Italian family-owned drugmaker Chiesi Group and Sweden’s Key2Brain today announced a worldwide license agreement to advance the development of two blood-brain barrier (BBB)-crossing recombinant enzyme replacement therapies (ERT) for lysosomal storage disorders (LSD), including alpha-mannosidosis (aMann) and Krabbe disease (KD), ultra-rare diseases that affect the central nervous system.
Additionally, the accord includes a framework for the companies to include additional enzymes into the collaboration, enabling the expansion of Chiesi’s BBB-crossing ERT portfolio and further strengthening its development capabilities. The advancement of these programs will also contribute to Key2Brain’s goal of establishing its technology as a leading BBB-crossing platform. At Chiesi Group these programs are spearheaded by Chiesi Global Rare Diseases, the group’s dedicated business unit focused on research, development, and commercialization of therapies for rare and ultra-rare conditions.
“At Chiesi Global Rare Diseases, we are deeply committed to building a sustainable pipeline in rare diseases by embracing emerging technologies that can enhance the treatment landscape,” said Giacomo Chiesi, executive vice president, Chiesi Global Rare Diseases, adding: “This agreement exemplifies that vision - working to address areas of profound unmet medical need, including neurodegenerative manifestations of ultra-rare diseases like alpha-mannosidosis and Krabbe disease. For too many families, there are still no therapeutic options. Our goal is to bring them hope where today there is none.”
This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.
Login to your accountTry before you buy
7 day trial access
Become a subscriber
Or £77 per month
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed
Chairman, Sanofi Aventis UK
Copyright © The Pharma Letter 2025 | Headless Content Management with Blaze