New partnership targets CNS symptoms in ultra-rare diseases

Chiesi Group and Key2Brain have announced a global license agreement to accelerate the development of two recombinant enzyme replacement therapies (ERTs) capable of crossing the blood-brain barrier (BBB). The targeted therapies are aimed at addressing the neurological aspects of two ultra-rare lysosomal storage disorders (LSDs) – alpha-mannosidosis (aMann) and Krabbe disease (KD).

The collaboration will look to confront an urgent medical need – providing treatment options for patients suffering from rare neurodegenerative diseases for which no approved therapies exist. These programs will be led by Chiesi Global Rare Diseases, the Chiesi Group’s specialist division for rare and ultra-rare conditions.

Building a sustainable pipeline in rare diseases

“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. “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, Chiesi obtains a worldwide, royalty-bearing license to develop and commercialise two BBB-penetrant ERTs from Key2Brain. These therapies are built on a prior research collaboration focused on producing a recombinant alpha-mannosidase enzyme with the capability to reach the central nervous system.

A platform for broader innovation

The deal includes a framework for expanding the collaboration to cover additional enzymes in the future. This could allow for broader application of Key2Brain’s BBB-crossing technology and significantly expands Chiesi’s potential in developing central nervous system-targeting treatments for other LSDs.

Chiesi will assume responsibility for all research, development, and global commercialisation. In return, Key2Brain will receive an upfront payment, along with potential milestone-based and tiered royalty payments.

“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 R&D, 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.”

Advancing brain-targeted ERTs with proven technology

Key2Brain’s proprietary technology is designed to overcome one of the most significant challenges in treating neurodegenerative diseases – the impermeability of the BBB to large-molecule drugs such as enzymes. This partnership represents a significant backing of the platform’s therapeutic promise.

“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,” said Dr Elisabet Sjöström, Founder and CEO, Key2Brain. “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.”

A platform for the future

The partnership will continue to advance the aMann-K2B program and initiate new preclinical projects targeting other LSDs. With Chiesi’s rare disease expertise and Key2Brain’s enabling platform, this alliance is well-positioned to deliver innovative solutions for some of the most devastating and underserved neurological conditions.