Chiesi och Key2Brain i globalt licensavtal

Chiesi Group har ingått ett globalt licensavtal med Key2Brain för att utveckla behandlingar som kan passera blod-hjärnbarriären vid lysosomala sjukdomar.
Samarbetet bygger på en tidigare partnerskap från 2022 och fokuserar på att vidareutveckla Key2Brains teknologi, som visat lovande resultat i djurstudier. Målet är att ta fram enzymersättningsbehandlingar som effektivt når hjärnan vid dessa sällsynta, genetiska sjukdomar.

Chiesi Group and Key2Brain 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.

The agreement also includes a framework for the parties 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 BBBcrossing platform. At Chiesi Group these programs are spearheaded by Chiesi Global Rare Diseases, the Group’s dedicated business unit focused on research, development, and commercialisation 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.

“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 terms of the agreement, Key2Brain will provide Chiesi Group with a worldwide, royalty-bearing license to develop and commercialise two BBB-crossing ERTs. This agreement is built on an existing research collaboration that aimed to develop the production of a BBB-crossing recombinant alpha-mannosidase. Key2Brain will receive an upfront payment is eligible to receive development and sales-based milestone payments and tiered royalties on potential sales. Chiesi Group will fund all research, development, and subsequent commercialisation worldwide. As part of this agreement, there is also a possibility, upon mutual agreement, for the parties to expand the license to Key2Brain’s BBB-crossing technology platform for the development of additional BBB-crossing ERTs.

“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.”

Elisabet Sjöström, Ph.D., Founder and CEO, Key2Brain, said, “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 braintargeting therapies.”

This collaboration leverages Key2Brain’s proprietary BBB-crossing platform technology and Chiesi Global Rare Diseases’ capabilities and expertise in this ultra-rare disease landscape, creating a path to advance the ongoing aMann-K2B program as well as initiate new preclinical programs that apply Key2Brain’s technology to other LSDs.