Chiesi Group has signed a worldwide license agreement with Swedish biotech Key2Brain to accelerate the development of blood-brain barrier (BBB)-crossing enzyme replacement therapies (ERTs) for lysosomal storage disorders, including alpha-mannosidosis and Krabbe disease.

The agreement builds on a partnership established in 2022, and will now move forward with positive in vivo proof-of-concept data for two investigational therapies. It also provides a framework for adding further ERT candidates using Key2Brain’s proprietary BBB-crossing platform.

The collaboration brings together Key2Brain’s expertise in crossing the BBB with Chiesi Global Rare Diseases’ clinical and commercial development capabilities. The companies aim to address critical unmet needs for patients affected by ultra-rare, neurodegenerative conditions for which therapeutic options remain extremely limited.

“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 at Chiesi Global Rare Diseases.

“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.”

Under the agreement, Key2Brain grants Chiesi a worldwide, royalty-bearing license to develop and commercialise two BBB-penetrating ERTs. Chiesi will fully fund all ongoing and future development activities, and Key2Brain will receive an upfront payment as well as milestone payments and tiered royalties based on sales.

The agreement includes the potential for expanding the license to include additional programs using Key2Brain’s BBB-crossing technology. This creates an opportunity to build a broader pipeline of central nervous system-directed therapies for lysosomal storage disorders and other conditions.

“This exciting collaboration continuation with Key2Brain represents a strategic opportunity to build on the progress we’ve already made together,” said Mitch Goldman, senior vice president of R&D at Chiesi Global Rare Diseases. “By combining our deep therapeutic expertise with Key2Brain’s proprietary BBB-crossing platform technology, we aim to enhance the biodistribution, efficacy and tolerability of promising therapies that have historically faced challenges reaching the central nervous system. Together, our goal is to unlock new therapeutic pathways for patients with lysosomal storage disorders, ultimately delivering meaningful and lasting innovation.”

Key2Brain’s founder and CEO, Elisabet Sjöström, added: “Expanding our relationship with Chiesi Global Rare Disease allows us to build on a clinically validated foundation to address the neurological complications of lysosomal storage disorders, including alpha-mannosidosis and Krabbe disease.

“Through this agreement, we aim to accelerate the research of potential breakthrough therapies that address these neurodegenerative conditions. We believe this partnership serves as a testament to the versatility and competitiveness of our BBB-crossing technology, unique features that are being utilized in Key2Brain’s development of our proprietary next-generation brain-targeting therapies.”

Chiesi, a certified B Corp, is one of few global companies to formalise social responsibility into its corporate structure as a Benefit Corporation. With more than 7,500 employees and affiliates in 31 countries, its Global Rare Diseases unit is focused on delivering innovation in underserved areas, particularly for rare and ultra-rare conditions.