agreement also grants Chiesi the option to leverage Arbor’s gene-editing platform to develop additional liver-targeted therapies for rare diseases, with specific targets remaining undisclosed. While timelines for clinical trial readouts are not yet available, both companies emphasize a commitment to expedited development.
“Patients can’t wait for new solutions — that drives both organizations,” a company representative stated. “So we’re going to be expeditious and efficient in the future clinical development.”
