Chiesi bets big on Arbor's gene editing therapy for rare kidney disease

Chiesi Group is paying up to $115M for global rights to Arbor Biotechnologies’ early-stage gene editing therapy targeting primary hyperoxaluria type 1 (PH1), a rare kidney disorder.

Why it matters: This deal could bring a one-time, potentially lifelong treatment for PH1 patients, offering an alternative to current therapies that require ongoing injections.

Backstory: PH1 is a genetic condition causing excess oxalate production, leading to kidney stones and organ failure. Existing drugs, like Oxlumo and Rivfloza, offer relief but require frequent dosing. Arbor’s ABO-101 aims to fix the disease at the genetic level in a single dose.

Big picture: As funding for genetic therapies tightens, deals like this highlight continued faith in the long-term promise of gene editing, especially for rare diseases. Regulators are also signaling support, with new guidance to speed approvals for such treatments.

What’s next?: The deal could bring up to $2B for Arbor in the form of future payments and royalties, and hands Chiesi the option to license more liver-targeting gene therapies.