Chiesi Group Enters into Worldwide Licensing Agreement with Key2Brain AB to Advance Blood-Brain Barrier-Crossing Therapies for Lysosomal Storage Disorders
Collaboration will leverage Key2Brain’s technology to develop blood-brain barrier-crossing enzyme replacement therapies for lysosomal storage disorders
Agreement is built on a partnership initiated 2022, supporting continued development led by positive in vivo proof-of-concept data demonstrating the potential of Key2Brain's technology
PARMA, Italy and STOCKHOLM, June 11, 2025 (GLOBE NEWSWIRE) -- Chiesi Group and 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.
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 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 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 and 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 brain-targeting 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.
About Chiesi Group
Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.
By changing its legal status to a Benefit Corporation in Italy, the US, France and Colombia, Chiesi’s commitment to creating shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, Chiesi is part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035.
With 90 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees. The Group’s research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden.
About Chiesi Global Rare Diseases
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring voice to underserved people in the health care system.
About Key2Brain
Key2Brain is a Swedish biotechnology company dedicated to developing next generation brain targeting therapeutics utilizing its proprietary blood-brain barrier-crossing (BBB-crossing) technology. The technology enables efficient brain uptake and broad distribution of CNS-therapeutics, utilizing small and flexible antibody fragments (VHHs). Key2Brain’s VHHs can be integrated into a wide range of therapeutic molecules.
The pipeline is being developed internally and in partnerships where Key2Brain’s team leverages extensive industry experience in biologics and brain-targeting drugs, accelerating the translation from discovery to the clinic. Key2Brain was founded 2020 and is based in Stockholm.
About Key2Brain’s technology platform
Key2Brain’s technology enables efficient brain uptake and broad distribution of therapeutics, utilizing small (<15kDa) engineered single domain VHH antibodies with monovalent specificity for the Transferrin Receptor (TfR). The technology provides opportunities for the development of next generation brain targeting across a diverse range of therapeutic areas.
Key2Brain’s VHH technology can be efficiently integrated into or combined with therapeutic molecules without impacting either the disease target specificity, or the binding of transferrin to TfR. The modular and flexible platform is being applied to peptides, proteins, enzymes, and oligonucleotides.
Key2Brain’s VHHs can be expressed in both prokaryotic and eukaryotic systems and purified using commercial large-scale affinity purification systems. The proprietary platform includes species cross-reactive (human/primate/mouse) as well as human/primate specific binders together with a humanized TfR mouse model for translational studies.
Chiesi Global Rare Diseases Media Contact
Sky Striar
LifeSci Communications
sstriar@lifescicomms.com
Key2Brain Media Contact
Benjamin Nordin, CBO
Key2Brain
benjamin.nordin@key2brain.se
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