A few months after launching its lead gene therapy into the clinic, Arbor Biotechnologies has found a European partner to come along for the ride. Italian pharma Chiesi is fertilizing Arbor’s rare disease efforts with a collaboration and license agreement potentially worth more than $2 billion, the companies announced in an Oct. 6 release.
Chiesi is shelling out $115 million in upfront and near-term payments for the rights to develop and commercialize ABO-101, a liver-targeted gene therapy for the ultrarare genetic disease primary hyperoxaluria type 1 (PH1), along with the option to use Arbor’s editing technology in pursuit of other rare disease treatments.
Arbor can harvest up to $2 billion in milestone payments through the deal, according to the release, along with low double-digit tiered royalties.
The agreement brings Parma, Italy-based Chiesi on board ABO-101’s ongoing phase 1/2 trial, which dosed its first patient July 30.
“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,” Arbor Chief Medical Officer Dan Ory, M.D., said in the release.
PH1 is caused by a mutated version of the alanine-glyoxylate aminotransferase (AGXT) gene. AGXT is vital for the liver’s ability to break down oxalate and filter it out of the blood, preventing oxalate buildup to potentially toxic levels. Oxalate accumulation may cause symptoms like bloody urine, frequent kidney stones, loss of bladder control and, in serious cases, kidney failure.
Rather than targeting AGXT, Arbor’s gene therapy is designed to use CRISPR to direct an enzyme to cut a different gene, HAO1, and eliminate its function. The enzyme made by HAO1, glycolate oxidase, is a key part of the biochemical pathway that leads to oxalate production in PH1. In May, Arbor presented data at the American Society of Gene & Cell Therapy conference showing that one-time ABO-101 treatment reduced oxalate in the urine of mice by 40% for a year.
HAO1 is also the target of Alnylam Pharmaceuticals' Oxlumo (lumasiran), which blocks the gene’s activity using regular injected doses of small interference RNA rather than a one-time CRISPR knockout. Oxlumo was approved in November 2020 as the first medicine for PH1 and was followed three years later by Novo Nordisk’s RNAi med Rivfloza (nedosiran), which targets a different gene.
With ABO-101, Arbor and Chiesi are betting there will be a market for a one-time gene editing fix for PH1. Arbor raised nearly $74 million in series C funding back in March to support ABO-101, following a substantial $215 million series B in 2021.
Counting CRISPR pioneer Feng Zhang, Ph.D., among its co-founders, Arbor’s pipeline also boasts a handful of preclinical prospects, including partnerships with Vertex Pharmaceuticals, Allogene Therapeutics and EdiGene. The Massachusetts-based gene editing outfit was also a Fierce 15 honoree in 2022.