Nedosiran

Chiesi has struck a licensing deal potentially worth over $2.1 billion with Arbor Biotechnologies to advance gene editing programmes for rare liver and kidney disorders, led by Arbor's experimental therapy ABO-101 in primary hyperoxaluria type 1 (PH1).The agreement gives the Italian pharma exclusive rights to ABO-101 for PH1 and the option to use Arbor's knockout and reverse transcriptase editing technology to advance additional rare disease targets. In return, Arbor will receive up to $115 million in upfront and near-term payments, plus potential development and commercial milestones worth up to $2 billion.ABO-101 is designed as a one-time liver-directed gene-editing therapy intended to permanently disable the HAO1 gene and, in turn, reduce PH1-associated oxalate production. The overproduction of oxalate by the liver and eventual buildup of oxalate crystals in the kidney and other organ systems can cause recurring kidney stones, kidney damage and eventually lead to end-stage kidney disease and systemic oxalosis.Arbor's candidate uses a lipid nanoparticle system licensed from Acuitas Therapeutics to deliver mRNA expressing a novel Type V CRISPR Cas12i2 nuclease, paired with a guide RNA to target HAO1.Early study underwayWhile two siRNA therapies are available for lifelong treatment — Alnylam Pharmaceuticals' Oxlumo (lumasiran) and Novo Nordisk's Rivfloza (nedosiran) approved in 2020 and 2023, respectively — the only cure for PH1 is a dual liver and kidney transplant.Arbor started the Phase I/II redePHine trial in June assessing ABO-101's safety, pharmacodynamics and early efficacy in up to 23 participants with PH1. The open-label study is enrolling adults first in a dose-escalation phase before expanding to paediatric patients once a recommended dose is established. Primary completion is set for early 2029, according to ClinicalTrials.gov, and participants will be monitored long-term for durability and potential off-target effects. Chiesi and Arbor plan to jointly oversee the clinical programme."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 Arbor CEO Devyn Smith.

Chiesi Group has formed an alliance with Arbor Biotechnologies that gives the Italian pharmaceutical company ownership of an experimental gene editing treatment for a rare kidney condition.Under the terms of the deal announced Monday, Chiesi will pay Arbor up to $115 million in upfront and near-term payments for global rights ABO-101, which is being studied in a disease called primary hyperoxaluria type 1 and is currently in an early-stage clinical trial.The collaboration also gives Chiesi the option to license other liver-targeting gene editing treatments from Arbor for rare diseases. The biotechnology startup stands to receive up to $2 billion in downstream payments, as well as royalties, if a variety of unspecified milestones are met.In an interview with BioPharma Dive, Devyn Smith, Arbors CEO, said his company partnered with Chiesi because it is very committed to rare disease drug development.We didn't want someone to do the partnership, and then a year later, they change their mind strategically and move on to whatever the next thing is, he said.Chiesi also represented an attractive partnering option because of its success growing revenue in recent years, Smith said. The group has inked alliances with other biotechs, too, such as Gossamer Bio, and acquired others, including Amryt Pharma.Devyn Smith is the CEO of Arbor Biotechnologies.Permission granted by Arbor BiotechnologiesThe partnership with Arbor is driven by the potential of gene editing to transform care for patients, said Giacomo Chiesi, the executive vice president of the companys rare disease division.We share a clear alignment in therapeutic focus and values, which gives us confidence that together we can advance this work responsibly and effectively for patients.Primary hyperoxaluria type 1, or PH1, is a genetic disease that, according to some estimates, affects between1 to 3 out of every million people in the U.S. and Europe.The condition occurs when the body produces too much of a substance known as oxalate, which can lead to kidney stones, renal failure and organ damage.Two medicines are currently approved to treat PH1,Alnylam Pharmaceuticals Oxlumo and Novo Nordisk's Rivfloza, and both work by disrupting oxalate production. Both, however, require frequent injections, whereas Arbors therapy is meant to be a one-time treatment with lifelong impact.Developers of genetic medicines have struggled to attract funding of late, as investors have shown a preference towards simpler drugmaking methods with broader market potential. But U.S. regulators have voiced a desire to speed the development of gene therapies for rare conditions, and recently, published draft guidance outlining accelerated approval pathways and new clinical trial designs.The recent success of a bespoke gene editing treatment custom-made for a critically ill baby with an ultra-rare conditionis proof that regulators are open to the modality as a tool to address and fix chronic disease, Smith said.There is a unique opportunity to continue to think differently in this space, he said. '